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Breakthrough Therapies

The Breakthrough therapies chart is a list of all publicly announced breakthrough therapy designations since the program’s inception in 2012. The chart is updated daily and includes information about each individual agent or trade name of a drug or treatment, the sponsor company, the date of the designation’s public disclosure, the individual agent’s indication, or the broad disease category in which the designation was granted. The interactive search and sort feature lets you filter your results and download the table at any time into an excel format. Click here to learn more about the history of Breakthrough therapies. As of November 29, 2018 the FDA has approved 129* breakthrough therapy designated products and lists that there have been 724 total requests for the designation with 264** designations granted.

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Trade Name (Agent) Sponsor Date of BT Designation Disclosure Approval Date Indication Category
CP101 Finch Therapeutics Group, Inc. 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 AstraZeneca 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) Genentech Inc. 02/5/2019 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:
Indication: Patients with HER2-positive early breast cancer who have residual disease after neoadjuvant treatment
Category: Cancer
V114 Merck 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) TG Therapeutics 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) BeiGene, Ltd. 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) Novartis 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 Sanofi/Genzyme 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 Eiger BioPharmaceuticals 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) Seattle Genetics, Inc. 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) Novartis Pharmaceuticals Corp. 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
NGX-101 NeuroRx 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) UroGen Pharma 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 MYR Pharma 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 COMPASS Pathways 10/23/2018 Treatment-resistant Depression Other
Date of BT Designation Disclosure: 10/23/2018
Approval Date:
Indication: Treatment-resistant Depression
Category: Other
KD025 Kadmon Holdings, Inc. 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 Loxo Oncology, Inc. 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
LOXO-292 Loxo Oncology, Inc. 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
Rubraca (rucaparib) Clovis Oncology 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 Pfizer, Inc. 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 Concentric Analgesics, Inc. 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 AstraZeneca Plc & Amgen Inc. 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 Pfizer Inc. 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 Loxo Oncology, Inc. 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 Y-mAbs Therapeutics, Inc. 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) Genentech, Inc. 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) Array BioPharma and Eli Lilly and Co. 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 Daiichi Sankyo Company, Limited 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) Eisai Co. and Merck Sharp and Dohme Co. 08/1/2018 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:
Indication: Patients with previously untreated advanced and/or metastatic non-MSI-H/pMMR endometrial carcinoma
Category: Cancer
Tecentriq (atezolizumab) + Avastin (bevacizumab) Genentech Inc. 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) Genentech Inc. 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 uniQure N.V. 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 Heron Therapeutics, Inc. 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) Novartis Pharmaceuticals Corp. 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) Novartis Pharmaceuticals Corp. 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) Stemline Therapeutics, Inc. 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) Pfizer Inc. 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) Pfizer Inc. 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
Lenti-D Bluebird bio Inc. 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 Pfizer Inc. 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 Harmony Biosciences 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 Omeros Corporation 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) Pfizer Inc. 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) Genentech Inc. 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 Johnson & Johnson 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) Insmed Inc. 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) Fennec Pharmaceuticals Inc. 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 Astellas Pharma Inc. and Seattle Genetics Inc. 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
Erdafitinib Janssen Biotech Inc. 03/19/2018 Patients with metastatic urothelial cancer Cancer
Date of BT Designation Disclosure: 03/19/2018
Approval Date:
Indication: Patients with metastatic urothelial cancer
Category: Cancer
PTI-428 Proteostasis Therapeutics 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
Erdafitinib Janssen Pharmaceutical 03/15/2018 Urothelial cancer Cancer
Date of BT Designation Disclosure: 03/15/2018
Approval Date:
Indication: Urothelial cancer
Category: Cancer
Orkambi (lumacaftor/ivacaftor) Vertex Pharmaceuticals Inc. 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 Alnylam Pharmaceuticals, Inc. 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 Bioverativ Inc. 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 Galera Therapeutics 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 Bristol-Myers Squibb Company 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 Spark Therapeutics Inc. 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 Pfizer Inc. 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 Bristol-Myers Squibb Company 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 Sage Therapeutics 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) Zogenix, Inc. 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) Alnylam Pharmaceuticals, Inc. 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 Roche 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) Hoffman-La Roche Inc. 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
Lenvima (lenvatinib) in combination with Keytruda (pembrolizumab) Merck and Eisai 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) Abbvie 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
Voxelotor Global Blood Therapeutics, Inc. 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
Maribavir (SHP620) Shire 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) Novartis Pharmaceuticals Corp. 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) Novimmune and Sobi /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
Avelumab in combination with Inlyta (axitinib) EMD Serono and Pfizer 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) Novartis Pharmaceuticals Corp. 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
Oxervate (cenegermin-bkbj ophthalmic solution) Dompe farmaceutici 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 Celgene Corporation 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 BioMarin 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) Kyowa Kirin, Inc. 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) Abbvie Inc. and Genentech 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
MOR208 + Lenalidomide MorpoSys 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
Tafinlar (dabrafenib) + Mekinist (trametinib) Novartis Pharmaceuticals Corp. 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
Tafinler (dabrafenib) & Mekinist (trametinib) Novartis Pharmaceuticals Corp. 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
Opdivo (nivolumab) Bristol-Myers Squibb Company 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) Seattle Genetics Inc. 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 Ansun BioPharma 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) AstraZeneca 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) Seattle Genetics Inc. 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) Regeneron/Sanofi 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 Abeona Therapeutics Inc 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) Daiichi Sankyo 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) MAPS 08/28/2017 PTSD Other
Date of BT Designation Disclosure: 08/28/2017
Approval Date:
Indication: PTSD
Category: Other
Poteligeo (mogamulizumab-kpkc) Kyowa Kirin, Inc. 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) Wyeth Pharmaceuticals Inc. 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) Hoffmann-La Roche Inc. 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
Acalabrutinib AstraZeneca 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
Venetoclax Abbvie/Roche 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
Calquence (acalabrutinib) AstraZeneca UK Ltd. 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
Imfinzi (durvalumab) AstraZeneca/MedImmune 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) Merck & Co. Inc. 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) Cara Therapeutics Inc. 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 Omeros 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 Y-mAbs Therapeutics, Inc. 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 Blueprint Medicines Corp. 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) Alnylam 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) Achaogen 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 GlycoMimetics 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
Entrectinib Ignyta 05/15/2017 NTRK fusion–positive locally advanced or metastatic solid tumors Cancer
Date of BT Designation Disclosure: 05/15/2017
Approval Date:
Indication: NTRK fusion–positive locally advanced or metastatic solid tumors
Category: Cancer
Ribaxamase (SYN-004) Synthetic Biologics, Inc. 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 Proteon Therapeutics, Inc. 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 Pfizer 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 (CTL019) Novartis 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
Kymriah (tisagenlecleucel) Novartis Pharmaceutical Corp. 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
RVT-802 Enzyvant 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 Regeneron 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) Genentech 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) Merck Sharp & Dohme Corp. 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 Tocagen 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 uniQure 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
Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. /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
Mekinist (trametinib) Novartis Pharmaceuticals Corp. /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
Zykadia (ceritinib) Novartis Pharmaceuticals Corp. /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
Ibrance (palbociclib) Pfizer Inc. /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
Nuplazid (pimavanserin) Acadia Pharmaceuticals Inc. /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) Merck Sharp & Dohme Corp. /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) Merck Sharp & Dohme Corp. /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
Liso-cel (Lisocabtagene maraleucel) Juno Therapeutics/Celgene 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) Tonix Pharmaceuticals 12/19/2016 PTSD Other
Date of BT Designation Disclosure: 12/19/2016
Approval Date:
Indication: PTSD
Category: Other
Neridronic acid Grünenthal and Abiogen Pharma 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% Elevate Oral Care 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) Tesaro Inc. 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 Gamida Cell 10/10/2016 Nicord Cancer
Date of BT Designation Disclosure: 10/10/2016
Approval Date:
Indication: Nicord
Category: Cancer
Actemra (tocilizumab) Genentech Inc. 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) Roche 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) Regeneron/Sanofi 10/1/2016 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:
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) AbbVie Inc. 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
Brexanolone (SAGE-547) Sage Therapeutics 09/6/2016 Postpartum depression (PPD) Other
Date of BT Designation Disclosure: 09/6/2016
Approval Date:
Indication: Postpartum depression (PPD)
Category: Other
SL-401 Stemline Therapeutics 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 Janssen 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) Novartis Pharmaceuticals Corp. 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) Novartis Pharmaceuticals Corp. 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 MEI Pharma 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) Janssen Biotech Inc. 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 Merck 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 Pfizer/Spark 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- xxxx) AveXis 07/20/2016 Spinal muscular atrophy (SMA) Type 1 in pediatric patients Other
Date of BT Designation Disclosure: 07/20/2016
Approval Date:
Indication: Spinal muscular atrophy (SMA) Type 1 in pediatric patients
Category: Other
Vitrakvi (larotrectinib) Loxo Oncology 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
Larotrectinib Loxo Oncology, Inc. 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
Imbruvica (ibrutinib) Pharmacyclics LLC 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) Ultragenyx Pharmaceutical Inc. 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) Bristol-Myers Squibb Company 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
Ruxolitinib (Jakafi) Incyte 06/23/2016 Acute graft-versus-host disease (GVHD) Other
Date of BT Designation Disclosure: 06/23/2016
Approval Date:
Indication: Acute graft-versus-host disease (GVHD)
Category: Other
SHP621 (budesonide oral suspension, or BOS) Shire 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
SHP625 (maralixibat) Shire 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
VYXEOS (cytarabine and daunorubicin) Celator Pharmaceuticals Inc. 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 Duke Medicine 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) Novartis Pharmaceuticals Corp. 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) Novartis Pharmaceuticals Corp. 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
Ilaris (canakinumab) Novartis Pharmaceuticals Corp. 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
Opdivo (nivolumab) Bristol-Myers Squibb Company 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) Merck Sharp & Dohme Corp. 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) Clovis Oncology Inc. 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 Novimmune 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) Trevena, Inc. 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) Novartis 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) Genentech Inc. 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) AstraZeneca 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) AstraZeneca UK Ltd. 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 Adaptimmune 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) Immunomedics 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) Allergan 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) Novartis Pharmaceuticals Corp. 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 AbbVie/Roche 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) AstraZeneca 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 AbbVie Inc. and Genentech Inc. 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 Rhythm 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 Vtesse 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) Janssen Biotech Inc. /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) Vertex Pharmaceuticals Inc. /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) Pfizer Inc. /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 Boehringer Ingelheim 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) Kite Pharma Inc. 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) EMD Serono Inc. 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) Teva Branded Pharmaceutical Products R and D Inc. 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) Merck 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
Pexidartinib (formerly PLX3397) Daiichi Sankyo 10/30/2015 Tenosynovial Giant Cell Tumor (TGCT) Cancer
Date of BT Designation Disclosure: 10/30/2015
Approval Date:
Indication: Tenosynovial Giant Cell Tumor (TGCT)
Category: Cancer
Epclusa (sofosbuvir and velpatasvir) Gilead Sciences Inc. 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 Pfizer 10/19/2015 ALL Cancer
Date of BT Designation Disclosure: 10/19/2015
Approval Date:
Indication: ALL
Category: Cancer
RBX2660 Rebiotix, Inc. 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) Eli Lilly and Co. 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
Verzenio (abemaciclib) Eli Lilly and Co. 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
Opdivo (nivolumab) Bristol-Myers Squibb Company 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) Genentech Inc. 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) Bristol-Myers Squibb Company 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) Biomarin Pharmaceutical Inc. 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) Exelixis Inc. 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) Eisai Inc. 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) Progenics Pharmaceuticals Inc. 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) Novartis Pharmaceuticals Corp. 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 BMS 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  Dyax 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) Shire 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 Aimmune 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 Seres 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 Genentech/Roche 06/10/2015 Systemic scerlosis Other
Date of BT Designation Disclosure: 06/10/2015
Approval Date:
Indication: Systemic scerlosis
Category: Other
Lartruvo (olaratumab) Eli Lilly and Co. 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 Sanofi/Genzyme 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) AbbVie Inc. 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) PF Prism CV 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 DBV 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) Merck Sharp & Dohme Corp. 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 Clovis 04/6/2015 Ovarian cancer Cancer
Date of BT Designation Disclosure: 04/6/2015
Approval Date:
Indication: Ovarian cancer
Category: Cancer
EBV-CTL Atara and MSKCC 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) TaiMed Biologics USA Corp 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) Celldex 02/23/2015 Glioblastoma (GBM) Cancer
Date of BT Designation Disclosure: 02/23/2015
Approval Date:
Indication: Glioblastoma (GBM)
Category: Cancer
tab-cel Altara Biotherapeutics Inc. 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 BlueBird 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 Genentech/Roche 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) Intercept 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) PF Prism CV /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) Genentech Inc. 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 Takeda 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 Juno Therapeutics 11/24/2014 ALL Cancer
Date of BT Designation Disclosure: 11/24/2014
Approval Date:
Indication: ALL
Category: Cancer
Dupixent (dupilumab) Regeneron Pharmaceuticals Inc. 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 Spark Therapeutics 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) Neurocrine Biosciences Inc. 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) Merck Sharp & Dohme Corp. 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 Ariad 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) Ariad Pharmaceuticals Inc. 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) Bristol-Myers Squibb Company 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) Spark Therapeutics Inc. 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) Regeneron Pharmaceuticals Inc. 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) Acadia Pharmaceuticals Inc. 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 Aduro 07/21/2014 Pancreatic cancer Cancer
Date of BT Designation Disclosure: 07/21/2014
Approval Date:
Indication: Pancreatic cancer
Category: Cancer
Esbriet (pirfenidone) Genentech Inc. 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) Boehringer Ingelheim Pharmaceuticals Inc. 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) Novartis Pharmaceuticals Corp. 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) Amgen Inc. 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) AbbVie Inc. 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) Boehringer Ingelheim Pharmaceuticals Inc. 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) Insmed 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) Genentech Inc. 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) Genentech Inc. 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) Bristol-Myers Squibb Company 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) Clovis Oncology 05/19/2014 NSCLC Cancer
Date of BT Designation Disclosure: 05/19/2014
Approval Date:
Indication: NSCLC
Category: Cancer
Opdivo (nivolumab) Bristol-Myers Squibb Company 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) Wellstat Therapeutics Corp. 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)  AstraZeneca Pharmaceuticals LP 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 Celladon  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) Novartis Vaccines and Diagnostics, Inc. 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) Pfizer Inc. 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 BMS 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) Novartis Pharmaceuticals Corp. 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) Vertex Pharmaceuticals Inc. 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) Vertex Pharmaceuticals Inc. 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 J&J 01/21/2014 Treatment resistant depression Other
Date of BT Designation Disclosure: 01/21/2014
Approval Date:
Indication: Treatment resistant depression
Category: Other
Dabrafenib GlaxoSmithKline 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) Vertex Pharmaceuticals Inc. /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) GlaxoSmithKline 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) Portola Pharmaceuticals, Inc. 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) Gilead Sciences Inc. 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
Sovaldi (sofosbuvir) Gilead Sciences Inc. 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) Alexion 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 Merck 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) Hoffman-La Roche Inc. 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 Boehringer Ingelheim 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) Novartis Pharmaceuticals Corp. 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 Syndax 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) Catalyst 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) Novartis 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) Gilead Sciences Inc. 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 GlaxoSmithKline/Prosensa 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  Novartis 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) Hoffman-La Roche Inc. 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) Alexion Pharmaceuticals Inc. 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) Synageva Biopharma Corp. 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) Genentech Inc. 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) AbbVie Inc. 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) Janssen Biotech Inc. 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 Scioderm 04/29/2013 Epidermolysis bullosa Other
Date of BT Designation Disclosure: 04/29/2013
Approval Date:
Indication: Epidermolysis bullosa
Category: Other
Daclatasvir BMS 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) Merck Sharp & Dohme Corp. 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) Pfizer Inc. 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) Pharmacyclics LLC 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) Novartis Pharmaceuticals Corp. 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) Bristol-Myers Squibb Company 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) Pharmacyclics LLC 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) Pharmacyclics LLC 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) Vertex Pharmaceuticals Inc. /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) Vertex 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) Vertex Pharmaceuticals Inc. 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
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