|
tepotinib |
09/11/2019 |
|
Patients with metastatic non-small cell lung cancer (NSCLC) harboring a MET exon 14 skipping alteration who have progressed following platinum-based chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 09/11/2019
Approval Date: |
| Indication: Patients with metastatic non-small cell lung cancer (NSCLC) harboring a MET exon 14 skipping alteration who have progressed following platinum-based chemotherapy |
| Category: Cancer |
|
|
capmatinib (INC280) |
09/6/2019 |
|
First-line treatment of patients with MET exon14 skipping mutated non-small cell lung cancer (NSCLC) |
Cancer |
|
Date of BT Designation Disclosure: 09/6/2019
Approval Date: |
| Indication: First-line treatment of patients with MET exon14 skipping mutated non-small cell lung cancer (NSCLC) |
| Category: Cancer |
|
|
Nirogacestat |
08/29/2019 |
|
Adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis |
Cancer |
|
Date of BT Designation Disclosure: 08/29/2019
Approval Date: |
| Indication: Adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis |
| Category: Cancer |
|
|
NKTR-214 (bempegaldesleukin) with Opdivo (nivolumab) |
08/23/2019 |
|
Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery |
Cancer |
|
Date of BT Designation Disclosure: 08/23/2019
Approval Date: |
| Indication: Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery |
| Category: Cancer |
|
|
Peginterferon Lambda |
08/20/2019 |
|
Hepatitis delta virus (HDV) infection |
Infectious Disease |
|
Date of BT Designation Disclosure: 08/20/2019
Approval Date: |
| Indication: Hepatitis delta virus (HDV) infection |
| Category: Infectious Disease |
|
|
Calquence (acalabrutinib) |
08/14/2019 |
|
Monotherapy treatment for adult patients with chronic lymphocytic leukemia (CLL), one of the most common types of leukaemia in adults |
Cancer |
|
Date of BT Designation Disclosure: 08/14/2019
Approval Date: |
| Indication: Monotherapy treatment for adult patients with chronic lymphocytic leukemia (CLL), one of the most common types of leukaemia in adults |
| Category: Cancer |
|
|
DCR-PHXC |
07/15/2019 |
|
Primary hyperoxaluria type 1 (caused by a mutation in the AGXT gene) |
Other |
|
Date of BT Designation Disclosure: 07/15/2019
Approval Date: |
| Indication: Primary hyperoxaluria type 1 (caused by a mutation in the AGXT gene) |
| Category: Other |
|
|
Avexitide |
06/17/2019 |
|
Post-bariatric hypoglycemia (PBH) |
Other |
|
Date of BT Designation Disclosure: 06/17/2019
Approval Date: |
| Indication: Post-bariatric hypoglycemia (PBH) |
| Category: Other |
|
|
Aliqopa (copanlisib) |
05/30/2019 |
|
Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies |
Cancer |
|
Date of BT Designation Disclosure: 05/30/2019
Approval Date: |
| Indication: Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies |
| Category: Cancer |
|
|
LN-145 |
05/22/2019 |
|
Patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 05/22/2019
Approval Date: |
| Indication: Patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy |
| Category: Cancer |
|
|
SER-109 |
04/29/2019 |
|
Recurrent C. difficile infection |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/29/2019
Approval Date: |
| Indication: Recurrent C. difficile infection |
| Category: Rare Inherited Disorders |
|
|
LJPC-0118 |
04/24/2019 |
|
Patients with severe malaria |
Infectious Disease |
|
Date of BT Designation Disclosure: 04/24/2019
Approval Date: |
| Indication: Patients with severe malaria |
| Category: Infectious Disease |
|
|
elafibranor |
04/19/2019 |
|
Patients with primary biliary cholangitis (PBC) |
Other |
|
Date of BT Designation Disclosure: 04/19/2019
Approval Date: |
| Indication: Patients with primary biliary cholangitis (PBC) |
| Category: Other |
|
|
inebilizumab |
04/18/2019 |
|
Patients with neuromyelitis optica spectrum disorder (NMOSD) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/18/2019
Approval Date: |
| Indication: Patients with neuromyelitis optica spectrum disorder (NMOSD) |
| Category: Rare Inherited Disorders |
|
|
PB2452 |
04/8/2019 |
|
Reversal agent for the antiplatelet drug ticagrelor |
Other |
|
Date of BT Designation Disclosure: 04/8/2019
Approval Date: |
| Indication: Reversal agent for the antiplatelet drug ticagrelor |
| Category: Other |
|
|
Selumetinib (AZD6244) |
04/1/2019 |
|
Pediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic and/or progressive, inoperable plexiform neurofibromas (PN) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/1/2019
Approval Date: |
| Indication: Pediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic and/or progressive, inoperable plexiform neurofibromas (PN) |
| Category: Rare Inherited Disorders |
|
|
Tibsovo (ivosidenib) and azacitidine |
03/27/2019 |
|
newly diagnosed patients with IDH1-mutant acute myeloid leukemia (AML) ≥75 years old or are ineligible for intensive induction chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 03/27/2019
Approval Date: |
| Indication: newly diagnosed patients with IDH1-mutant acute myeloid leukemia (AML) ≥75 years old or are ineligible for intensive induction chemotherapy |
| Category: Cancer |
|
|
AXS-05 |
03/27/2019 |
|
Major Depressive Disorder (MDD) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 03/27/2019
Approval Date: |
| Indication: Major Depressive Disorder (MDD) |
| Category: Rare Inherited Disorders |
|
|
Venclexta (venetoclax) + Gazyva (obinutuzumab) |
03/15/2019 |
05/15/2019 |
Adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) |
Cancer |
|
Date of BT Designation Disclosure: 03/15/2019
Approval Date: 05/15/2019 |
| Indication: Adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) |
| Category: Cancer |
|
|
AT-GAA (ATB200 + AT2221) |
02/26/2019 |
|
Patients with late onset Pompe disease |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 02/26/2019
Approval Date: |
| Indication: Patients with late onset Pompe disease |
| Category: Rare Inherited Disorders |
|
|
BLU-667 |
02/26/2019 |
|
Patients with RET fusion-positive NSCLC that has progressed on platinum-based chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 02/26/2019
Approval Date: |
| Indication: Patients with RET fusion-positive NSCLC that has progressed on platinum-based chemotherapy |
| Category: Cancer |
|
|
seladelpar |
02/20/2019 |
|
Patients with early stage primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA) |
Other |
|
Date of BT Designation Disclosure: 02/20/2019
Approval Date: |
| Indication: Patients with early stage primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA) |
| Category: Other |
|
|
CP101 |
02/9/2019 |
|
Patients with recurrent Clostridium difficile infection |
Infectious Disease |
|
Date of BT Designation Disclosure: 02/9/2019
Approval Date: |
| Indication: Patients with recurrent Clostridium difficile infection |
| Category: Infectious Disease |
|
|
MEDI8897 |
02/6/2019 |
|
Prevention of lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV) |
Other |
|
Date of BT Designation Disclosure: 02/6/2019
Approval Date: |
| Indication: Prevention of lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV) |
| Category: Other |
|
|
Kadcyla (ado-trastuzumab) |
02/5/2019 |
05/03/2019 |
Patients with HER2-positive early breast cancer who have residual disease after neoadjuvant treatment |
Cancer |
|
Date of BT Designation Disclosure: 02/5/2019
Approval Date: 05/03/2019 |
| Indication: Patients with HER2-positive early breast cancer who have residual disease after neoadjuvant treatment |
| Category: Cancer |
|
|
V114 |
01/28/2019 |
|
Prevention of invasive pneumococcal disease |
Infectious Disease |
|
Date of BT Designation Disclosure: 01/28/2019
Approval Date: |
| Indication: Prevention of invasive pneumococcal disease |
| Category: Infectious Disease |
|
|
umbralisib (TGR-1202) |
01/28/2019 |
|
Adult patients with marginal zone lymphoma (MZL) who did not respond to at least one anti-CD20 therapy |
Cancer |
|
Date of BT Designation Disclosure: 01/28/2019
Approval Date: |
| Indication: Adult patients with marginal zone lymphoma (MZL) who did not respond to at least one anti-CD20 therapy |
| Category: Cancer |
|
|
BGB-3111 (zanubrutinib) |
01/16/2019 |
|
Patients with mantle cell lymphoma who have received at least one prior therapy |
Cancer |
|
Date of BT Designation Disclosure: 01/16/2019
Approval Date: |
| Indication: Patients with mantle cell lymphoma who have received at least one prior therapy |
| Category: Cancer |
|
|
crizanlizumab (SEG101) |
01/8/2019 |
|
The prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/8/2019
Approval Date: |
| Indication: The prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease |
| Category: Rare Inherited Disorders |
|
|
Olipudase Alfa |
11/29/2018 |
|
Niemann-Pick disease Type B (NPB) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 11/29/2018
Approval Date: |
| Indication: Niemann-Pick disease Type B (NPB) |
| Category: Rare Inherited Disorders |
|
|
lonafarnib |
11/19/2018 |
|
Patients with Hutchinson-Gilford progeria syndrome (HGPS_ |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 11/19/2018
Approval Date: |
| Indication: Patients with Hutchinson-Gilford progeria syndrome (HGPS_ |
| Category: Rare Inherited Disorders |
|
|
Adcetris (brentuximab vedotin) |
11/16/2018 |
|
Previously untreated systemic anaplastic large cell lymphoma or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified, in combination with CHP |
Cancer |
|
Date of BT Designation Disclosure: 11/16/2018
Approval Date: |
| Indication: Previously untreated systemic anaplastic large cell lymphoma or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified, in combination with CHP |
| Category: Cancer |
|
|
Promacta (eltrombopag) |
11/16/2018 |
|
Low platelet counts in people exposed to radiation |
Other |
|
Date of BT Designation Disclosure: 11/16/2018
Approval Date: |
| Indication: Low platelet counts in people exposed to radiation |
| Category: Other |
|
|
Emgality (galcanezumab-gnlm) |
11/15/2018 |
06/04/2019 |
Episodic cluster headache in adults |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 11/15/2018
Approval Date: 06/04/2019 |
| Indication: Episodic cluster headache in adults |
| Category: Rare Inherited Disorders |
|
|
NGX-101 |
11/13/2018 |
|
Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy |
Other |
|
Date of BT Designation Disclosure: 11/13/2018
Approval Date: |
| Indication: Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy |
| Category: Other |
|
|
UGN-101 (mitomycin gel) |
10/31/2018 |
|
Patients with low-grade upper tract urothelial cancer |
Cancer |
|
Date of BT Designation Disclosure: 10/31/2018
Approval Date: |
| Indication: Patients with low-grade upper tract urothelial cancer |
| Category: Cancer |
|
|
Myrcludex |
10/23/2018 |
|
Chronic hepatitis delta (HDV) infection |
Infectious Disease |
|
Date of BT Designation Disclosure: 10/23/2018
Approval Date: |
| Indication: Chronic hepatitis delta (HDV) infection |
| Category: Infectious Disease |
|
|
Psilocybin therapy |
10/23/2018 |
|
Treatment-resistant Depression |
Other |
|
Date of BT Designation Disclosure: 10/23/2018
Approval Date: |
| Indication: Treatment-resistant Depression |
| Category: Other |
|
|
KD025 |
10/17/2018 |
|
Patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy |
Other |
|
Date of BT Designation Disclosure: 10/17/2018
Approval Date: |
| Indication: Patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy |
| Category: Other |
|
|
LOXO-292 |
10/16/2018 |
|
Patients with metastatic, RET-fusion-positive non-small-cell lung cancer (NSCLC) who require systemic therapy and progressed following anti PD-L1 therapy or platinum-based chemotherapy plus an anti-PD-1 therapy |
Cancer |
|
Date of BT Designation Disclosure: 10/16/2018
Approval Date: |
| Indication: Patients with metastatic, RET-fusion-positive non-small-cell lung cancer (NSCLC) who require systemic therapy and progressed following anti PD-L1 therapy or platinum-based chemotherapy plus an anti-PD-1 therapy |
| Category: Cancer |
|
|
LOXO-292 |
10/16/2018 |
|
Patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment, and have no acceptable alternative treatment options |
Cancer |
|
Date of BT Designation Disclosure: 10/16/2018
Approval Date: |
| Indication: Patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment, and have no acceptable alternative treatment options |
| Category: Cancer |
|
|
Rubraca (rucaparib) |
10/3/2018 |
|
BRCA1/BRCA2-mutated, metastatic, castration-resistant prostate cancer |
Cancer |
|
Date of BT Designation Disclosure: 10/3/2018
Approval Date: |
| Indication: BRCA1/BRCA2-mutated, metastatic, castration-resistant prostate cancer |
| Category: Cancer |
|
|
PF-06482077 |
09/20/2018 |
|
Prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae serotypes in the vaccine in adults aged 18 years and older |
Infectious Disease |
|
Date of BT Designation Disclosure: 09/20/2018
Approval Date: |
| Indication: Prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae serotypes in the vaccine in adults aged 18 years and older |
| Category: Infectious Disease |
|
|
CA-008 |
09/11/2018 |
|
Patients needing long-lasting, non-opioid pain relief |
Other |
|
Date of BT Designation Disclosure: 09/11/2018
Approval Date: |
| Indication: Patients needing long-lasting, non-opioid pain relief |
| Category: Other |
|
|
Tezepelumab |
09/7/2018 |
|
Patients with severe asthma, without an eosinophilic phenotype, who are receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers |
Other |
|
Date of BT Designation Disclosure: 09/7/2018
Approval Date: |
| Indication: Patients with severe asthma, without an eosinophilic phenotype, who are receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers |
| Category: Other |
|
|
PF-06651600 |
09/6/2018 |
|
Patients with alopecia areata |
Other |
|
Date of BT Designation Disclosure: 09/6/2018
Approval Date: |
| Indication: Patients with alopecia areata |
| Category: Other |
|
|
LOXO-292 |
09/5/2018 |
|
Patients with metastatic RET-fusion-positive non-small cell lung cancer; patients with RET-mutant medullary thyroid cancer |
Cancer |
|
Date of BT Designation Disclosure: 09/5/2018
Approval Date: |
| Indication: Patients with metastatic RET-fusion-positive non-small cell lung cancer; patients with RET-mutant medullary thyroid cancer |
| Category: Cancer |
|
|
naxitamab |
08/23/2018 |
|
In combination with GM-CSF for patients with high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow |
Cancer |
|
Date of BT Designation Disclosure: 08/23/2018
Approval Date: |
| Indication: In combination with GM-CSF for patients with high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow |
| Category: Cancer |
|
|
Xolair (omalizumab) |
08/13/2018 |
09/28/2018 |
Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies |
Other |
|
Date of BT Designation Disclosure: 08/13/2018
Approval Date: 09/28/2018 |
| Indication: Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies |
| Category: Other |
|
|
Braftovi (encorafenib) + Mektovi (binimetinib) + Erbitux (cetuximab) |
08/7/2018 |
|
Patients with BRAF V600E-mutation metastatic colorectal caner following 1 or 2 prior lines of treatment in the metastatic setting |
Cancer |
|
Date of BT Designation Disclosure: 08/7/2018
Approval Date: |
| Indication: Patients with BRAF V600E-mutation metastatic colorectal caner following 1 or 2 prior lines of treatment in the metastatic setting |
| Category: Cancer |
|
|
Quizartinib |
08/1/2018 |
|
Adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML) |
Cancer |
|
Date of BT Designation Disclosure: 08/1/2018
Approval Date: |
| Indication: Adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML) |
| Category: Cancer |
|
|
Lenvima (lenvatinib) + Keytruda (pembrolizumab) |
08/1/2018 |
09/17/2019 |
Patients with previously untreated advanced and/or metastatic non-MSI-H/pMMR endometrial carcinoma |
Cancer |
|
Date of BT Designation Disclosure: 08/1/2018
Approval Date: 09/17/2019 |
| Indication: Patients with previously untreated advanced and/or metastatic non-MSI-H/pMMR endometrial carcinoma |
| Category: Cancer |
|
|
Tecentriq (atezolizumab) + Avastin (bevacizumab) |
07/25/2018 |
|
First-line treatment of patients with advanced or metastatic hepatocellular carcinoma (HCC) |
|
|
Date of BT Designation Disclosure: 07/25/2018
Approval Date: |
| Indication: First-line treatment of patients with advanced or metastatic hepatocellular carcinoma (HCC) |
| Category: |
|
|
Tecentriq (atezolizumab) |
07/18/2018 |
|
In combination with Avastin (bevacizumab) as a first-line treatment for patients with advanced or metastatic hepatocellular carcinoma (HCC) |
|
|
Date of BT Designation Disclosure: 07/18/2018
Approval Date: |
| Indication: In combination with Avastin (bevacizumab) as a first-line treatment for patients with advanced or metastatic hepatocellular carcinoma (HCC) |
| Category: |
|
|
AMT-601 |
06/28/2018 |
|
Patients with severe and moderately severe hemophilia B |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 06/28/2018
Approval Date: |
| Indication: Patients with severe and moderately severe hemophilia B |
| Category: Rare Inherited Disorders |
|
|
HTX-011 |
06/21/2018 |
|
Postoperative Pain Management |
Other |
|
Date of BT Designation Disclosure: 06/21/2018
Approval Date: |
| Indication: Postoperative Pain Management |
| Category: Other |
|
|
Kisqali (ribociclib) |
06/20/2018 |
07/18/2018 |
In combination with an aromatase inhibitor for pre/perimenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy |
Cancer |
|
Date of BT Designation Disclosure: 06/20/2018
Approval Date: 07/18/2018 |
| Indication: In combination with an aromatase inhibitor for pre/perimenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy |
| Category: Cancer |
|
|
Kisqali (ribociclib) |
06/20/2018 |
07/18/2018 |
In combination with fulvestrant for postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy or following disease progression on endocrine therapy |
Cancer |
|
Date of BT Designation Disclosure: 06/20/2018
Approval Date: 07/18/2018 |
| Indication: In combination with fulvestrant for postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy or following disease progression on endocrine therapy |
| Category: Cancer |
|
|
Elzonris (tagraxofusp) |
06/15/2018 |
12/21/2018 |
Adults and children aged 2 years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN) |
Cancer |
|
Date of BT Designation Disclosure: 06/15/2018
Approval Date: 12/21/2018 |
| Indication: Adults and children aged 2 years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN) |
| Category: Cancer |
|
|
Xalkori (crizotinib) |
05/29/2018 |
|
Patients with metastatic non-small cell lung cancer with MET exon 14 alterations with disease progression on or after platinum-based chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 05/29/2018
Approval Date: |
| Indication: Patients with metastatic non-small cell lung cancer with MET exon 14 alterations with disease progression on or after platinum-based chemotherapy |
| Category: Cancer |
|
|
Xalkori (crizotinib) |
05/29/2018 |
|
Patients with relapsed or refractory systemic anaplastic large cell lymphoma that is anaplastic lymphoma kinase-positive |
Cancer |
|
Date of BT Designation Disclosure: 05/29/2018
Approval Date: |
| Indication: Patients with relapsed or refractory systemic anaplastic large cell lymphoma that is anaplastic lymphoma kinase-positive |
| Category: Cancer |
|
|
Vyndaqel (tafamidis meglumine) |
05/24/2018 |
05/06/2019 |
Patients with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) |
Other |
|
Date of BT Designation Disclosure: 05/24/2018
Approval Date: 05/06/2019 |
| Indication: Patients with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) |
| Category: Other |
|
|
Lenti-D |
05/23/2018 |
|
Cerebral adrenoleukodystrophy |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 05/23/2018
Approval Date: |
| Indication: Cerebral adrenoleukodystrophy |
| Category: Rare Inherited Disorders |
|
|
Tafamidis |
05/23/2018 |
|
Transthyretin Cardiomyopathy (ATTR-ACT) |
Cardiovascular |
|
Date of BT Designation Disclosure: 05/23/2018
Approval Date: |
| Indication: Transthyretin Cardiomyopathy (ATTR-ACT) |
| Category: Cardiovascular |
|
|
Pitolisant |
05/21/2018 |
|
Excessive daytime sleepiness and cataplexy in patients with narcolepsy |
Other |
|
Date of BT Designation Disclosure: 05/21/2018
Approval Date: |
| Indication: Excessive daytime sleepiness and cataplexy in patients with narcolepsy |
| Category: Other |
|
|
OMS721 |
04/26/2018 |
|
Patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patents who have persistent TMA despite modification of immunosupressive therapy |
Other |
|
Date of BT Designation Disclosure: 04/26/2018
Approval Date: |
| Indication: Patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patents who have persistent TMA despite modification of immunosupressive therapy |
| Category: Other |
|
|
Trumenba (Meningococcal Group B Vaccine) |
04/23/2018 |
|
Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 1 through 9 years of age |
|
|
Date of BT Designation Disclosure: 04/23/2018
Approval Date: |
| Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 1 through 9 years of age |
| Category: |
|
|
Hemlibra (emicizumab-kxwh) |
04/17/2018 |
10/04/2018 |
Patients with hemophilia A without factor VIII inhibitors |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/17/2018
Approval Date: 10/04/2018 |
| Indication: Patients with hemophilia A without factor VIII inhibitors |
| Category: Rare Inherited Disorders |
|
|
Ketamine |
04/16/2018 |
|
Patients with depression |
Other |
|
Date of BT Designation Disclosure: 04/16/2018
Approval Date: |
| Indication: Patients with depression |
| Category: Other |
|
|
ALIS (amikacin liposome inhalation suspension) |
03/29/2018 |
|
Adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex |
Other |
|
Date of BT Designation Disclosure: 03/29/2018
Approval Date: |
| Indication: Adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex |
| Category: Other |
|
|
Pedmark (sodium thiosulfate) |
03/27/2018 |
|
Prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB) |
Other |
|
Date of BT Designation Disclosure: 03/27/2018
Approval Date: |
| Indication: Prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB) |
| Category: Other |
|
|
enfortumab vedotin |
03/26/2018 |
|
Patients with locally advanced or metastatic urothelial carcinoma |
Cancer |
|
Date of BT Designation Disclosure: 03/26/2018
Approval Date: |
| Indication: Patients with locally advanced or metastatic urothelial carcinoma |
| Category: Cancer |
|
|
Enfortumab Vedotin |
03/26/2018 |
|
Patients with metastatic urothelial cancer who were previously treated with checkpoint inhibitors |
Cancer |
|
Date of BT Designation Disclosure: 03/26/2018
Approval Date: |
| Indication: Patients with metastatic urothelial cancer who were previously treated with checkpoint inhibitors |
| Category: Cancer |
|
|
PTI-428 |
03/16/2018 |
|
Cystic Fibrosis for patients with two copies of the F508del mutation — one from their mother and one from their father — who are receiving Vertex’s Orkambi (lumacaftor/ivacaftor) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 03/16/2018
Approval Date: |
| Indication: Cystic Fibrosis for patients with two copies of the F508del mutation — one from their mother and one from their father — who are receiving Vertex’s Orkambi (lumacaftor/ivacaftor) |
| Category: Rare Inherited Disorders |
|
|
Balversa (erdafitinib) |
03/15/2018 |
04/12/2019 |
Adult patients with locally advanced or metastatic bladder cancer that has a type of susceptible genetic alteration known as FGFR3 or FGFR2, and that has progressed during or following prior platinum-containing chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 03/15/2018
Approval Date: 04/12/2019 |
| Indication: Adult patients with locally advanced or metastatic bladder cancer that has a type of susceptible genetic alteration known as FGFR3 or FGFR2, and that has progressed during or following prior platinum-containing chemotherapy |
| Category: Cancer |
|
|
Orkambi (lumacaftor/ivacaftor) |
03/12/2018 |
08/07/2018 |
Treatment of cystic fibrosis (CF) in patients 2 years and older, homozygous for the F508del-CFTR mutation in the CFTR gene |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 03/12/2018
Approval Date: 08/07/2018 |
| Indication: Treatment of cystic fibrosis (CF) in patients 2 years and older, homozygous for the F508del-CFTR mutation in the CFTR gene |
| Category: Rare Inherited Disorders |
|
|
Lumasiran |
03/12/2018 |
|
Primary Hyperoxaluria Type 1 (PH1) |
Infectious Disease |
|
Date of BT Designation Disclosure: 03/12/2018
Approval Date: |
| Indication: Primary Hyperoxaluria Type 1 (PH1) |
| Category: Infectious Disease |
|
|
BIVV009 |
03/6/2018 |
|
Patients with cold agglutinin disease (CAgD) |
Other |
|
Date of BT Designation Disclosure: 03/6/2018
Approval Date: |
| Indication: Patients with cold agglutinin disease (CAgD) |
| Category: Other |
|
|
GC4419 |
02/28/2018 |
|
Reduction of the duration, severity, and incidence of severe oral mucositis induced by radiation therapy |
Other |
|
Date of BT Designation Disclosure: 02/28/2018
Approval Date: |
| Indication: Reduction of the duration, severity, and incidence of severe oral mucositis induced by radiation therapy |
| Category: Other |
|
|
Opdivo (nivolumab) + Yervoy (ipilimumab) combination |
02/16/2018 |
04/16/2018 |
Patients with previously untreated renal cell carcinoma that is of intermediate or poor risk |
Cancer |
|
Date of BT Designation Disclosure: 02/16/2018
Approval Date: 04/16/2018 |
| Indication: Patients with previously untreated renal cell carcinoma that is of intermediate or poor risk |
| Category: Cancer |
|
|
SPK-8011 |
02/15/2018 |
|
Patients with hemophilia A. |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 02/15/2018
Approval Date: |
| Indication: Patients with hemophilia A. |
| Category: Rare Inherited Disorders |
|
|
PF-04965842 |
02/14/2018 |
|
Patients with moderate-to-severe atopic dermatitis (AD) |
Other |
|
Date of BT Designation Disclosure: 02/14/2018
Approval Date: |
| Indication: Patients with moderate-to-severe atopic dermatitis (AD) |
| Category: Other |
|
|
Opdivo (nivolumab) + Yervoy (ipilimumab) combination |
02/10/2018 |
07/10/2018 |
Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan |
Cancer |
|
Date of BT Designation Disclosure: 02/10/2018
Approval Date: 07/10/2018 |
| Indication: Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan |
| Category: Cancer |
|
|
SAGE-217 |
02/7/2018 |
|
Patients with major depressive disorder (MDD) |
Other |
|
Date of BT Designation Disclosure: 02/7/2018
Approval Date: |
| Indication: Patients with major depressive disorder (MDD) |
| Category: Other |
|
|
ZX008 (fenfluramine) |
02/6/2018 |
|
Patients who have seizures associated with Dravet syndrome |
Other |
|
Date of BT Designation Disclosure: 02/6/2018
Approval Date: |
| Indication: Patients who have seizures associated with Dravet syndrome |
| Category: Other |
|
|
Onpattro (patisiran) |
02/2/2018 |
08/10/2018 |
Peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 02/2/2018
Approval Date: 08/10/2018 |
| Indication: Peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients |
| Category: Rare Inherited Disorders |
|
|
Balovaptan |
01/29/2018 |
|
Autism spectrum disorder (ASD) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/29/2018
Approval Date: |
| Indication: Autism spectrum disorder (ASD) |
| Category: Rare Inherited Disorders |
|
|
Balovaptan (RG7314) |
01/28/2018 |
|
A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD) |
Other |
|
Date of BT Designation Disclosure: 01/28/2018
Approval Date: |
| Indication: A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD) |
| Category: Other |
|
|
Kisquali Femara Co-Pack (Letrozole and Ribociclib |
01/10/2018 |
02/13/2019 |
Treatment of pre/perimenopausal women with HR-positive, HER2- negative advanced or metastatic breast cancer |
Cancer |
|
Date of BT Designation Disclosure: 01/10/2018
Approval Date: 02/13/2019 |
| Indication: Treatment of pre/perimenopausal women with HR-positive, HER2- negative advanced or metastatic breast cancer |
| Category: Cancer |
|
|
Voxelotor |
01/9/2018 |
|
Sickle cell disease (SCD) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/9/2018
Approval Date: |
| Indication: Sickle cell disease (SCD) |
| Category: Rare Inherited Disorders |
|
|
Lenvima (lenvatinib) in combination with Keytruda (pembrolizumab) |
01/9/2018 |
|
Advanced and/or metastatic renal cell carcinoma (RCC) |
Cancer |
|
Date of BT Designation Disclosure: 01/9/2018
Approval Date: |
| Indication: Advanced and/or metastatic renal cell carcinoma (RCC) |
| Category: Cancer |
|
|
Upadacitinib (ABT-494) |
01/9/2018 |
|
Adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy |
Other |
|
Date of BT Designation Disclosure: 01/9/2018
Approval Date: |
| Indication: Adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy |
| Category: Other |
|
|
Maribavir (SHP620) |
01/8/2018 |
|
Cytomegalovirus (CMV) infection in transplant patients. |
Infectious Disease |
|
Date of BT Designation Disclosure: 01/8/2018
Approval Date: |
| Indication: Cytomegalovirus (CMV) infection in transplant patients. |
| Category: Infectious Disease |
|
|
Promacta (eltrombopag) |
01/4/2018 |
11/17/2018 |
First-line treatment for adults and pediatric patients two years and older with SAA in combination with standard immunosuppressive therapy (IST) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/4/2018
Approval Date: 11/17/2018 |
| Indication: First-line treatment for adults and pediatric patients two years and older with SAA in combination with standard immunosuppressive therapy (IST) |
| Category: Rare Inherited Disorders |
|
|
Gamifant (emapalumab-Izsg) |
/2018 |
11/21/2018 |
Adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent, or progressive disease or intolerance with conventional therapy (HLH) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: /2018
Approval Date: 11/21/2018 |
| Indication: Adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent, or progressive disease or intolerance with conventional therapy (HLH) |
| Category: Rare Inherited Disorders |
|
|
Bavencio (avelumab) plus Inlyta (axitinib) |
12/22/2017 |
05/14/2019 |
First-line treatment of patients with advanced renal cell carcinoma (RCC) |
Cancer |
|
Date of BT Designation Disclosure: 12/22/2017
Approval Date: 05/14/2019 |
| Indication: First-line treatment of patients with advanced renal cell carcinoma (RCC) |
| Category: Cancer |
|
|
Avelumab in combination with Inlyta (axitinib) |
12/21/2017 |
|
Treatment-naïve patients with advanced renal cell carcinoma (RCC) |
Cancer |
|
Date of BT Designation Disclosure: 12/21/2017
Approval Date: |
| Indication: Treatment-naïve patients with advanced renal cell carcinoma (RCC) |
| Category: Cancer |
|
|
Gilenya (fingolimod) |
12/18/2017 |
05/11/2018 |
Relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older. |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 12/18/2017
Approval Date: 05/11/2018 |
| Indication: Relapsing multiple sclerosis (MS) in children and adolescents age 10 years and older. |
| Category: Rare Inherited Disorders |
|
|
Polivy (polatuzumab vedotin-piiq) |
12/10/2017 |
06/10/2019 |
Adult patients with diffuse large B-cell lymphoma (DLBCL) that has progressed or returned after at least two prior therapies |
Cancer |
|
Date of BT Designation Disclosure: 12/10/2017
Approval Date: 06/10/2019 |
| Indication: Adult patients with diffuse large B-cell lymphoma (DLBCL) that has progressed or returned after at least two prior therapies |
| Category: Cancer |
|
|
Keytruda (pembrolizumab) plus Inlyta (axitinib) |
12/10/2017 |
04/19/2019 |
First-line treatment of patients with advanced renal cell carcinoma (RCC) |
Cancer |
|
Date of BT Designation Disclosure: 12/10/2017
Approval Date: 04/19/2019 |
| Indication: First-line treatment of patients with advanced renal cell carcinoma (RCC) |
| Category: Cancer |
|
|
Oxervate (cenegermin-bkbj ophthalmic solution) |
11/30/2017 |
08/22/2018 |
Patients with neurotrophic keratitis (NK) |
Other |
|
Date of BT Designation Disclosure: 11/30/2017
Approval Date: 08/22/2018 |
| Indication: Patients with neurotrophic keratitis (NK) |
| Category: Other |
|
|
bb2121 |
11/4/2017 |
|
Patients with relapsed or refractory multiple myeloma |
Cancer |
|
Date of BT Designation Disclosure: 11/4/2017
Approval Date: |
| Indication: Patients with relapsed or refractory multiple myeloma |
| Category: Cancer |
|
|
Valoctocogene Roxaparvovec |
10/26/2017 |
|
Hemophilia A |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 10/26/2017
Approval Date: |
| Indication: Hemophilia A |
| Category: Rare Inherited Disorders |
|
|
Poteligeo (mogamulizumab-kpkc) |
10/25/2017 |
08/08/2018 |
Adult patients Sézary syndrome (SS) after at least one prior systemic therapy |
Cancer |
|
Date of BT Designation Disclosure: 10/25/2017
Approval Date: 08/08/2018 |
| Indication: Adult patients Sézary syndrome (SS) after at least one prior systemic therapy |
| Category: Cancer |
|
|
Venclexta (venetoclax) |
10/25/2017 |
11/26/2018 |
In combination with azacitidine, decitabine, or low-dose cytarabine for adults aged 75 and older with treatment-naive acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 10/25/2017
Approval Date: 11/26/2018 |
| Indication: In combination with azacitidine, decitabine, or low-dose cytarabine for adults aged 75 and older with treatment-naive acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy |
| Category: Cancer |
|
|
Tafinler (dabrafenib) & Mekinist (trametinib) |
10/23/2017 |
05/04/2018 |
In combination for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (ATC) with BRAF V600E mutation and with no satisfactory locoregional treatment options |
Cancer |
|
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 05/04/2018 |
| Indication: In combination for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (ATC) with BRAF V600E mutation and with no satisfactory locoregional treatment options |
| Category: Cancer |
|
|
Tafinlar (dabrafenib) + Mekinist (trametinib) |
10/23/2017 |
04/30/2018 |
Adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection |
Cancer |
|
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 04/30/2018 |
| Indication: Adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection |
| Category: Cancer |
|
|
MOR208 + Lenalidomide |
10/23/2017 |
|
Relapsed/Refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation |
Cancer |
|
Date of BT Designation Disclosure: 10/23/2017
Approval Date: |
| Indication: Relapsed/Refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation |
| Category: Cancer |
|
|
Opdivo (nivolumab) |
10/17/2017 |
12/20/2017 |
Adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection |
Cancer |
|
Date of BT Designation Disclosure: 10/17/2017
Approval Date: 12/20/2017 |
| Indication: Adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection |
| Category: Cancer |
|
|
Adcetris (brentuximab vedotin) |
10/15/2017 |
03/20/2018 |
Treatment for adult patients with previously untreated Stage III or IV classical Hodgkin Lymphoma, in combination with chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 10/15/2017
Approval Date: 03/20/2018 |
| Indication: Treatment for adult patients with previously untreated Stage III or IV classical Hodgkin Lymphoma, in combination with chemotherapy |
| Category: Cancer |
|
|
DAS181 |
10/10/2017 |
|
Treatment of lower respiratory tract parainfluenza virus (PIV) infection in immunocompromised patients |
Infectious Disease |
|
Date of BT Designation Disclosure: 10/10/2017
Approval Date: |
| Indication: Treatment of lower respiratory tract parainfluenza virus (PIV) infection in immunocompromised patients |
| Category: Infectious Disease |
|
|
Tagrisso (osimertinib) |
10/9/2017 |
04/18/2018 |
First line treatment of patients with EGFR mutation-positive non-small cell lung cancer |
Cancer |
|
Date of BT Designation Disclosure: 10/9/2017
Approval Date: 04/18/2018 |
| Indication: First line treatment of patients with EGFR mutation-positive non-small cell lung cancer |
| Category: Cancer |
|
|
Adcetris (brentuximab vedotin) |
10/2/2017 |
11/09/2017 |
Patients with primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides who have received prior systemic therapy |
Cancer |
|
Date of BT Designation Disclosure: 10/2/2017
Approval Date: 11/09/2017 |
| Indication: Patients with primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides who have received prior systemic therapy |
| Category: Cancer |
|
|
Libtayo (cemiplimab-rwlc) |
09/8/2017 |
09/28/2018 |
Adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC |
Cancer |
|
Date of BT Designation Disclosure: 09/8/2017
Approval Date: 09/28/2018 |
| Indication: Adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC |
| Category: Cancer |
|
|
EB-101 |
08/30/2017 |
|
Recessive Dystrophic Epidermolysis Bullosa (RDEB) |
Other |
|
Date of BT Designation Disclosure: 08/30/2017
Approval Date: |
| Indication: Recessive Dystrophic Epidermolysis Bullosa (RDEB) |
| Category: Other |
|
|
Trastuzumab Deruxtecan (DS-8201) |
08/29/2017 |
|
HER2-positive, locally advanced, or metastatic breast cancer who have been treated with trastuzumab (Herceptin) and pertuzumab (Perjeta) and have disease progression after ado-trastuzumab emtansine (T-DM1; Kadcyla) |
Cancer |
|
Date of BT Designation Disclosure: 08/29/2017
Approval Date: |
| Indication: HER2-positive, locally advanced, or metastatic breast cancer who have been treated with trastuzumab (Herceptin) and pertuzumab (Perjeta) and have disease progression after ado-trastuzumab emtansine (T-DM1; Kadcyla) |
| Category: Cancer |
|
|
Methylenedioxymethamphetamine (MDMA) |
08/28/2017 |
|
PTSD |
Other |
|
Date of BT Designation Disclosure: 08/28/2017
Approval Date: |
| Indication: PTSD |
| Category: Other |
|
|
Poteligeo (mogamulizumab-kpkc) |
08/25/2017 |
08/08/2018 |
Adult patients with relapsed or refractory mycosis fungoides (MF) after at least one prior systemic therapy |
Cancer |
|
Date of BT Designation Disclosure: 08/25/2017
Approval Date: 08/08/2018 |
| Indication: Adult patients with relapsed or refractory mycosis fungoides (MF) after at least one prior systemic therapy |
| Category: Cancer |
|
|
Besponsa (inotuzumab ozogamicin) |
08/17/2017 |
|
Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) |
Cancer |
|
Date of BT Designation Disclosure: 08/17/2017
Approval Date: |
| Indication: Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) |
| Category: Cancer |
|
|
Zelboraf (vemurafenib) |
08/7/2017 |
11/06/2017 |
Patients with Erdheim-Chester disease (ECD) with a BRAF V600 mutation |
Other |
|
Date of BT Designation Disclosure: 08/7/2017
Approval Date: 11/06/2017 |
| Indication: Patients with Erdheim-Chester disease (ECD) with a BRAF V600 mutation |
| Category: Other |
|
|
Calquence (acalabrutinib) |
08/1/2017 |
10/31/2017 |
Adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy |
Cancer |
|
Date of BT Designation Disclosure: 08/1/2017
Approval Date: 10/31/2017 |
| Indication: Adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy |
| Category: Cancer |
|
|
Venetoclax |
08/1/2017 |
|
Elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 08/1/2017
Approval Date: |
| Indication: Elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy |
| Category: Cancer |
|
|
Acalabrutinib |
08/1/2017 |
|
Mantle cell lymphoma for patients who have received at least one prior line of therapy |
Cancer |
|
Date of BT Designation Disclosure: 08/1/2017
Approval Date: |
| Indication: Mantle cell lymphoma for patients who have received at least one prior line of therapy |
| Category: Cancer |
|
|
Imfinzi (durvalumab) |
07/31/2017 |
02/16/2018 |
Patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy |
Cancer |
|
Date of BT Designation Disclosure: 07/31/2017
Approval Date: 02/16/2018 |
| Indication: Patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy |
| Category: Cancer |
|
|
Keytruda (pembrolizumab) |
07/1/2017 |
12/20/2018 |
Adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC) |
Cancer |
|
Date of BT Designation Disclosure: 07/1/2017
Approval Date: 12/20/2018 |
| Indication: Adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC) |
| Category: Cancer |
|
|
Korsuva (difelikefalin) |
06/22/2017 |
|
Chronic kidney disease, associated pruritius in hemodialysis patients |
Other |
|
Date of BT Designation Disclosure: 06/22/2017
Approval Date: |
| Indication: Chronic kidney disease, associated pruritius in hemodialysis patients |
| Category: Other |
|
|
OMS721 |
06/13/2017 |
|
Immunoglobulin A (IgA) nephropathy |
Other |
|
Date of BT Designation Disclosure: 06/13/2017
Approval Date: |
| Indication: Immunoglobulin A (IgA) nephropathy |
| Category: Other |
|
|
Burtomab |
06/7/2017 |
|
Pediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis |
Cancer |
|
Date of BT Designation Disclosure: 06/7/2017
Approval Date: |
| Indication: Pediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis |
| Category: Cancer |
|
|
Avapritinib |
06/1/2017 |
|
Patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation |
Cancer |
|
Date of BT Designation Disclosure: 06/1/2017
Approval Date: |
| Indication: Patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation |
| Category: Cancer |
|
|
Givosiran (ALN-AS1) |
05/30/2017 |
|
Acute hepatic porphyria (AHP) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 05/30/2017
Approval Date: |
| Indication: Acute hepatic porphyria (AHP) |
| Category: Rare Inherited Disorders |
|
|
Zemdri (plazomicin) |
05/23/2017 |
|
Antibacterials addressing multi-drug resistant (MDR) gram-negative infections |
Other |
|
Date of BT Designation Disclosure: 05/23/2017
Approval Date: |
| Indication: Antibacterials addressing multi-drug resistant (MDR) gram-negative infections |
| Category: Other |
|
|
GMI-1271 |
05/17/2017 |
|
Adult Relapsed/Refractory Acute Myeloid Leukemia |
Cancer |
|
Date of BT Designation Disclosure: 05/17/2017
Approval Date: |
| Indication: Adult Relapsed/Refractory Acute Myeloid Leukemia |
| Category: Cancer |
|
|
Rozlytrek (entrectinib) |
05/15/2017 |
08/15/2019 |
NTRK fusion–positive locally advanced or metastatic solid tumors |
Cancer |
|
Date of BT Designation Disclosure: 05/15/2017
Approval Date: 08/15/2019 |
| Indication: NTRK fusion–positive locally advanced or metastatic solid tumors |
| Category: Cancer |
|
|
Ribaxamase (SYN-004) |
05/11/2017 |
|
prevention of Clostridium difficile infection |
Infectious Disease |
|
Date of BT Designation Disclosure: 05/11/2017
Approval Date: |
| Indication: prevention of Clostridium difficile infection |
| Category: Infectious Disease |
|
|
Vonapanitase |
05/10/2017 |
|
Increasing arteriovenous fistula secondary patency (i.e., survival of the fistula without abandonment) and use for hemodialysis in patients on or expected to initiate hemodialysis |
Other |
|
Date of BT Designation Disclosure: 05/10/2017
Approval Date: |
| Indication: Increasing arteriovenous fistula secondary patency (i.e., survival of the fistula without abandonment) and use for hemodialysis in patients on or expected to initiate hemodialysis |
| Category: Other |
|
|
Lorlatinib |
04/27/2017 |
|
ALK-positive metastatic non-small cell lung cancer (NSCLC) who have previously received 1 or more ALK inhibitors |
Cancer |
|
Date of BT Designation Disclosure: 04/27/2017
Approval Date: |
| Indication: ALK-positive metastatic non-small cell lung cancer (NSCLC) who have previously received 1 or more ALK inhibitors |
| Category: Cancer |
|
|
Kymriah (tisagenlecleucel) |
04/18/2017 |
|
Patients with large B-cell lymphoma |
Cancer |
|
Date of BT Designation Disclosure: 04/18/2017
Approval Date: |
| Indication: Patients with large B-cell lymphoma |
| Category: Cancer |
|
|
Kymriah (CTL019) |
04/18/2017 |
|
Adult patients with relapsed and refractory diffuse large B-cell lymphoma, who have failed two or more prior therapies |
Cancer |
|
Date of BT Designation Disclosure: 04/18/2017
Approval Date: |
| Indication: Adult patients with relapsed and refractory diffuse large B-cell lymphoma, who have failed two or more prior therapies |
| Category: Cancer |
|
|
RVT-802 |
04/17/2017 |
|
Complete DiGeorge Syndrome (cDGS) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/17/2017
Approval Date: |
| Indication: Complete DiGeorge Syndrome (cDGS) |
| Category: Rare Inherited Disorders |
|
|
Evinacumab |
04/7/2017 |
|
Homozygous familial hypercholesterolemia (HOFH) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/7/2017
Approval Date: |
| Indication: Homozygous familial hypercholesterolemia (HOFH) |
| Category: Rare Inherited Disorders |
|
|
Rituximab (Rituxin) |
03/24/2017 |
06/07/2018 |
Pemphigus vulgaris |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 03/24/2017
Approval Date: 06/07/2018 |
| Indication: Pemphigus vulgaris |
| Category: Rare Inherited Disorders |
|
|
Prevymis (letermovir) |
02/27/2017 |
11/08/2017 |
Prophylaxis of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT) |
Infectious Disease |
|
Date of BT Designation Disclosure: 02/27/2017
Approval Date: 11/08/2017 |
| Indication: Prophylaxis of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT) |
| Category: Infectious Disease |
|
|
Toca 511 & Toca FC |
02/23/2017 |
|
Recurrent high grade glioma |
Cancer |
|
Date of BT Designation Disclosure: 02/23/2017
Approval Date: |
| Indication: Recurrent high grade glioma |
| Category: Cancer |
|
|
AMT-060 |
01/20/2017 |
|
Hemophilia B |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/20/2017
Approval Date: |
| Indication: Hemophilia B |
| Category: Rare Inherited Disorders |
|
|
Nuplazid (pimavanserin) |
/2017 |
|
Patients with dementia-related psychosis (DRP) |
Other |
|
Date of BT Designation Disclosure: /2017
Approval Date: |
| Indication: Patients with dementia-related psychosis (DRP) |
| Category: Other |
|
|
Keytruda (pembrolizumab) |
/2017 |
05/23/2017 |
Adult and pediatric patients with unresectable or metastatic MSI-H or mismatch repair deficient solid tumors that have progressed following prior treatment or metastatic, MSI-H or mistmatch repair colorectal cancer that has progressed following treatment |
Cancer |
|
Date of BT Designation Disclosure: /2017
Approval Date: 05/23/2017 |
| Indication: Adult and pediatric patients with unresectable or metastatic MSI-H or mismatch repair deficient solid tumors that have progressed following prior treatment or metastatic, MSI-H or mistmatch repair colorectal cancer that has progressed following treatment |
| Category: Cancer |
|
|
Keytruda (pembrolizumab) |
/2017 |
05/18/2017 |
Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neo- or neo-adjuvant treatment with platinum-containing chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: /2017
Approval Date: 05/18/2017 |
| Indication: Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neo- or neo-adjuvant treatment with platinum-containing chemotherapy |
| Category: Cancer |
|
|
Keytruda (pembrolizumab) |
/2017 |
06/13/2018 |
Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after two or more prior lines of therapy |
Cancer |
|
Date of BT Designation Disclosure: /2017
Approval Date: 06/13/2018 |
| Indication: Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after two or more prior lines of therapy |
| Category: Cancer |
|
|
Zykadia (ceritinib) |
/2017 |
05/26/2017 |
Patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA approved test |
Cancer |
|
Date of BT Designation Disclosure: /2017
Approval Date: 05/26/2017 |
| Indication: Patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA approved test |
| Category: Cancer |
|
|
Mekinist (trametinib) |
/2017 |
06/22/2017 |
Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test |
Cancer |
|
Date of BT Designation Disclosure: /2017
Approval Date: 06/22/2017 |
| Indication: Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test |
| Category: Cancer |
|
|
Ibrance (palbociclib) |
/2017 |
04/04/2019 |
In combination with an aromatase inhibitor, and in combination with fulvestrant, for treatment of male patients with advanced or metastatic breast cancer |
Cancer |
|
Date of BT Designation Disclosure: /2017
Approval Date: 04/04/2019 |
| Indication: In combination with an aromatase inhibitor, and in combination with fulvestrant, for treatment of male patients with advanced or metastatic breast cancer |
| Category: Cancer |
|
|
Ibrance (palbociclib) |
/2017 |
03/31/2017 |
Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, in combination with an aromatase inhibitor as initial endocrine-based therapy |
Cancer |
|
Date of BT Designation Disclosure: /2017
Approval Date: 03/31/2017 |
| Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, in combination with an aromatase inhibitor as initial endocrine-based therapy |
| Category: Cancer |
|
|
Liso-cel (Lisocabtagene maraleucel) |
12/20/2016 |
|
Relapsed/refractory (r/r) aggressive large B-cell NHL, including diffuse large B-cell lymphoma (DLBCL), not otherwise specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma |
Cancer |
|
Date of BT Designation Disclosure: 12/20/2016
Approval Date: |
| Indication: Relapsed/refractory (r/r) aggressive large B-cell NHL, including diffuse large B-cell lymphoma (DLBCL), not otherwise specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma |
| Category: Cancer |
|
|
Tonmya (TNX-102 SL) |
12/19/2016 |
|
PTSD |
Other |
|
Date of BT Designation Disclosure: 12/19/2016
Approval Date: |
| Indication: PTSD |
| Category: Other |
|
|
Neridronic acid |
12/16/2016 |
|
Complex regional pain syndrome (CRPS) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 12/16/2016
Approval Date: |
| Indication: Complex regional pain syndrome (CRPS) |
| Category: Rare Inherited Disorders |
|
|
Advantage Arrest™ Silver Diamine Fluoride 38% |
11/3/2016 |
|
Arrest of tooth decay in children and adults |
Other |
|
Date of BT Designation Disclosure: 11/3/2016
Approval Date: |
| Indication: Arrest of tooth decay in children and adults |
| Category: Other |
|
|
Zejula (niraparib) |
10/14/2016 |
03/27/2017 |
Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 10/14/2016
Approval Date: 03/27/2017 |
| Indication: Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy |
| Category: Cancer |
|
|
Elbit Imaging |
10/10/2016 |
|
Nicord |
Cancer |
|
Date of BT Designation Disclosure: 10/10/2016
Approval Date: |
| Indication: Nicord |
| Category: Cancer |
|
|
Actemra (tocilizumab) |
10/5/2016 |
05/22/2017 |
Adult patients with giant cell arteritis (GCA) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 10/5/2016
Approval Date: 05/22/2017 |
| Indication: Adult patients with giant cell arteritis (GCA) |
| Category: Rare Inherited Disorders |
|
|
Alecensa (alectinib) |
10/4/2016 |
|
Adult patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor |
Cancer |
|
Date of BT Designation Disclosure: 10/4/2016
Approval Date: |
| Indication: Adult patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor |
| Category: Cancer |
|
|
Dupixent (Dupilumab) |
10/1/2016 |
03/11/2019 |
Moderate-to-severe (12 to less than 18 years of age) and severe (six months to less than 12 years of age) atopic dermatitis in pediatric patients who are not adequately controlled with, or who are intolerant to, topical medication |
Other |
|
Date of BT Designation Disclosure: 10/1/2016
Approval Date: 03/11/2019 |
| Indication: Moderate-to-severe (12 to less than 18 years of age) and severe (six months to less than 12 years of age) atopic dermatitis in pediatric patients who are not adequately controlled with, or who are intolerant to, topical medication |
| Category: Other |
|
|
Mavyret (glecaprevir & pibrentasvir) |
09/30/2016 |
08/03/2017 |
Patients with chronic hepatitis C virus (HCV) genotype (GT) 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis; and also for patients with HCV GT1 infection previously treated w/ HCV NS5A or NS3/4A inhibitor but not both |
Infectious Disease |
|
Date of BT Designation Disclosure: 09/30/2016
Approval Date: 08/03/2017 |
| Indication: Patients with chronic hepatitis C virus (HCV) genotype (GT) 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis; and also for patients with HCV GT1 infection previously treated w/ HCV NS5A or NS3/4A inhibitor but not both |
| Category: Infectious Disease |
|
|
Zulresso (Brexanolone) |
09/6/2016 |
03/19/2019 |
Postpartum depression (PPD) |
Other |
|
Date of BT Designation Disclosure: 09/6/2016
Approval Date: 03/19/2019 |
| Indication: Postpartum depression (PPD) |
| Category: Other |
|
|
SL-401 |
08/23/2016 |
|
Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 08/23/2016
Approval Date: |
| Indication: Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123) |
| Category: Rare Inherited Disorders |
|
|
Esketamine |
08/16/2016 |
|
Major depressive disorder with imminent risk for suicide |
Other |
|
Date of BT Designation Disclosure: 08/16/2016
Approval Date: |
| Indication: Major depressive disorder with imminent risk for suicide |
| Category: Other |
|
|
Kisqali (ribociclib) |
08/3/2016 |
03/13/2017 |
Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer as initial endocrine-based therapy |
Cancer |
|
Date of BT Designation Disclosure: 08/3/2016
Approval Date: 03/13/2017 |
| Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer as initial endocrine-based therapy |
| Category: Cancer |
|
|
Kisqali Femara Co-Pack (letrozole and ribociclib) |
08/2/2016 |
05/04/2017 |
Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer |
Cancer |
|
Date of BT Designation Disclosure: 08/2/2016
Approval Date: 05/04/2017 |
| Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer |
| Category: Cancer |
|
|
Pracinostat |
08/1/2016 |
|
In combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 08/1/2016
Approval Date: |
| Indication: In combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy |
| Category: Cancer |
|
|
Darzalex (daratumumab) |
07/26/2016 |
11/21/2016 |
Patients with multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone |
Cancer |
|
Date of BT Designation Disclosure: 07/26/2016
Approval Date: 11/21/2016 |
| Indication: Patients with multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone |
| Category: Cancer |
|
|
V920 |
07/25/2016 |
|
Ebola Zaire |
Infectious Disease |
|
Date of BT Designation Disclosure: 07/25/2016
Approval Date: |
| Indication: Ebola Zaire |
| Category: Infectious Disease |
|
|
SPK-9001 |
07/21/2016 |
|
Hemophilia B. |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 07/21/2016
Approval Date: |
| Indication: Hemophilia B. |
| Category: Rare Inherited Disorders |
|
|
Zolgensma (onasemnogene abeparvovec-xioi) |
07/20/2016 |
05/24/2019 |
Spinal muscular atrophy (SMA) Type 1 in children less than two years old |
Other |
|
Date of BT Designation Disclosure: 07/20/2016
Approval Date: 05/24/2019 |
| Indication: Spinal muscular atrophy (SMA) Type 1 in children less than two years old |
| Category: Other |
|
|
Larotrectinib |
07/13/2016 |
|
Unresectable or metastatic solid tumors with NTRK-fusion proteins in adult and pediatric patients who require systemic therapy and who have either progressed following prior treatment or who have no acceptable alternative treatments |
Cancer |
|
Date of BT Designation Disclosure: 07/13/2016
Approval Date: |
| Indication: Unresectable or metastatic solid tumors with NTRK-fusion proteins in adult and pediatric patients who require systemic therapy and who have either progressed following prior treatment or who have no acceptable alternative treatments |
| Category: Cancer |
|
|
Vitrakvi (larotrectinib) |
07/13/2016 |
11/26/2018 |
Adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion who have no satisfactory alternative treatments or that have progressed following treatment |
Cancer |
|
Date of BT Designation Disclosure: 07/13/2016
Approval Date: 11/26/2018 |
| Indication: Adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion who have no satisfactory alternative treatments or that have progressed following treatment |
| Category: Cancer |
|
|
Imbruvica (ibrutinib) |
06/29/2016 |
08/02/2017 |
Adult patients with chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy |
Infectious Disease |
|
Date of BT Designation Disclosure: 06/29/2016
Approval Date: 08/02/2017 |
| Indication: Adult patients with chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy |
| Category: Infectious Disease |
|
|
Crysvita (burosumab-twza) |
06/28/2016 |
04/17/2018 |
Patients aged 1 year and older with x-linked hypophosphatemia (XLH), a rare, inherited form of rickets |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 06/28/2016
Approval Date: 04/17/2018 |
| Indication: Patients aged 1 year and older with x-linked hypophosphatemia (XLH), a rare, inherited form of rickets |
| Category: Rare Inherited Disorders |
|
|
Opdivo (nivolumab) |
06/27/2016 |
02/02/2017 |
Patients with locally advanced or metastatic urothelial carcinoma in patients who have disease progression during or following platinum-containing chemotherapy; have disease progression within 12 months neo- or neoadjuvant treatment with platinum chemo |
Cancer |
|
Date of BT Designation Disclosure: 06/27/2016
Approval Date: 02/02/2017 |
| Indication: Patients with locally advanced or metastatic urothelial carcinoma in patients who have disease progression during or following platinum-containing chemotherapy; have disease progression within 12 months neo- or neoadjuvant treatment with platinum chemo |
| Category: Cancer |
|
|
Jakafi (Ruxolitinib) |
06/23/2016 |
05/28/2019 |
Patients aged 12 years and older with steroid-refractory acute graft versus-host disease (GVHD) |
Other |
|
Date of BT Designation Disclosure: 06/23/2016
Approval Date: 05/28/2019 |
| Indication: Patients aged 12 years and older with steroid-refractory acute graft versus-host disease (GVHD) |
| Category: Other |
|
|
SHP625 (maralixibat) |
06/13/2016 |
|
Progressive familial intrahepatic cholestasis type 2 (PFIC2) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 06/13/2016
Approval Date: |
| Indication: Progressive familial intrahepatic cholestasis type 2 (PFIC2) |
| Category: Rare Inherited Disorders |
|
|
SHP621 (budesonide oral suspension, or BOS) |
06/13/2016 |
|
Eosinophilic esophagitis (EoE) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 06/13/2016
Approval Date: |
| Indication: Eosinophilic esophagitis (EoE) |
| Category: Rare Inherited Disorders |
|
|
VYXEOS (cytarabine and daunorubicin) |
05/20/2016 |
08/03/2017 |
Patients with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) |
Cancer |
|
Date of BT Designation Disclosure: 05/20/2016
Approval Date: 08/03/2017 |
| Indication: Patients with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) |
| Category: Cancer |
|
|
PVS-RIPO |
05/15/2016 |
|
Recurrent glioblastoma multiforme (GBM) |
Cancer |
|
Date of BT Designation Disclosure: 05/15/2016
Approval Date: |
| Indication: Recurrent glioblastoma multiforme (GBM) |
| Category: Cancer |
|
|
Ilaris (canakinumab) |
04/27/2016 |
09/23/2016 |
Patients with Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/27/2016
Approval Date: 09/23/2016 |
| Indication: Patients with Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) |
| Category: Rare Inherited Disorders |
|
|
Ilaris (canakinumab) |
04/27/2016 |
09/23/2016 |
Patients with Hyperimmunoglobulin D Syndrome (HIDS) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/27/2016
Approval Date: 09/23/2016 |
| Indication: Patients with Hyperimmunoglobulin D Syndrome (HIDS) |
| Category: Rare Inherited Disorders |
|
|
Ilaris (canakinumab) |
04/27/2016 |
09/23/2016 |
Patients with Familial Mediterranean Fever (FMF) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/27/2016
Approval Date: 09/23/2016 |
| Indication: Patients with Familial Mediterranean Fever (FMF) |
| Category: Rare Inherited Disorders |
|
|
Opdivo (nivolumab) |
04/25/2016 |
11/10/2016 |
Patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum based therapy |
Cancer |
|
Date of BT Designation Disclosure: 04/25/2016
Approval Date: 11/10/2016 |
| Indication: Patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum based therapy |
| Category: Cancer |
|
|
Keytruda (pembrolizumab) |
04/18/2016 |
03/14/2017 |
Patients with hematological malignancies: Hodgkin Lymphoma |
Cancer |
|
Date of BT Designation Disclosure: 04/18/2016
Approval Date: 03/14/2017 |
| Indication: Patients with hematological malignancies: Hodgkin Lymphoma |
| Category: Cancer |
|
|
Rubraca (rucaparib) |
04/6/2016 |
12/19/2016 |
Patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more chemotherapies |
Cancer |
|
Date of BT Designation Disclosure: 04/6/2016
Approval Date: 12/19/2016 |
| Indication: Patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more chemotherapies |
| Category: Cancer |
|
|
NI-0501 |
03/16/2016 |
|
Primary Hemophagocytic Lymphohistiocytosis (HLH) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 03/16/2016
Approval Date: |
| Indication: Primary Hemophagocytic Lymphohistiocytosis (HLH) |
| Category: Rare Inherited Disorders |
|
|
Oliceridine (TRV130) |
02/22/2016 |
|
Moderate-to-Severe Acute Pain |
Other |
|
Date of BT Designation Disclosure: 02/22/2016
Approval Date: |
| Indication: Moderate-to-Severe Acute Pain |
| Category: Other |
|
|
PKC412 (midostaurin) |
02/19/2016 |
|
Newly-diagnosed FLT3-mutated AML |
Cancer |
|
Date of BT Designation Disclosure: 02/19/2016
Approval Date: |
| Indication: Newly-diagnosed FLT3-mutated AML |
| Category: Cancer |
|
|
Ocrevus (ocrelizumab) |
02/17/2016 |
03/28/2017 |
Patients with relapsing or primary progressive forms of multiple sclerosis |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 02/17/2016
Approval Date: 03/28/2017 |
| Indication: Patients with relapsing or primary progressive forms of multiple sclerosis |
| Category: Rare Inherited Disorders |
|
|
Durvalumab (MEDI4736) |
02/17/2016 |
|
PD-L1+ inoperable/metastatic urothelial bladder cancer |
Cancer |
|
Date of BT Designation Disclosure: 02/17/2016
Approval Date: |
| Indication: PD-L1+ inoperable/metastatic urothelial bladder cancer |
| Category: Cancer |
|
|
Imfinzi (durvalumab) |
02/16/2016 |
05/01/2017 |
Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neo- or adjuvant treatment with platinum-containing chemo |
Cancer |
|
Date of BT Designation Disclosure: 02/16/2016
Approval Date: 05/01/2017 |
| Indication: Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neo- or adjuvant treatment with platinum-containing chemo |
| Category: Cancer |
|
|
Affinity enhanced T-cell therapy |
02/9/2016 |
|
NY-ESO in synovial sarcoma |
Cancer |
|
Date of BT Designation Disclosure: 02/9/2016
Approval Date: |
| Indication: NY-ESO in synovial sarcoma |
| Category: Cancer |
|
|
Sacituzumab govitecan (IMMU-132) |
02/8/2016 |
|
Triple-Negative Breast Cancer (TNBC) |
Cancer |
|
Date of BT Designation Disclosure: 02/8/2016
Approval Date: |
| Indication: Triple-Negative Breast Cancer (TNBC) |
| Category: Cancer |
|
|
Rapastinel (GLYX-13) |
01/31/2016 |
|
Major Depressive Disorder (MDD) |
Other |
|
Date of BT Designation Disclosure: 01/31/2016
Approval Date: |
| Indication: Major Depressive Disorder (MDD) |
| Category: Other |
|
|
Rydapt (midostaurin) |
01/29/2016 |
04/28/2017 |
Adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive (FLT3+), as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation |
Cancer |
|
Date of BT Designation Disclosure: 01/29/2016
Approval Date: 04/28/2017 |
| Indication: Adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive (FLT3+), as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation |
| Category: Cancer |
|
|
Venetoclax |
01/28/2016 |
|
AML combination therapy |
Cancer |
|
Date of BT Designation Disclosure: 01/28/2016
Approval Date: |
| Indication: AML combination therapy |
| Category: Cancer |
|
|
Lynparza (olaparib) |
01/27/2016 |
|
Metastatic Castration Resistant Prostate Cancer |
Cancer |
|
Date of BT Designation Disclosure: 01/27/2016
Approval Date: |
| Indication: Metastatic Castration Resistant Prostate Cancer |
| Category: Cancer |
|
|
Venclexta (venetoclax) + Rituximab |
01/20/2016 |
06/08/2018 |
Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy |
Cancer |
|
Date of BT Designation Disclosure: 01/20/2016
Approval Date: 06/08/2018 |
| Indication: Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy |
| Category: Cancer |
|
|
Setmelanotide |
01/7/2016 |
|
Pro-opiomelanocortin (POMC) deficiency obesity and obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway (the MC4 pathway) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/7/2016
Approval Date: |
| Indication: Pro-opiomelanocortin (POMC) deficiency obesity and obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway (the MC4 pathway) |
| Category: Rare Inherited Disorders |
|
|
VTS 270 |
01/6/2016 |
|
Niemann-Pick Type C1 Disease (NPC) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/6/2016
Approval Date: |
| Indication: Niemann-Pick Type C1 Disease (NPC) |
| Category: Rare Inherited Disorders |
|
|
Darzalex (daratumumab) |
/2016 |
11/21/2016 |
Patients with multiple myeloma who have received at least one prior therapy, in combination with lenalidomide and dexamethasone |
Cancer |
|
Date of BT Designation Disclosure: /2016
Approval Date: 11/21/2016 |
| Indication: Patients with multiple myeloma who have received at least one prior therapy, in combination with lenalidomide and dexamethasone |
| Category: Cancer |
|
|
Orkambi (lumacaftor and ivacaftor) |
/2016 |
09/28/2016 |
Patients ages 6-11 with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: /2016
Approval Date: 09/28/2016 |
| Indication: Patients ages 6-11 with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene |
| Category: Rare Inherited Disorders |
|
|
Ibrance (palbociclib) |
/2016 |
02/19/2016 |
Patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer in combination with fulvestrant in women with disease progression following endocrine therapy |
Cancer |
|
Date of BT Designation Disclosure: /2016
Approval Date: 02/19/2016 |
| Indication: Patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer in combination with fulvestrant in women with disease progression following endocrine therapy |
| Category: Cancer |
|
|
BI 1482694 |
12/18/2015 |
|
Advanced/pre-treated EGFR mutation-positive NSCLC |
Cancer |
|
Date of BT Designation Disclosure: 12/18/2015
Approval Date: |
| Indication: Advanced/pre-treated EGFR mutation-positive NSCLC |
| Category: Cancer |
|
|
Yescarta (axicabtagene ciloleucel) |
12/7/2015 |
10/18/2017 |
Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy |
Cancer |
|
Date of BT Designation Disclosure: 12/7/2015
Approval Date: 10/18/2017 |
| Indication: Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy |
| Category: Cancer |
|
|
Bavencio (avelumab) |
11/18/2015 |
03/23/2017 |
Adult and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC) |
Cancer |
|
Date of BT Designation Disclosure: 11/18/2015
Approval Date: 03/23/2017 |
| Indication: Adult and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC) |
| Category: Cancer |
|
|
Austedo (deutetrabenazine) |
11/9/2015 |
08/30/2017 |
Patients with tardive dyskinesia |
Other |
|
Date of BT Designation Disclosure: 11/9/2015
Approval Date: 08/30/2017 |
| Indication: Patients with tardive dyskinesia |
| Category: Other |
|
|
Pembrolizumab (Keytruda) |
11/2/2015 |
|
Microsatellite Instability-High (MSI-H) metastatic colorectal cancer (mCRC) |
Cancer |
|
Date of BT Designation Disclosure: 11/2/2015
Approval Date: |
| Indication: Microsatellite Instability-High (MSI-H) metastatic colorectal cancer (mCRC) |
| Category: Cancer |
|
|
Turalio (pexidartinib) |
10/30/2015 |
08/02/2019 |
Tenosynovial Giant Cell Tumor (TGCT) |
Cancer |
|
Date of BT Designation Disclosure: 10/30/2015
Approval Date: 08/02/2019 |
| Indication: Tenosynovial Giant Cell Tumor (TGCT) |
| Category: Cancer |
|
|
Epclusa (sofosbuvir and velpatasvir) |
10/28/2015 |
06/28/2016 |
Adult patients with chronic hepatitis C virus (HCV) genotypes 1,2,3,4,5, or 6 infection: -without cirrhosis or with compensated cirrhosis -with decompensated cirrhosis for use in combination with ribavirin |
Infectious Disease |
|
Date of BT Designation Disclosure: 10/28/2015
Approval Date: 06/28/2016 |
| Indication: Adult patients with chronic hepatitis C virus (HCV) genotypes 1,2,3,4,5, or 6 infection: -without cirrhosis or with compensated cirrhosis -with decompensated cirrhosis for use in combination with ribavirin |
| Category: Infectious Disease |
|
|
Inotuzumab Ozogamicin |
10/19/2015 |
|
ALL |
Cancer |
|
Date of BT Designation Disclosure: 10/19/2015
Approval Date: |
| Indication: ALL |
| Category: Cancer |
|
|
RBX2660 |
10/12/2015 |
|
Recurrent Clostridium difficile (C diff) infection |
Infectious Disease |
|
Date of BT Designation Disclosure: 10/12/2015
Approval Date: |
| Indication: Recurrent Clostridium difficile (C diff) infection |
| Category: Infectious Disease |
|
|
Verzenio (abemaciclib) |
10/8/2015 |
02/26/2018 |
Adult patients with HR-positive, HER2-negative advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine-based therapy |
Cancer |
|
Date of BT Designation Disclosure: 10/8/2015
Approval Date: 02/26/2018 |
| Indication: Adult patients with HR-positive, HER2-negative advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine-based therapy |
| Category: Cancer |
|
|
Verzenio (abemaciclib) |
10/8/2015 |
09/28/2017 |
Patients with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting |
Cancer |
|
Date of BT Designation Disclosure: 10/8/2015
Approval Date: 09/28/2017 |
| Indication: Patients with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting |
| Category: Cancer |
|
|
Opdivo (nivolumab) |
09/16/2015 |
11/23/2015 |
Patients with advanced renal cell carcinoma who have received prior antiangiogenic therapy |
Cancer |
|
Date of BT Designation Disclosure: 09/16/2015
Approval Date: 11/23/2015 |
| Indication: Patients with advanced renal cell carcinoma who have received prior antiangiogenic therapy |
| Category: Cancer |
|
|
Hemlibra (emicizumab-kxwh) |
09/4/2015 |
11/16/2017 |
Prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A with factor VIII inhibitors |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 09/4/2015
Approval Date: 11/16/2017 |
| Indication: Prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A with factor VIII inhibitors |
| Category: Rare Inherited Disorders |
|
|
Opdivo (nivolumab) |
09/2/2015 |
10/09/2015 |
Patients with metastatic non-squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 09/2/2015
Approval Date: 10/09/2015 |
| Indication: Patients with metastatic non-squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy |
| Category: Cancer |
|
|
Brineura (cerliponase alfa) |
08/27/2015 |
04/27/2017 |
Pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 08/27/2015
Approval Date: 04/27/2017 |
| Indication: Pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency |
| Category: Rare Inherited Disorders |
|
|
Cabotetyx (cabozantinib) |
08/24/2015 |
04/25/2016 |
Patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy |
Cancer |
|
Date of BT Designation Disclosure: 08/24/2015
Approval Date: 04/25/2016 |
| Indication: Patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy |
| Category: Cancer |
|
|
Lenvima (lenvatinib mesylatel) |
07/29/2015 |
05/13/2016 |
Patients with renal cell carcinoma (RCC): in combination with evorlimus, for patients with advanced RCC following one prior anti-angiogenic therapy |
Cancer |
|
Date of BT Designation Disclosure: 07/29/2015
Approval Date: 05/13/2016 |
| Indication: Patients with renal cell carcinoma (RCC): in combination with evorlimus, for patients with advanced RCC following one prior anti-angiogenic therapy |
| Category: Cancer |
|
|
Azedra (iobenguane I 131) |
07/28/2015 |
07/30/2018 |
Adult and pediatric patients (12 years and older) with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) who require systemic anticancer therapy |
Cancer |
|
Date of BT Designation Disclosure: 07/28/2015
Approval Date: 07/30/2018 |
| Indication: Adult and pediatric patients (12 years and older) with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) who require systemic anticancer therapy |
| Category: Cancer |
|
|
Tafinlar (dabrafenib) |
07/24/2015 |
06/22/2017 |
Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test |
Cancer |
|
Date of BT Designation Disclosure: 07/24/2015
Approval Date: 06/22/2017 |
| Indication: Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test |
| Category: Cancer |
|
|
BMS-663068 |
07/21/2015 |
|
HIV-1 in heavily treatment-experienced adult patients |
Infectious Disease |
|
Date of BT Designation Disclosure: 07/21/2015
Approval Date: |
| Indication: HIV-1 in heavily treatment-experienced adult patients |
| Category: Infectious Disease |
|
|
DX-2930 |
07/7/2015 |
|
Hereditary angioedema (HAE) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 07/7/2015
Approval Date: |
| Indication: Hereditary angioedema (HAE) |
| Category: Rare Inherited Disorders |
|
|
Takhzyro (lanadelumab-flyo) |
07/2/2015 |
08/23/2018 |
Prevention of attacks of hereditary angioedema (HAE) in people 12 years of age and older |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 07/2/2015
Approval Date: 08/23/2018 |
| Indication: Prevention of attacks of hereditary angioedema (HAE) in people 12 years of age and older |
| Category: Rare Inherited Disorders |
|
|
AR101 |
06/18/2015 |
|
Peanut allergy in children and adolescents |
Other |
|
Date of BT Designation Disclosure: 06/18/2015
Approval Date: |
| Indication: Peanut allergy in children and adolescents |
| Category: Other |
|
|
SER-109 |
06/12/2015 |
|
Clostridium difficile infection (CDI) |
Infectious Disease |
|
Date of BT Designation Disclosure: 06/12/2015
Approval Date: |
| Indication: Clostridium difficile infection (CDI) |
| Category: Infectious Disease |
|
|
ACTEMRA/RoACTEMRA |
06/10/2015 |
|
Systemic scerlosis |
Other |
|
Date of BT Designation Disclosure: 06/10/2015
Approval Date: |
| Indication: Systemic scerlosis |
| Category: Other |
|
|
Lartruvo (olaratumab) |
06/8/2015 |
10/19/2016 |
Patients with soft tissue sarcoma (STS) with a histologic subtype, in combination with doxorubicin, for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery |
Cancer |
|
Date of BT Designation Disclosure: 06/8/2015
Approval Date: 10/19/2016 |
| Indication: Patients with soft tissue sarcoma (STS) with a histologic subtype, in combination with doxorubicin, for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery |
| Category: Cancer |
|
|
Olipudase alfa |
06/4/2015 |
|
Niemann-Pick disease Type B |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 06/4/2015
Approval Date: |
| Indication: Niemann-Pick disease Type B |
| Category: Rare Inherited Disorders |
|
|
Venclexta (venetoclax) |
05/6/2015 |
04/11/2016 |
Patients with chronic lymphocytic leukemia (CLL) with 17p deletion who have received at least one prior therapy |
Cancer |
|
Date of BT Designation Disclosure: 05/6/2015
Approval Date: 04/11/2016 |
| Indication: Patients with chronic lymphocytic leukemia (CLL) with 17p deletion who have received at least one prior therapy |
| Category: Cancer |
|
|
Xalkori (crizotinib) |
04/21/2015 |
03/11/2016 |
Patients with non-small cell lung cancer whose tumors are ROS-1 positive |
Cancer |
|
Date of BT Designation Disclosure: 04/21/2015
Approval Date: 03/11/2016 |
| Indication: Patients with non-small cell lung cancer whose tumors are ROS-1 positive |
| Category: Cancer |
|
|
Viaskin Peanut |
04/9/2015 |
|
Peanut allergy in children |
Other |
|
Date of BT Designation Disclosure: 04/9/2015
Approval Date: |
| Indication: Peanut allergy in children |
| Category: Other |
|
|
Zepatier (grazoprevir/elbasvir) |
04/8/2015 |
01/28/2016 |
Adult patients with chronic hepatitis C virus (HCV) genotypes 1 and 4 infections |
Infectious Disease |
|
Date of BT Designation Disclosure: 04/8/2015
Approval Date: 01/28/2016 |
| Indication: Adult patients with chronic hepatitis C virus (HCV) genotypes 1 and 4 infections |
| Category: Infectious Disease |
|
|
Rucaparib |
04/6/2015 |
|
Ovarian cancer |
Cancer |
|
Date of BT Designation Disclosure: 04/6/2015
Approval Date: |
| Indication: Ovarian cancer |
| Category: Cancer |
|
|
EBV-CTL |
03/2/2015 |
|
EBV-associated lymphoproliferative disease |
Cancer |
|
Date of BT Designation Disclosure: 03/2/2015
Approval Date: |
| Indication: EBV-associated lymphoproliferative disease |
| Category: Cancer |
|
|
Trogarzo (ibalizumab) |
02/27/2015 |
03/06/2018 |
Treatment of human immunodeficiency virus type 1 (HIV-1) infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen |
Infectious Disease |
|
Date of BT Designation Disclosure: 02/27/2015
Approval Date: 03/06/2018 |
| Indication: Treatment of human immunodeficiency virus type 1 (HIV-1) infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen |
| Category: Infectious Disease |
|
|
Rindopepimut (Rintega) |
02/23/2015 |
|
Glioblastoma (GBM) |
Cancer |
|
Date of BT Designation Disclosure: 02/23/2015
Approval Date: |
| Indication: Glioblastoma (GBM) |
| Category: Cancer |
|
|
tab-cel |
02/15/2015 |
|
Patients with EBV+ PTLD following allogeneic hematopoietic stem cell transplant |
Other |
|
Date of BT Designation Disclosure: 02/15/2015
Approval Date: |
| Indication: Patients with EBV+ PTLD following allogeneic hematopoietic stem cell transplant |
| Category: Other |
|
|
LentiGlobin |
02/2/2015 |
|
Beta-thalassemia |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 02/2/2015
Approval Date: |
| Indication: Beta-thalassemia |
| Category: Rare Inherited Disorders |
|
|
MPDL3280A |
02/1/2015 |
|
Non-small cell lung cancer |
Cancer |
|
Date of BT Designation Disclosure: 02/1/2015
Approval Date: |
| Indication: Non-small cell lung cancer |
| Category: Cancer |
|
|
Obeticholic acid (OCA) |
01/29/2015 |
|
Nonalcoholic steatohepatitis (NASH) |
Other |
|
Date of BT Designation Disclosure: 01/29/2015
Approval Date: |
| Indication: Nonalcoholic steatohepatitis (NASH) |
| Category: Other |
|
|
Rapamune (sirolimus) |
/2015 |
05/28/2015 |
Patients with lymphangioleiomyomatosis (LAM) |
Other |
|
Date of BT Designation Disclosure: /2015
Approval Date: 05/28/2015 |
| Indication: Patients with lymphangioleiomyomatosis (LAM) |
| Category: Other |
|
|
Lucentis (ranibizumab) |
12/15/2014 |
02/06/2015 |
Patients with Diabetic Retinopathy (DR) in patients with Diabetic Macula Edema (DME) |
Other |
|
Date of BT Designation Disclosure: 12/15/2014
Approval Date: 02/06/2015 |
| Indication: Patients with Diabetic Retinopathy (DR) in patients with Diabetic Macula Edema (DME) |
| Category: Other |
|
|
Ixazomib |
12/2/2014 |
|
Systemic light-chain (AL) amyloidosis |
Other |
|
Date of BT Designation Disclosure: 12/2/2014
Approval Date: |
| Indication: Systemic light-chain (AL) amyloidosis |
| Category: Other |
|
|
JCAR015 |
11/24/2014 |
|
ALL |
Cancer |
|
Date of BT Designation Disclosure: 11/24/2014
Approval Date: |
| Indication: ALL |
| Category: Cancer |
|
|
Dupixent (dupilumab) |
11/20/2014 |
03/28/2017 |
Adult patients with moderate-to-severe atopic dermatitis (AD) whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable |
Other |
|
Date of BT Designation Disclosure: 11/20/2014
Approval Date: 03/28/2017 |
| Indication: Adult patients with moderate-to-severe atopic dermatitis (AD) whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable |
| Category: Other |
|
|
SPK-RPE65 |
11/6/2014 |
|
Inherited retinal dystrophy (IRD) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 11/6/2014
Approval Date: |
| Indication: Inherited retinal dystrophy (IRD) |
| Category: Rare Inherited Disorders |
|
|
Ingrezza (valbenazine) |
10/30/2014 |
04/11/2017 |
Patients with tardive dyskinesia |
Other |
|
Date of BT Designation Disclosure: 10/30/2014
Approval Date: 04/11/2017 |
| Indication: Patients with tardive dyskinesia |
| Category: Other |
|
|
Keytruda (pembrolizumab) |
10/27/2014 |
10/02/2015 |
Patients with metastatic, PD-L1 positive, non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 10/27/2014
Approval Date: 10/02/2015 |
| Indication: Patients with metastatic, PD-L1 positive, non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy |
| Category: Cancer |
|
|
AP26113 |
10/2/2014 |
|
ALK-positive NSCLC |
Cancer |
|
Date of BT Designation Disclosure: 10/2/2014
Approval Date: |
| Indication: ALK-positive NSCLC |
| Category: Cancer |
|
|
Alunbrig (brigatinib) |
10/1/2014 |
04/28/2017 |
Patients with anaplastic lymphoma kinase (ALK)-positive mestastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib |
Cancer |
|
Date of BT Designation Disclosure: 10/1/2014
Approval Date: 04/28/2017 |
| Indication: Patients with anaplastic lymphoma kinase (ALK)-positive mestastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib |
| Category: Cancer |
|
|
Opdivo (nivolumab) |
09/26/2014 |
12/22/2014 |
Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600+, a BRAF inhibitor |
Cancer |
|
Date of BT Designation Disclosure: 09/26/2014
Approval Date: 12/22/2014 |
| Indication: Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600+, a BRAF inhibitor |
| Category: Cancer |
|
|
Luxturna (voretigene neparvovec-rzyl) |
09/24/2014 |
12/19/2017 |
Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 09/24/2014
Approval Date: 12/19/2017 |
| Indication: Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy |
| Category: Rare Inherited Disorders |
|
|
Eylea (aflibercept) |
09/16/2014 |
03/25/2015 |
Patients with diabetic retinopathy (DR) in patients with diabetic macular edema (DME) |
Other |
|
Date of BT Designation Disclosure: 09/16/2014
Approval Date: 03/25/2015 |
| Indication: Patients with diabetic retinopathy (DR) in patients with diabetic macular edema (DME) |
| Category: Other |
|
|
Nuplazid (pimavanserin) |
09/2/2014 |
04/29/2016 |
Patients with hallucinations and delusions associated with Parkinson's disease psychosis |
Other |
|
Date of BT Designation Disclosure: 09/2/2014
Approval Date: 04/29/2016 |
| Indication: Patients with hallucinations and delusions associated with Parkinson's disease psychosis |
| Category: Other |
|
|
CRS-207 and GVAX |
07/21/2014 |
|
Pancreatic cancer |
Cancer |
|
Date of BT Designation Disclosure: 07/21/2014
Approval Date: |
| Indication: Pancreatic cancer |
| Category: Cancer |
|
|
Esbriet (pirfenidone) |
07/17/2014 |
10/15/2014 |
Patients with idiopathic pulmonary fibrosis (IPF) |
Other |
|
Date of BT Designation Disclosure: 07/17/2014
Approval Date: 10/15/2014 |
| Indication: Patients with idiopathic pulmonary fibrosis (IPF) |
| Category: Other |
|
|
Ofev (nintedanib) |
07/16/2014 |
10/15/2014 |
Patients with idiopathic pulmonary fibrosis (IPF) |
Other |
|
Date of BT Designation Disclosure: 07/16/2014
Approval Date: 10/15/2014 |
| Indication: Patients with idiopathic pulmonary fibrosis (IPF) |
| Category: Other |
|
|
Kymriah (tisagenlecleucel) |
07/7/2014 |
08/30/2017 |
Pediatric and young adult patients (age 3-25 years) with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse |
Cancer |
|
Date of BT Designation Disclosure: 07/7/2014
Approval Date: 08/30/2017 |
| Indication: Pediatric and young adult patients (age 3-25 years) with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse |
| Category: Cancer |
|
|
Blincyto (blinatumomab) |
07/1/2014 |
12/03/2014 |
Patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia |
Cancer |
|
Date of BT Designation Disclosure: 07/1/2014
Approval Date: 12/03/2014 |
| Indication: Patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia |
| Category: Cancer |
|
|
Technivie (ombitasvir, paritaprevir, ritonavir) |
06/30/2014 |
07/24/2015 |
Patients with genotype 4 chronic hepatitis C viruse (HCV) infection without cirrhosis |
Infectious Disease |
|
Date of BT Designation Disclosure: 06/30/2014
Approval Date: 07/24/2015 |
| Indication: Patients with genotype 4 chronic hepatitis C viruse (HCV) infection without cirrhosis |
| Category: Infectious Disease |
|
|
Praxbind (idarucizumab) |
06/26/2014 |
10/16/2015 |
Patients treated with Pradaxa when reversal of the anticoagulant effects of dabigatran is needed for emergency surgery/urgent procedures and in life-threatening or uncontrolled bleeding |
Other |
|
Date of BT Designation Disclosure: 06/26/2014
Approval Date: 10/16/2015 |
| Indication: Patients treated with Pradaxa when reversal of the anticoagulant effects of dabigatran is needed for emergency surgery/urgent procedures and in life-threatening or uncontrolled bleeding |
| Category: Other |
|
|
Arikayce (amikacin liposome inhalation suspension) |
06/17/2014 |
09/28/2018 |
Patients with lung disease caused by Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment |
Infectious Disease |
|
Date of BT Designation Disclosure: 06/17/2014
Approval Date: 09/28/2018 |
| Indication: Patients with lung disease caused by Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment |
| Category: Infectious Disease |
|
|
Tecentriq (atezolizumab) |
05/31/2014 |
10/18/2016 |
Patients with metastatic non-small cell lung cancer who have disease progression during or following platinum-containing chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 05/31/2014
Approval Date: 10/18/2016 |
| Indication: Patients with metastatic non-small cell lung cancer who have disease progression during or following platinum-containing chemotherapy |
| Category: Cancer |
|
|
Tecentriq (atezolizumab) |
05/22/2014 |
05/18/2016 |
Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neo- or adjuvant treatment with platinum-containing chemotherapy |
Cancer |
|
Date of BT Designation Disclosure: 05/22/2014
Approval Date: 05/18/2016 |
| Indication: Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neo- or adjuvant treatment with platinum-containing chemotherapy |
| Category: Cancer |
|
|
Empliciti (elotuzumab) |
05/19/2014 |
11/30/2015 |
Patients with multiple myeloma who have received one to three prior therapies |
Cancer |
|
Date of BT Designation Disclosure: 05/19/2014
Approval Date: 11/30/2015 |
| Indication: Patients with multiple myeloma who have received one to three prior therapies |
| Category: Cancer |
|
|
Rociletinib (CO-1686) |
05/19/2014 |
|
NSCLC |
Cancer |
|
Date of BT Designation Disclosure: 05/19/2014
Approval Date: |
| Indication: NSCLC |
| Category: Cancer |
|
|
Opdivo (nivolumab) |
05/14/2014 |
05/17/2016 |
Patients with Hodgkin Lymphoma |
Cancer |
|
Date of BT Designation Disclosure: 05/14/2014
Approval Date: 05/17/2016 |
| Indication: Patients with Hodgkin Lymphoma |
| Category: Cancer |
|
|
Xuriden (uridine triacetate) |
04/30/2014 |
09/04/2015 |
Patients with hereditary orotic aciduria |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 04/30/2014
Approval Date: 09/04/2015 |
| Indication: Patients with hereditary orotic aciduria |
| Category: Rare Inherited Disorders |
|
|
Tagrisso (osimertinib) |
04/24/2014 |
11/13/2015 |
Patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive-non-small-cell lung cancer (NSCLC), as detected by and FDA approved test who have progressed on or after EGFR TKI therapy |
Cancer |
|
Date of BT Designation Disclosure: 04/24/2014
Approval Date: 11/13/2015 |
| Indication: Patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive-non-small-cell lung cancer (NSCLC), as detected by and FDA approved test who have progressed on or after EGFR TKI therapy |
| Category: Cancer |
|
|
MYDICAR |
04/10/2014 |
|
Heart failure |
Cardiovascular |
|
Date of BT Designation Disclosure: 04/10/2014
Approval Date: |
| Indication: Heart failure |
| Category: Cardiovascular |
|
|
Bexsero (Meningococcal Group B Vaccine) |
04/7/2014 |
01/23/2015 |
Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age |
Infectious Disease |
|
Date of BT Designation Disclosure: 04/7/2014
Approval Date: 01/23/2015 |
| Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age |
| Category: Infectious Disease |
|
|
Trumenba (Meningococcal Group B Vaccine) |
03/20/2014 |
10/29/2014 |
Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age |
Infectious Disease |
|
Date of BT Designation Disclosure: 03/20/2014
Approval Date: 10/29/2014 |
| Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age |
| Category: Infectious Disease |
|
|
Daclatasvir/asunaprevir combination |
02/24/2014 |
|
Hepatitis C |
Infectious Disease |
|
Date of BT Designation Disclosure: 02/24/2014
Approval Date: |
| Indication: Hepatitis C |
| Category: Infectious Disease |
|
|
Promacta (eltrombopag) |
02/3/2014 |
08/26/2014 |
Cytopenias in patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy |
Other |
|
Date of BT Designation Disclosure: 02/3/2014
Approval Date: 08/26/2014 |
| Indication: Cytopenias in patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy |
| Category: Other |
|
|
Orkambi (lumacaftor and ivacaftor) |
01/29/2014 |
07/02/2015 |
Patients 12 years and older with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/29/2014
Approval Date: 07/02/2015 |
| Indication: Patients 12 years and older with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene |
| Category: Rare Inherited Disorders |
|
|
Symdeko (tezacaftor + ivacaftor) |
01/28/2014 |
02/12/2018 |
Patients with cystic fibrosis aged 12 year and older who are homozygous for the F508del mutation who have at least one mutation in the CTFR gene that is responsive to Symdeko based on in vitro data or clinical evidence |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 01/28/2014
Approval Date: 02/12/2018 |
| Indication: Patients with cystic fibrosis aged 12 year and older who are homozygous for the F508del mutation who have at least one mutation in the CTFR gene that is responsive to Symdeko based on in vitro data or clinical evidence |
| Category: Rare Inherited Disorders |
|
|
Esketamine |
01/21/2014 |
|
Treatment resistant depression |
Other |
|
Date of BT Designation Disclosure: 01/21/2014
Approval Date: |
| Indication: Treatment resistant depression |
| Category: Other |
|
|
Dabrafenib |
01/13/2014 |
|
NSCLC w/ BRAF mutation |
Cancer |
|
Date of BT Designation Disclosure: 01/13/2014
Approval Date: |
| Indication: NSCLC w/ BRAF mutation |
| Category: Cancer |
|
|
Kalydeco (ivacaftor) |
/2014 |
12/29/2014 |
Patients age 6 years and older with cystic fibrosis who have the R117H mutation in the CF transmembrane conductance regulator (CTFR gene) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: /2014
Approval Date: 12/29/2014 |
| Indication: Patients age 6 years and older with cystic fibrosis who have the R117H mutation in the CF transmembrane conductance regulator (CTFR gene) |
| Category: Rare Inherited Disorders |
|
|
Krintafel (tafenoquine) |
12/18/2013 |
07/20/2018 |
Plasmodium vivax malaria |
Infectious Disease |
|
Date of BT Designation Disclosure: 12/18/2013
Approval Date: 07/20/2018 |
| Indication: Plasmodium vivax malaria |
| Category: Infectious Disease |
|
|
Andexxa (andexanet alfa) |
11/25/2013 |
05/03/2018 |
patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding |
Cardiovascular |
|
Date of BT Designation Disclosure: 11/25/2013
Approval Date: 05/03/2018 |
| Indication: patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding |
| Category: Cardiovascular |
|
|
Zydelig (idelalisib) |
11/18/2013 |
07/23/2014 |
Patients with relapsed chronic lymphocytic leukemia, in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other co-morbidities |
Cancer |
|
Date of BT Designation Disclosure: 11/18/2013
Approval Date: 07/23/2014 |
| Indication: Patients with relapsed chronic lymphocytic leukemia, in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other co-morbidities |
| Category: Cancer |
|
|
Spravato (esketamine) |
11/15/2013 |
03/05/2019 |
In conjunction with an oral antidepressant for the treatment of depression in adults who have tried other antidepressant medicines but have not benefited from them (treatment-resistant depression) |
Other |
|
Date of BT Designation Disclosure: 11/15/2013
Approval Date: 03/05/2019 |
| Indication: In conjunction with an oral antidepressant for the treatment of depression in adults who have tried other antidepressant medicines but have not benefited from them (treatment-resistant depression) |
| Category: Other |
|
|
Sovaldi (sofosbuvir) |
10/25/2013 |
12/06/2013 |
Patients with chronic hepatitis C infection |
Infectious Disease |
|
Date of BT Designation Disclosure: 10/25/2013
Approval Date: 12/06/2013 |
| Indication: Patients with chronic hepatitis C infection |
| Category: Infectious Disease |
|
|
cPMP (ALXN1011) |
10/24/2013 |
|
Molybdenum cofactor deficiency (MoCD) type A |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 10/24/2013
Approval Date: |
| Indication: Molybdenum cofactor deficiency (MoCD) type A |
| Category: Rare Inherited Disorders |
|
|
Grazoprevir/Elbasvir |
10/22/2013 |
|
Hepatitis C |
Infectious Disease |
|
Date of BT Designation Disclosure: 10/22/2013
Approval Date: |
| Indication: Hepatitis C |
| Category: Infectious Disease |
|
|
Alecensa (alectinib) |
09/23/2013 |
11/06/2017 |
Patients with anaplastic lymphoma kinase (ALK)-positive, metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test |
Cancer |
|
Date of BT Designation Disclosure: 09/23/2013
Approval Date: 11/06/2017 |
| Indication: Patients with anaplastic lymphoma kinase (ALK)-positive, metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test |
| Category: Cancer |
|
|
Volasertib |
09/17/2013 |
|
AML combination therapy |
Cancer |
|
Date of BT Designation Disclosure: 09/17/2013
Approval Date: |
| Indication: AML combination therapy |
| Category: Cancer |
|
|
Arzerra (ofatumumab) |
09/13/2013 |
04/17/2014 |
Previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate |
Cancer |
|
Date of BT Designation Disclosure: 09/13/2013
Approval Date: 04/17/2014 |
| Indication: Previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate |
| Category: Cancer |
|
|
Entinostat |
09/11/2013 |
|
ER-positive metastatic breast cancer combination |
Cancer |
|
Date of BT Designation Disclosure: 09/11/2013
Approval Date: |
| Indication: ER-positive metastatic breast cancer combination |
| Category: Cancer |
|
|
Firdapse (amifampridine) |
08/27/2013 |
11/28/2018 |
Patients with Lambert–Eaton myasthenic syndrome (LEMS) in adults |
Other |
|
Date of BT Designation Disclosure: 08/27/2013
Approval Date: 11/28/2018 |
| Indication: Patients with Lambert–Eaton myasthenic syndrome (LEMS) in adults |
| Category: Other |
|
|
BYM338 (Bimagrumab) |
08/20/2013 |
|
Sporadic inclusion body myositis (sIBM) |
Other |
|
Date of BT Designation Disclosure: 08/20/2013
Approval Date: |
| Indication: Sporadic inclusion body myositis (sIBM) |
| Category: Other |
|
|
Harvoni (sofosbuvir/ledipasvir) |
07/25/2013 |
10/10/2014 |
Patients with chronic hepatitis C, genotype 1 infection |
Infectious Disease |
|
Date of BT Designation Disclosure: 07/25/2013
Approval Date: 10/10/2014 |
| Indication: Patients with chronic hepatitis C, genotype 1 infection |
| Category: Infectious Disease |
|
|
Drisapersen |
06/27/2013 |
|
Duchenne muscular dystrophy (DMD) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 06/27/2013
Approval Date: |
| Indication: Duchenne muscular dystrophy (DMD) |
| Category: Rare Inherited Disorders |
|
|
Serelaxin |
06/21/2013 |
|
Acute heart failure |
Cardiovascular |
|
Date of BT Designation Disclosure: 06/21/2013
Approval Date: |
| Indication: Acute heart failure |
| Category: Cardiovascular |
|
|
Alecensa (alectinib) |
06/15/2013 |
12/11/2015 |
Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC), who have progressed on or are intolerant to crizotinib |
Cancer |
|
Date of BT Designation Disclosure: 06/15/2013
Approval Date: 12/11/2015 |
| Indication: Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC), who have progressed on or are intolerant to crizotinib |
| Category: Cancer |
|
|
Strensiq (asfotase alfa) |
05/28/2013 |
10/23/2015 |
Patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP) |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 05/28/2013
Approval Date: 10/23/2015 |
| Indication: Patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP) |
| Category: Rare Inherited Disorders |
|
|
Kanuma (sebelipase alfa) |
05/20/2013 |
12/08/2015 |
Patients with a diagnosis of lysosomal acid lipase (LAL) deficiency |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 05/20/2013
Approval Date: 12/08/2015 |
| Indication: Patients with a diagnosis of lysosomal acid lipase (LAL) deficiency |
| Category: Rare Inherited Disorders |
|
|
Gazyva (obinutuzumab) |
05/15/2013 |
11/01/2013 |
Patients with previously untreated chronic lymphocytic leukemia in combination with chlorambucil |
Cancer |
|
Date of BT Designation Disclosure: 05/15/2013
Approval Date: 11/01/2013 |
| Indication: Patients with previously untreated chronic lymphocytic leukemia in combination with chlorambucil |
| Category: Cancer |
|
|
Viekira Pak (ombitasvir, paritaprevir, ritonavir) |
05/6/2013 |
12/19/2014 |
Patients with genotype 1 chronic hepatitis C virus (HCV) infection including those with compensated cirrhosis |
Infectious Disease |
|
Date of BT Designation Disclosure: 05/6/2013
Approval Date: 12/19/2014 |
| Indication: Patients with genotype 1 chronic hepatitis C virus (HCV) infection including those with compensated cirrhosis |
| Category: Infectious Disease |
|
|
Darzalex (daratumumab) |
05/1/2013 |
11/16/2015 |
Patients with multiple myeloma who have received at least 3 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or are double refractory to a proteasome inhibitor and an immunomodulatory agent |
Cancer |
|
Date of BT Designation Disclosure: 05/1/2013
Approval Date: 11/16/2015 |
| Indication: Patients with multiple myeloma who have received at least 3 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or are double refractory to a proteasome inhibitor and an immunomodulatory agent |
| Category: Cancer |
|
|
SD101 |
04/29/2013 |
|
Epidermolysis bullosa |
Other |
|
Date of BT Designation Disclosure: 04/29/2013
Approval Date: |
| Indication: Epidermolysis bullosa |
| Category: Other |
|
|
Daclatasvir |
04/25/2013 |
|
Hepatitis C combination therapy |
Infectious Disease |
|
Date of BT Designation Disclosure: 04/25/2013
Approval Date: |
| Indication: Hepatitis C combination therapy |
| Category: Infectious Disease |
|
|
Keytruda (pembrolizumab) |
04/24/2013 |
12/18/2015 |
Patients with unresectable or metastatic melanoma |
Cancer |
|
Date of BT Designation Disclosure: 04/24/2013
Approval Date: 12/18/2015 |
| Indication: Patients with unresectable or metastatic melanoma |
| Category: Cancer |
|
|
Ibrance (palbociclib) |
04/10/2013 |
02/03/2015 |
Postmenopausal women with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer as initial endocrine-based therapy for their metastatic disease |
Cancer |
|
Date of BT Designation Disclosure: 04/10/2013
Approval Date: 02/03/2015 |
| Indication: Postmenopausal women with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer as initial endocrine-based therapy for their metastatic disease |
| Category: Cancer |
|
|
Imbruvica (ibrutinib) |
04/8/2013 |
07/28/2014 |
Patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy and CLL with 17p deletion |
Cancer |
|
Date of BT Designation Disclosure: 04/8/2013
Approval Date: 07/28/2014 |
| Indication: Patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy and CLL with 17p deletion |
| Category: Cancer |
|
|
Zykadia (ceritinib) |
03/15/2013 |
04/29/2014 |
Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib |
Cancer |
|
Date of BT Designation Disclosure: 03/15/2013
Approval Date: 04/29/2014 |
| Indication: Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib |
| Category: Cancer |
|
|
Opdivo (nivolumab) |
02/15/2013 |
09/04/2014 |
Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor |
Cancer |
|
Date of BT Designation Disclosure: 02/15/2013
Approval Date: 09/04/2014 |
| Indication: Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor |
| Category: Cancer |
|
|
Imbruvica (ibrutinib) |
02/12/2013 |
11/13/2013 |
Patients with mantle cell lymphoma (MCL) |
Cancer |
|
Date of BT Designation Disclosure: 02/12/2013
Approval Date: 11/13/2013 |
| Indication: Patients with mantle cell lymphoma (MCL) |
| Category: Cancer |
|
|
Imbruvica (ibrutinib) |
02/12/2013 |
01/29/2015 |
Patients with Waldenstrom's macroglobulinemia (WM) |
Cancer |
|
Date of BT Designation Disclosure: 02/12/2013
Approval Date: 01/29/2015 |
| Indication: Patients with Waldenstrom's macroglobulinemia (WM) |
| Category: Cancer |
|
|
Kalydeco (ivacaftor) |
/2013 |
02/21/2014 |
Patients age 6 years and older with cystic fibrosis who have mutations in the CTFR gene |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: /2013
Approval Date: 02/21/2014 |
| Indication: Patients age 6 years and older with cystic fibrosis who have mutations in the CTFR gene |
| Category: Rare Inherited Disorders |
|
|
Ivacaftor (Kalydeco) |
12/15/2012 |
|
CF combination therapy |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 12/15/2012
Approval Date: |
| Indication: CF combination therapy |
| Category: Rare Inherited Disorders |
|
|
Kalydeco (ivacaftor) |
12/15/2012 |
03/17/2015 |
Patients 2 years and older with cystic fibrosis and have that one of the following CFTR gene mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H |
Rare Inherited Disorders |
|
Date of BT Designation Disclosure: 12/15/2012
Approval Date: 03/17/2015 |
| Indication: Patients 2 years and older with cystic fibrosis and have that one of the following CFTR gene mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H |
| Category: Rare Inherited Disorders |
|
