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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 January 3, 2018 the FDA has approved 68* breakthrough therapy designated products and lists that there have been 603 total requests for the designation with 210** designations granted.

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Agent (Trade Name) Sponsor Date of BT Designation Disclosure Indication Category
SAGE-217 Sage Therapeutics 02/7/2018 Patients with major depressive disorder (MDD) Other
Date of BT Designation Disclosure: 02/7/2018
Indication: Patients with major depressive disorder (MDD)
Category: Other
PF-04965842 Pfizer Inc. 02/14/2018 Patients with moderate-to-severe atopic dermatitis (AD) Other
Date of BT Designation Disclosure: 02/14/2018
Indication: Patients with moderate-to-severe atopic dermatitis (AD)
Category: Other
Patisiran Alnylam Pharmaceuticals 02/2/2018 Patients with hereditary (hATTR) amyloidosis Rare Inherited Disorders
Date of BT Designation Disclosure: 02/2/2018
Indication: Patients with hereditary (hATTR) amyloidosis
Category: Rare Inherited Disorders
ZX008 (fenfluramine) Zogenix, Inc. 02/6/2018 Patients who have seizures associated with Dravet syndrome Other
Date of BT Designation Disclosure: 02/6/2018
Indication: Patients who have seizures associated with Dravet syndrome
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
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
Indication: Adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy
Category: Other
Balovaptan Roche 01/29/2018 Autism spectrum disorder (ASD) Rare Inherited Disorders
Date of BT Designation Disclosure: 01/29/2018
Indication: Autism spectrum disorder (ASD)
Category: Rare Inherited Disorders
Voxelotor Global Blood Therapeutics, Inc. 01/9/2018 Sickle cell disease (SCD) Rare Inherited Disorders
Date of BT Designation Disclosure: 01/9/2018
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
Indication: Cytomegalovirus (CMV) infection in transplant patients.
Category: Infectious Disease
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
Indication: A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD)
Category: Other
Adcetris (brentuximab vedotin) Seattle Genetics Inc. 01/2/2018 In combination with chemotherapy for the first line treatment of patients with advanced classical Hodgkin lymphoma Cancer
Date of BT Designation Disclosure: 01/2/2018
Indication: In combination with chemotherapy for the first line treatment of patients with advanced classical Hodgkin lymphoma
Category: Cancer
Luxturna (voretigene neparvovec-rzyl) Spark Therapeutics Inc. 12/19/2017 Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients Rare Inherited Disorders
Date of BT Designation Disclosure: 12/19/2017
Indication: Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients
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
Indication: Treatment-naïve patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Prevymis (letermovir) Merck Sharp & Dohme Corp. 11/8/2017 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: 11/8/2017
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
Adcetris (brentuximab vedotin) Seattle Genetics Inc. 10/2/2017 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
Indication: Patients with primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides who have received prior systemic therapy
Category: Cancer
Opdivo (nivolumab) Bristol-Myers Squibb Company 10/17/2017 Adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease, and who have undergone complete resection Cancer
Date of BT Designation Disclosure: 10/17/2017
Indication: Adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease, and who have undergone complete resection
Category: Cancer
Osimertinib (Tagrisso) AstraZeneca 10/9/2017 First line treatment of patients with EGFR mutation-positive non-small cell lung cancer Cancer
Date of BT Designation Disclosure: 10/9/2017
Indication: First line treatment of patients with EGFR mutation-positive non-small cell lung cancer
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
Indication: Treatment of lower respiratory tract parainfluenza virus (PIV) infection in immunocompromised patients
Category: Infectious Disease
Tafinlar + Mekinist Novartis 10/23/2017 Adjuvant treatment of patients with stage III melanoma with a BRAF V600 mutation following complete resection Cancer
Date of BT Designation Disclosure: 10/23/2017
Indication: Adjuvant treatment of patients with stage III melanoma with a BRAF V600 mutation following complete resection
Category: Cancer
Mogamulizumab Poteligeo, Kyowa Hakko Kirin 10/25/2017 adults with mycosis fungoides and Sézary syndrome who received at least one prior systemic therapy Other
Date of BT Designation Disclosure: 10/25/2017
Indication: adults with mycosis fungoides and Sézary syndrome who received at least one prior systemic therapy
Category: Other
Venetoclax Abbvie, Roche 10/25/2017 In combination with cytarabine for elderly patients with treatment-naive acute myeloid leukemia who are ineligible for intensive chemotherapy Cancer
Date of BT Designation Disclosure: 10/25/2017
Indication: In combination with cytarabine for elderly patients with treatment-naive acute myeloid leukemia who are ineligible for intensive chemotherapy
Category: Cancer
Valoctocogene Roxaparvovec BioMarin 10/26/2017 Hemophilia A Rare Inherited Disorders
Date of BT Designation Disclosure: 10/26/2017
Indication: Hemophilia A
Category: Rare Inherited Disorders
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
Indication: Relapsed/Refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation
Category: Cancer
Cemiplimab (REGN2810) Regeneron/Sanofi 09/8/2017 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
Indication: Adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC
Category: Cancer
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
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
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
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
Indication: Elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy
Category: Cancer
Zelboraf (vemurafenib) Hoffmann-La Roche Inc. 08/7/2017 Patients with Erdheim-Chester disease (ECD) with a BRAF V600 mutation Other
Date of BT Designation Disclosure: 08/7/2017
Indication: Patients with Erdheim-Chester disease (ECD) with a BRAF V600 mutation
Category: Other
Calquence (acalabrutinib) AstraZeneca UK Ltd. 08/1/2017 Patients with mantle cell lymphoma who have received at least one prior therapy Cancer
Date of BT Designation Disclosure: 08/1/2017
Indication: Patients with mantle cell lymphoma who have received at least one prior therapy
Category: Cancer
Mogamulizumab Kyowa Hakko Kirin Co, Ltd 08/25/2017 Treatment of 2 subtytes of cutaneous T-cell lymphoma (CTCL) – mycosis fungoisis (MF) and Sézary syndrome (SS) Cancer
Date of BT Designation Disclosure: 08/25/2017
Indication: Treatment of 2 subtytes of cutaneous T-cell lymphoma (CTCL) – mycosis fungoisis (MF) and Sézary syndrome (SS)
Category: Cancer
Besponsa (inotuzumab ozogamicin) Wyeth Pharmaceuticals Inc. 08/17/2017 Treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) Cancer
Date of BT Designation Disclosure: 08/17/2017
Indication: Treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)
Category: Cancer
Methylenedioxymethamphetamine (MDMA) MAPS 08/28/2017 PTSD Other
Date of BT Designation Disclosure: 08/28/2017
Indication: PTSD
Category: Other
EB-101 Abeona Therapeutics Inc 08/30/2017 Recessive Dystrophic Epidermolysis Bullosa (RDEB) Other
Date of BT Designation Disclosure: 08/30/2017
Indication: Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Category: Other
Imfinzi (durvalumab) AstraZeneca/MedImmune 07/31/2017 Locally-advanced, unresectable non-small cell lung cancer (NSCLC) whose disease has not progressed following platinum-based chemoradiation therapy Cancer
Date of BT Designation Disclosure: 07/31/2017
Indication: Locally-advanced, unresectable non-small cell lung cancer (NSCLC) whose disease has not progressed following platinum-based chemoradiation therapy
Category: Cancer
Mekinist (trametinib) Novartis Pharmaceuticals Corp. 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: 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
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
Indication: Pediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis
Category: Cancer
OMS721 Omeros 06/13/2017 Immunoglobulin A (IgA) nephropathy Other
Date of BT Designation Disclosure: 06/13/2017
Indication: Immunoglobulin A (IgA) nephropathy
Category: Other
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
Indication: Chronic kidney disease, associated pruritius in hemodialysis patients
Category: Other
Kisqali Femara Co-Pack (letrozole and ribociclib) Novartis Pharmaceuticals Corp. 05/4/2017 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: 05/4/2017
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer
Category: Cancer
Imfinzi (durvalumab) AstraZeneca UK Ltd. 05/1/2017 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: 05/1/2017
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
Entrectinib Ignyta 05/15/2017 NTRK fusion–positive locally advanced or metastatic solid tumors Cancer
Date of BT Designation Disclosure: 05/15/2017
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
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
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
GMI-1271 GlycoMimetics 05/17/2017 Adult Relapsed/Refractory Acute Myeloid Leukemia Cancer
Date of BT Designation Disclosure: 05/17/2017
Indication: Adult Relapsed/Refractory Acute Myeloid Leukemia
Category: Cancer
Givosiran (ALN-AS1) Alnylam 05/30/2017 Acute hepatic porphyria (AHP) Rare Inherited Disorders
Date of BT Designation Disclosure: 05/30/2017
Indication: Acute hepatic porphyria (AHP)
Category: Rare Inherited Disorders
Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. 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 Cancer
Date of BT Designation Disclosure: 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
Category: Cancer
Plazomicin Achaogen 05/23/2017 Antibacterials addressing multi-drug resistant (MDR) gram-negative infections Other
Date of BT Designation Disclosure: 05/23/2017
Indication: Antibacterials addressing multi-drug resistant (MDR) gram-negative infections
Category: Other
Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. 05/1/2017 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: 05/1/2017
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
Alunbrig (brigatinib) Ariad Pharmaceuticals Inc. 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: 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
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
Indication: ALK-positive metastatic non-small cell lung cancer (NSCLC) who have previously received 1 or more ALK inhibitors
Category: Cancer
Rydapt (midostaurin) Novartis Pharmaceuticals Corp. 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: 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
Brineura (cerliponase alfa) Biomarin Pharmaceutical Inc. 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: 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
Evinacumab Regeneron 04/7/2017 Homozygous familial hypercholesterolemia (HOFH) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/7/2017
Indication: Homozygous familial hypercholesterolemia (HOFH)
Category: Rare Inherited Disorders
RVT-802 Enzyvant 04/17/2017 Complete DiGeorge Syndrome (cDGS) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/17/2017
Indication: Complete DiGeorge Syndrome (cDGS)
Category: Rare Inherited Disorders
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
Indication: Adult patients with relapsed and refractory diffuse large B-cell lymphoma, who have failed two or more prior therapies
Category: Cancer
Rituximab (Rituxin) Genentech 03/24/2017 Pemphigus vulgaris Rare Inherited Disorders
Date of BT Designation Disclosure: 03/24/2017
Indication: Pemphigus vulgaris
Category: Rare Inherited Disorders
Toca 511 & Toca FC Tocagen 02/23/2017 Recurrent high grade glioma Cancer
Date of BT Designation Disclosure: 02/23/2017
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
Indication: Hemophilia B
Category: Rare Inherited Disorders
Nuplazid (pimavanserin) Acadia Pharmaceuticals Inc. /2017 Patients with dementia-related psychosis (DRP) Other
Date of BT Designation Disclosure: /2017
Indication: Patients with dementia-related psychosis (DRP)
Category: Other
JCAR017 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
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
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
Indication: Complex regional pain syndrome (CRPS)
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
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
Elbit Imaging Gamida Cell 10/10/2016 Nicord Cancer
Date of BT Designation Disclosure: 10/10/2016
Indication: Nicord
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
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
Actemra (tocilizumab) Genentech Inc. 10/5/2016 Adult patients with giant cell arteritis (GCA) Rare Inherited Disorders
Date of BT Designation Disclosure: 10/5/2016
Indication: Adult patients with giant cell arteritis (GCA)
Category: Rare Inherited Disorders
SAGE-547 Sage Therapeutics 09/6/2016 Postpartum depression (PPD) in women Other
Date of BT Designation Disclosure: 09/6/2016
Indication: Postpartum depression (PPD) in women
Category: Other
Mavyret (glecaprevir & pibrentasvir) AbbVie Inc. 09/30/2016 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
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
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
Indication: In combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy
Category: Cancer
Esketamine Janssen 08/16/2016 Major depressive disorder with imminent risk for suicide Other
Date of BT Designation Disclosure: 08/16/2016
Indication: Major depressive disorder with imminent risk for suicide
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
Indication: Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123)
Category: Rare Inherited Disorders
Kisqali (ribociclib) Novartis Pharmaceuticals Corp. 08/3/2016 First-line treatment for patients with HR+/HER2- advanced breast cancer; Initial endocrine-based treatment of pre- or perimenopausal women with HR+/HER2- advanced or metastatic breast cancer in combination w/ tamoxifen or an aromatase inhibitor Cancer
Date of BT Designation Disclosure: 08/3/2016
Indication: First-line treatment for patients with HR+/HER2- advanced breast cancer; Initial endocrine-based treatment of pre- or perimenopausal women with HR+/HER2- advanced or metastatic breast cancer in combination w/ tamoxifen or an aromatase inhibitor
Category: Cancer
V920 Merck 07/25/2016 Ebola Zaire Infectious Disease
Date of BT Designation Disclosure: 07/25/2016
Indication: Ebola Zaire
Category: Infectious Disease
LOXO-101 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
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
SPK-9001 Pfizer/Spark 07/21/2016 Hemophilia B. Rare Inherited Disorders
Date of BT Designation Disclosure: 07/21/2016
Indication: Hemophilia B.
Category: Rare Inherited Disorders
AVXS-101 AveXis 07/20/2016 Spinal muscular atrophy (SMA) Type 1 in pediatric patients Other
Date of BT Designation Disclosure: 07/20/2016
Indication: Spinal muscular atrophy (SMA) Type 1 in pediatric patients
Category: Other
SHP625 (maralixibat) Shire 06/13/2016 Progressive familial intrahepatic cholestasis type 2 (PFIC2) Rare Inherited Disorders
Date of BT Designation Disclosure: 06/13/2016
Indication: Progressive familial intrahepatic cholestasis type 2 (PFIC2)
Category: Rare Inherited Disorders
Ruxolitinib (Jakafi) Incyte 06/23/2016 Acute graft-versus-host disease (GVHD) Other
Date of BT Designation Disclosure: 06/23/2016
Indication: Acute graft-versus-host disease (GVHD)
Category: Other
KRN23 Ultragenyx 06/28/2016 X-linked hypophosphatemia (XLH) in pediatric patients one year of age and older Rare Inherited Disorders
Date of BT Designation Disclosure: 06/28/2016
Indication: X-linked hypophosphatemia (XLH) in pediatric patients one year of age and older
Category: Rare Inherited Disorders
SHP621 (budesonide oral suspension, or BOS) Shire 06/13/2016 Eosinophilic esophagitis (EoE) Rare Inherited Disorders
Date of BT Designation Disclosure: 06/13/2016
Indication: Eosinophilic esophagitis (EoE)
Category: Rare Inherited Disorders
Opdivo (nivolumab) Bristol-Myers Squibb Company 06/27/2016 Patients with locally advanced or metastatic urothelial carcinoma in patients who have disease progression during or following platinum-containing chemotherapy Cancer
Date of BT Designation Disclosure: 06/27/2016
Indication: Patients with locally advanced or metastatic urothelial carcinoma in patients who have disease progression during or following platinum-containing chemotherapy
Category: Cancer
Imbruvica (ibrutinib) Pharmacyclics LLC 06/29/2016 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
Indication: Adult patients with chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy
Category: Infectious Disease
PVS-RIPO Duke Medicine 05/15/2016 Recurrent glioblastoma multiforme (GBM) Cancer
Date of BT Designation Disclosure: 05/15/2016
Indication: Recurrent glioblastoma multiforme (GBM)
Category: Cancer
Tecentriq (atezolizumab) Genentech Inc. 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 neo-adjuvant treatment with platinum-containing chemotherapy Cancer
Date of BT Designation Disclosure: 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 neo-adjuvant treatment with platinum-containing chemotherapy
Category: Cancer
VYXEOS (cytarabine and daunorubicin) Celator Pharmaceuticals Inc. 05/20/2016 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
Indication: Patients with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)
Category: Cancer
Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. 04/18/2016 Patients with hematological malignancies: Hodgkin Lymphoma Cancer
Date of BT Designation Disclosure: 04/18/2016
Indication: Patients with hematological malignancies: Hodgkin Lymphoma
Category: Cancer
Rubraca (rucaparib) Clovis Oncology Inc. 04/6/2016 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
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
Opdivo (nivolumab) Bristol-Myers Squibb Company 04/25/2016 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
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
Ilaris (canakinumab) Novartis Pharmaceuticals Corp. 04/27/2016 Patients with Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/27/2016
Indication: Patients with Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS)
Category: Rare Inherited Disorders
Ilaris (canakinumab) Novartis Pharmaceuticals Corp. 04/27/2016 Patients with Hyperimmunoglobulin D Syndrome (HIDS) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/27/2016
Indication: Patients with Hyperimmunoglobulin D Syndrome (HIDS)
Category: Rare Inherited Disorders
Ilaris (canakinumab) Novartis Pharmaceuticals Corp. 04/27/2016 Patients with Familial Mediterranean Fever (FMF) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/27/2016
Indication: Patients with Familial Mediterranean Fever (FMF)
Category: Rare Inherited Disorders
NI-0501 Novimmune 03/16/2016 Primary Hemophagocytic Lymphohistiocytosis (HLH) Rare Inherited Disorders
Date of BT Designation Disclosure: 03/16/2016
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
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
Indication: Newly-diagnosed FLT3-mutated AML
Category: Cancer
Durvalumab (MEDI4736) AstraZeneca 02/17/2016 PD-L1+ inoperable/metastatic urothelial bladder cancer Cancer
Date of BT Designation Disclosure: 02/17/2016
Indication: PD-L1+ inoperable/metastatic urothelial bladder cancer
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
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
Indication: Triple-Negative Breast Cancer (TNBC)
Category: Cancer
Ocrevus (ocrelizumab) Genentech Inc. 02/17/2016 Patients with relapsing or primary progressive forms of multiple sclerosis Rare Inherited Disorders
Date of BT Designation Disclosure: 02/17/2016
Indication: Patients with relapsing or primary progressive forms of multiple sclerosis
Category: Rare Inherited Disorders
Venetoclax AbbVie/Roche 01/20/2016 CLL combination therapy Cancer
Date of BT Designation Disclosure: 01/20/2016
Indication: CLL combination therapy
Category: Cancer
VTS 270 Vtesse 01/6/2016 Niemann-Pick Type C1 Disease (NPC) Rare Inherited Disorders
Date of BT Designation Disclosure: 01/6/2016
Indication: Niemann-Pick Type C1 Disease (NPC)
Category: Rare Inherited Disorders
Rapastinel (GLYX-13) Allergan 01/31/2016 Major Depressive Disorder (MDD) Other
Date of BT Designation Disclosure: 01/31/2016
Indication: Major Depressive Disorder (MDD)
Category: Other
Venetoclax AbbVie/Roche 01/28/2016 AML combination therapy Cancer
Date of BT Designation Disclosure: 01/28/2016
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
Indication: Metastatic Castration Resistant Prostate Cancer
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
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
Zejula (niraparib) Tesaro Inc. /2016 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: /2016
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
BI 1482694 Boehringer Ingelheim 12/18/2015 Advanced/pre-treated EGFR mutation-positive NSCLC Cancer
Date of BT Designation Disclosure: 12/18/2015
Indication: Advanced/pre-treated EGFR mutation-positive NSCLC
Category: Cancer
Yescarta (axicabtagene ciloleucel) Kite Pharma Inc. 12/7/2015 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
Indication: Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy
Category: Cancer
Pembrolizumab (Keytruda) Merck 11/2/2015 Microsatellite Instability-High (MSI-H) metastatic colorectal cancer (mCRC) Cancer
Date of BT Designation Disclosure: 11/2/2015
Indication: Microsatellite Instability-High (MSI-H) metastatic colorectal cancer (mCRC)
Category: Cancer
Bavencio (avelumab) EMD Serono Inc. 11/18/2015 Adult and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC) Cancer
Date of BT Designation Disclosure: 11/18/2015
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 Patients with tardive dyskinesia Other
Date of BT Designation Disclosure: 11/9/2015
Indication: Patients with tardive dyskinesia
Category: Other
Pexidartinib (formerly PLX3397) Daiichi Sankyo 10/30/2015 Tenosynovial Giant Cell Tumor (TGCT) Cancer
Date of BT Designation Disclosure: 10/30/2015
Indication: Tenosynovial Giant Cell Tumor (TGCT)
Category: Cancer
Inotuzumab Ozogamicin Pfizer 10/19/2015 ALL Cancer
Date of BT Designation Disclosure: 10/19/2015
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
Indication: Recurrent Clostridium difficile (C diff) infection
Category: Infectious Disease
Verzenio (abemaciclib) Eli Lilly and Co. 10/8/2015 Adult 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
Indication: Adult patients with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting
Category: Cancer
Opdivo (nivolumab) Bristol-Myers Squibb Company 09/16/2015 Patients with advanced renal cell carcinoma who have received prior antiangiogenic therapy Cancer
Date of BT Designation Disclosure: 09/16/2015
Indication: Patients with advanced renal cell carcinoma who have received prior antiangiogenic therapy
Category: Cancer
Hemlibra (emicizumab-kxwh) Genentech Inc. 09/4/2015 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
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 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
Indication: Patients with metastatic non-squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy
Category: Cancer
Cabotetyx (cabozantinib) Exelixis Inc. 08/24/2015 Patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy Cancer
Date of BT Designation Disclosure: 08/24/2015
Indication: Patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy
Category: Cancer
Ultratrace iobenguane I-131 (Azedra) Progenics 07/28/2015 Pheochromocytoma and paraganglioma Cancer
Date of BT Designation Disclosure: 07/28/2015
Indication: Pheochromocytoma and paraganglioma
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
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
Indication: Hereditary angioedema (HAE)
Category: Rare Inherited Disorders
Lenvima (lenvatinib) Eisai Inc. 07/29/2015 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
Indication: Patients with renal cell carcinoma (RCC): in combination with evorlimus, for patients with advanced RCC following one prior anti-angiogenic therapy
Category: Cancer
Tafinlar (dabrafenib) Novartis Pharmaceuticals Corp. 07/24/2015 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
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test
Category: Cancer
AR101 Aimmune 06/18/2015 Peanut allergy in children and adolescents Other
Date of BT Designation Disclosure: 06/18/2015
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
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
Indication: Systemic scerlosis
Category: Other
Olipudase alfa Sanofi/Genzyme 06/4/2015 Niemann-Pick disease Type B Rare Inherited Disorders
Date of BT Designation Disclosure: 06/4/2015
Indication: Niemann-Pick disease Type B
Category: Rare Inherited Disorders
Lartruvo (olaratumab) Eli Lilly and Co. 06/8/2015 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
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
Rapamune (sirolimus) PF Prism CV 05/28/2015 Patients with lymphangioleiomyomatosis (LAM) Other
Date of BT Designation Disclosure: 05/28/2015
Indication: Patients with lymphangioleiomyomatosis (LAM)
Category: Other
Venclexta (venetoclax) AbbVie Inc. 05/6/2015 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
Indication: Patients with chronic lymphocytic leukemia (CLL) with 17p deletion who have received at least one prior therapy
Category: Cancer
Viaskin Peanut DBV 04/9/2015 Peanut allergy in children Other
Date of BT Designation Disclosure: 04/9/2015
Indication: Peanut allergy in children
Category: Other
Rucaparib Clovis 04/6/2015 Ovarian cancer Cancer
Date of BT Designation Disclosure: 04/6/2015
Indication: Ovarian cancer
Category: Cancer
Xalkori (crizotinib) PF Prism CV 04/21/2015 Patients with non-small cell lung cancer whose tumors are ROS-1 positive Cancer
Date of BT Designation Disclosure: 04/21/2015
Indication: Patients with non-small cell lung cancer whose tumors are ROS-1 positive
Category: Cancer
Zepatier (grazoprevir/elbasvir) Merck Sharp & Dohme Corp. 04/8/2015 Adult patients with chronic hepatitis C virus (HCV) genotypes 1 and 4 infections Infectious Disease
Date of BT Designation Disclosure: 04/8/2015
Indication: Adult patients with chronic hepatitis C virus (HCV) genotypes 1 and 4 infections
Category: Infectious Disease
EBV-CTL Atara and MSKCC 03/2/2015 EBV-associated lymphoproliferative disease Cancer
Date of BT Designation Disclosure: 03/2/2015
Indication: EBV-associated lymphoproliferative disease
Category: Cancer
Ibalizumab (TMB355) TaiMed 02/27/2015 HIV Infectious Disease
Date of BT Designation Disclosure: 02/27/2015
Indication: HIV
Category: Infectious Disease
Rindopepimut (Rintega) Celldex 02/23/2015 Glioblastoma (GBM) Cancer
Date of BT Designation Disclosure: 02/23/2015
Indication: Glioblastoma (GBM)
Category: Cancer
LentiGlobin BlueBird 02/2/2015 Beta-thalassemia Rare Inherited Disorders
Date of BT Designation Disclosure: 02/2/2015
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
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
Indication: Nonalcoholic steatohepatitis (NASH)
Category: Other
Epclusa (sofosbuvir and velpatasvir) Gilead Sciences Inc. /2015 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: /2015
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
Ixazomib Takeda 12/2/2014 Systemic light-chain (AL) amyloidosis Other
Date of BT Designation Disclosure: 12/2/2014
Indication: Systemic light-chain (AL) amyloidosis
Category: Other
Lucentis (ranibizumab) Genentech Inc. 12/15/2014 Patients with Diabetic Retinopathy (DR) in patients with Diabetic Macula Edema (DME) Other
Date of BT Designation Disclosure: 12/15/2014
Indication: Patients with Diabetic Retinopathy (DR) in patients with Diabetic Macula Edema (DME)
Category: Other
JCAR015 Juno Therapeutics 11/24/2014 ALL Cancer
Date of BT Designation Disclosure: 11/24/2014
Indication: ALL
Category: Cancer
SPK-RPE65 Spark Therapeutics 11/6/2014 Inherited retinal dystrophy (IRD) Rare Inherited Disorders
Date of BT Designation Disclosure: 11/6/2014
Indication: Inherited retinal dystrophy (IRD)
Category: Rare Inherited Disorders
DUPIXENT (dupilumab) Regeneron Pharmaceuticals Inc. 11/20/2014 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
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
AP26113 Ariad 10/2/2014 ALK-positive NSCLC Cancer
Date of BT Designation Disclosure: 10/2/2014
Indication: ALK-positive NSCLC
Category: Cancer
Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. 10/27/2014 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
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
Ingrezza (valbenazine) Neurocrine Biosciences Inc. 10/30/2014 Patients with tardive dyskinesia Other
Date of BT Designation Disclosure: 10/30/2014
Indication: Patients with tardive dyskinesia
Category: Other
Opdivo (nivolumab) Bristol-Myers Squibb Company 09/26/2014 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
Indication: Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600+, a BRAF inhibitor
Category: Cancer
Eylea (aflibercept) Regeneron Pharmaceuticals Inc. 09/16/2014 Patients with diabetic retinopathy (DR) in patients with diabetic macular edema (DME) Other
Date of BT Designation Disclosure: 09/16/2014
Indication: Patients with diabetic retinopathy (DR) in patients with diabetic macular edema (DME)
Category: Other
Nuplazid (pimavanserin) Acadia Pharmaceuticals Inc. 09/2/2014 Patients with hallucinations and delusions associated with Parkinson's disease psychosis Other
Date of BT Designation Disclosure: 09/2/2014
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
Indication: Pancreatic cancer
Category: Cancer
Esbriet (pirfenidone) Genentech Inc. 07/17/2014 Patients with idiopathic pulmonary fibrosis (IPF) Other
Date of BT Designation Disclosure: 07/17/2014
Indication: Patients with idiopathic pulmonary fibrosis (IPF)
Category: Other
Ofev (nintedanib) Boehringer Ingelheim Pharmaceuticals Inc. 07/16/2014 Patients with idiopathic pulmonary fibrosis (IPF) Other
Date of BT Designation Disclosure: 07/16/2014
Indication: Patients with idiopathic pulmonary fibrosis (IPF)
Category: Other
Kymriah (tisagenlecleucel) Novartis Pharmaceuticals Corp. 07/7/2014 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
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 (blintumomab) Amgen Inc. 07/1/2014 Patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia Cancer
Date of BT Designation Disclosure: 07/1/2014
Indication: Patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia
Category: Cancer
Arikayce Insmed 06/17/2014 Infectious Disease
Date of BT Designation Disclosure: 06/17/2014
Indication:
Category: Infectious Disease
Praxbind (idarucizumab) Boehringer Ingelheim Pharmaceuticals Inc. 06/26/2014 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
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
Technivie (ombitasvir, paritaprevir, ritonavir) AbbVie Inc. 06/30/2014 Patients with genotype 4 chronic hepatitis C viruse (HCV) infection without cirrhosis Infectious Disease
Date of BT Designation Disclosure: 06/30/2014
Indication: Patients with genotype 4 chronic hepatitis C viruse (HCV) infection without cirrhosis
Category: Infectious Disease
Rociletinib (CO-1686) Clovis Oncology 05/19/2014 NSCLC Cancer
Date of BT Designation Disclosure: 05/19/2014
Indication: NSCLC
Category: Cancer
Tecentriq (atezolizumab) Genentech Inc. 05/31/2014 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
Indication: Patients with metastatic non-small cell lung cancer who have disease progression during or following platinum-containing chemotherapy
Category: Cancer
Empliciti (elotuzumab) Bristol-Myers Squibb Company 05/19/2014 Patients with multiple myeloma who have received one to three prior therapies Cancer
Date of BT Designation Disclosure: 05/19/2014
Indication: Patients with multiple myeloma who have received one to three prior therapies
Category: Cancer
Opdivo (nivolumab) Bristol-Myers Squibb Company 05/14/2014 Patients with Hodgkin Lymphoma Cancer
Date of BT Designation Disclosure: 05/14/2014
Indication: Patients with Hodgkin Lymphoma
Category: Cancer
MYDICAR Celladon  04/10/2014 Heart failure Cardiovascular
Date of BT Designation Disclosure: 04/10/2014
Indication: Heart failure
Category: Cardiovascular
Xuriden (uridine triacetate) Wellstat Therapeutics Corp. 04/30/2014 Patients with hereditary orotic aciduria Rare Inherited Disorders
Date of BT Designation Disclosure: 04/30/2014
Indication: Patients with hereditary orotic aciduria
Category: Rare Inherited Disorders
Tagrisso (osimertinib)  AstraZeneca Pharmaceuticals LP 04/24/2014 Patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive-non-small-cell lung cancer (NSCLC), as detected by an FDA approved test, who have progressed on or after EGFR TKI therapy Cancer
Date of BT Designation Disclosure: 04/24/2014
Indication: Patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive-non-small-cell lung cancer (NSCLC), as detected by an FDA approved test, who have progressed on or after EGFR TKI therapy
Category: Cancer
Bexsero (Meningococcal Group B Vaccine) Novartis Vaccines and Diagnostics, Inc. 04/7/2014 Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 2 through 25 years of age Infectious Disease
Date of BT Designation Disclosure: 04/7/2014
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 2 through 25 years of age
Category: Infectious Disease
Trumenba (Meningococcal Group B Vaccine) Wyeth Pharmaceuticals Inc. 03/20/2014 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
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
Indication: Hepatitis C
Category: Infectious Disease
Promacta (eltrombopag) Novartis Pharmaceuticals Corp. 02/3/2014 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
Indication: Cytopenias in patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy 
Category: Other
Esketamine J&J 01/21/2014 Treatment resistant depression Other
Date of BT Designation Disclosure: 01/21/2014
Indication: Treatment resistant depression
Category: Other
Dabrafenib GlaxoSmithKline 01/13/2014 NSCLC w/ BRAF mutation Cancer
Date of BT Designation Disclosure: 01/13/2014
Indication: NSCLC w/ BRAF mutation
Category: Cancer
Orkambi (lumacaftor and ivacaftor) Vertex Pharmaceuticals Inc. 01/29/2014 Patients 6 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
Indication: Patients 6 years and older with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene
Category: Rare Inherited Disorders
Tafenoquine GlaxoSmithKline 12/20/2013 Plasmodium vivax malaria Infectious Disease
Date of BT Designation Disclosure: 12/20/2013
Indication: Plasmodium vivax malaria
Category: Infectious Disease
Andexanet alfa (PRT4445) Portola 11/25/2013 Factor Xa inhibitor antidote Cardiovascular
Date of BT Designation Disclosure: 11/25/2013
Indication: Factor Xa inhibitor antidote
Category: Cardiovascular
Zydelig (idelalisib) Gilead Sciences Inc. 11/18/2013 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
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
cPMP (ALXN1011) Alexion 10/24/2013 Molybdenum cofactor deficiency (MoCD) type A Rare Inherited Disorders
Date of BT Designation Disclosure: 10/24/2013
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
Indication: Hepatitis C
Category: Infectious Disease
Sovaldi (sofosbuvir) Gilead Sciences Inc. 10/25/2013 Patients with chronic hepatitis C infection Infectious Disease
Date of BT Designation Disclosure: 10/25/2013
Indication: Patients with chronic hepatitis C infection
Category: Infectious Disease
Volasertib Boehringer Ingelheim 09/17/2013 AML combination therapy Cancer
Date of BT Designation Disclosure: 09/17/2013
Indication: AML combination therapy
Category: Cancer
Entinostat Syndax 09/11/2013 ER-positive metastatic breast cancer combination Cancer
Date of BT Designation Disclosure: 09/11/2013
Indication: ER-positive metastatic breast cancer combination
Category: Cancer
Arzerra (ofatumumab) Novartis Pharmaceuticals Corp. 09/13/2013 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
Indication: Previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate
Category: Cancer
Alecensa (alectinib) Hoffman-La Roche Inc. 09/23/2013 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
Indication: Patients with anaplastic lymphoma kinase (ALK)-positive, metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test
Category: Cancer
Amifampridine phosphate (Firdapse) Catalyst 08/27/2013 Lambert–Eaton myasthenic syndrome Other
Date of BT Designation Disclosure: 08/27/2013
Indication: Lambert–Eaton myasthenic syndrome
Category: Other
BYM338 (Bimagrumab) Novartis 08/20/2013 Sporadic inclusion body myositis (sIBM) Other
Date of BT Designation Disclosure: 08/20/2013
Indication: Sporadic inclusion body myositis (sIBM)
Category: Other
Harvoni (sofosbuvir/ledipasvir) Gilead Sciences Inc. 07/25/2013 Patients with chronic hepatitis C, genotype 1 infection Infectious Disease
Date of BT Designation Disclosure: 07/25/2013
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
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
Indication: Acute heart failure
Category: Cardiovascular
Gazyva (obinutuzumab) Genentech Inc. 05/15/2013 Patients with previously untreated chronic lymphocytic leukemia in combination with chlorambucil Cancer
Date of BT Designation Disclosure: 05/15/2013
Indication: Patients with previously untreated chronic lymphocytic leukemia in combination with chlorambucil
Category: Cancer
Viekira Pak (ombitasvir, paritaprevir, ritonavir) AbbVie Inc. 05/6/2013 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
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 Patients with multiple myeloma who have received at least on prior therapy, in combination with lenalidomide and dexamethasone or with bortezomib and dexamethasone; patients with multiple myeloma who have received at least 3 prior lines of therapy Cancer
Date of BT Designation Disclosure: 05/1/2013
Indication: Patients with multiple myeloma who have received at least on prior therapy, in combination with lenalidomide and dexamethasone or with bortezomib and dexamethasone; patients with multiple myeloma who have received at least 3 prior lines of therapy
Category: Cancer
Strensiq (asfotase alfa) Alexion Pharmaceuticals Inc. 05/28/2013 Patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP) Rare Inherited Disorders
Date of BT Designation Disclosure: 05/28/2013
Indication: Patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP)
Category: Rare Inherited Disorders
Kanuma (sebelipase alfa) Synegava Biopharma Corp. 05/20/2013 Patients with a diagnosis of lysosomal acid lipase (LAL) deficiency Rare Inherited Disorders
Date of BT Designation Disclosure: 05/20/2013
Indication: Patients with a diagnosis of lysosomal acid lipase (LAL) deficiency
Category: Rare Inherited Disorders
SD101 Scioderm 04/29/2013 Epidermolysis bullosa Other
Date of BT Designation Disclosure: 04/29/2013
Indication: Epidermolysis bullosa
Category: Other
Daclatasvir BMS 04/25/2013 Hepatitis C combination therapy Infectious Disease
Date of BT Designation Disclosure: 04/25/2013
Indication: Hepatitis C combination therapy
Category: Infectious Disease
Keytruda (prembrolizumab) Merck Sharp & Dohme Corp. 04/24/2013 Patients with unresectable or metastatic melanoma Cancer
Date of BT Designation Disclosure: 04/24/2013
Indication: Patients with unresectable or metastatic melanoma
Category: Cancer
Ibrance (palbociclib) Pfizer Inc. 04/10/2013 Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer; postmenopausal women with estrogen receptor (ER)-positive, (HER2)-negative metastatic breast cancer Cancer
Date of BT Designation Disclosure: 04/10/2013
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer; postmenopausal women with estrogen receptor (ER)-positive, (HER2)-negative metastatic breast cancer
Category: Cancer
Imbruvica (ibrutinib) Pharmacyclics LLC 04/8/2013 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
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 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: 03/15/2013
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
Imbruvica (ibrutinib) Pharmacyclics LLC 02/12/2013 Patients with mantle cell lymphoma (MCL) Cancer
Date of BT Designation Disclosure: 02/12/2013
Indication: Patients with mantle cell lymphoma (MCL)
Category: Cancer
Imbruvica (ibrutinib) Pharmacyclics LLC 02/12/2013 Patients with Waldenstrom's macroglobulinemia (WM) Cancer
Date of BT Designation Disclosure: 02/12/2013
Indication: Patients with Waldenstrom's macroglobulinemia (WM)
Category: Cancer
Ivacaftor (Kalydeco) Vertex 12/15/2012 CF combination therapy Rare Inherited Disorders
Date of BT Designation Disclosure: 12/15/2012
Indication: CF combination therapy
Category: Rare Inherited Disorders
Kalydeco (ivacaftor) Vertex Pharmaceuticals Inc. 12/15/2012 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; patients age 6 and older with cystic fibrosis and R117H or CTFR mutations Rare Inherited Disorders
Date of BT Designation Disclosure: 12/15/2012
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; patients age 6 and older with cystic fibrosis and R117H or CTFR mutations
Category: Rare Inherited Disorders
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