Error message

Notice: Undefined index: #children in theme_fieldset() (line 2934 of /home/focrorg/public_html/includes/form.inc).

You are here

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 June 8, 2016 the FDA has approved 49 breakthrough therapy designated products and lists that there have been 415 total requests for the designation with 130 designations granted.

Filter Results
filter
Agent (Trade Name) Sponsor Date of BT Designation Disclosure Indication Category
Nivolumab (Opdivo) BMS 06/27/2016 Unresectable locally advanced or metastatic urothelial carcinoma that has progressed on or after a platinum-containing regimen Cancer
Date of BT Designation Disclosure: 06/27/2016
Indication: Unresectable locally advanced or metastatic urothelial carcinoma that has progressed on or after a platinum-containing regimen
Category: Cancer
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
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
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
VYXEOS (CPX-351) Celator Pharmaceuticals 05/20/2016 Acute myeloid leukemia (AML) Cancer
Date of BT Designation Disclosure: 05/20/2016
Indication: Acute myeloid leukemia (AML)
Category: Cancer
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
Canakinumab (Ilaris) Novartis 04/27/2016 Familial Mediterranean Fever (FMF) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/27/2016
Indication: Familial Mediterranean Fever (FMF)
Category: Rare Inherited Disorders
Canakinumab (Ilaris) Novartis 04/27/2016 Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/27/2016
Indication: Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD)
Category: Rare Inherited Disorders
Canakinumab (Ilaris) Novartis 04/27/2016 Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) Rare Inherited Disorders
Date of BT Designation Disclosure: 04/27/2016
Indication: Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS)
Category: Rare Inherited Disorders
Nivolumab (Opdivo) BMS 04/25/2016 Squamous cell carcinoma of the head and neck (SCCHN) Cancer
Date of BT Designation Disclosure: 04/25/2016
Indication: Squamous cell carcinoma of the head and neck (SCCHN)
Category: Cancer
Pembrolizumab (Keytruda) Merck 04/18/2016 Relapsed or refractory classical Hodgkin lymphoma (CHL) Cancer
Date of BT Designation Disclosure: 04/18/2016
Indication: Relapsed or refractory classical Hodgkin lymphoma (CHL)
Category: Cancer
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
Ocrelizumab (OCREVUSTM) Genentech/Roche 02/17/2016 Primary Progressive Multiple Sclerosis Rare Inherited Disorders
Date of BT Designation Disclosure: 02/17/2016
Indication: Primary Progressive 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
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
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
Venetoclax AbbVie/Roche 01/20/2016 CLL combination therapy Cancer
Date of BT Designation Disclosure: 01/20/2016
Indication: CLL combination therapy
Category: Cancer
Setmelanotide Rhythm 01/7/2016 Pro-opiomelanocortin (POMC) deficiency obesity Rare Inherited Disorders
Date of BT Designation Disclosure: 01/7/2016
Indication: Pro-opiomelanocortin (POMC) deficiency obesity
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
Indication: Niemann-Pick Type C1 Disease (NPC)
Category: Rare Inherited Disorders
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
KTE-C19 Kite 12/7/2015 Refractory, Aggressive Non Hodgkin Lymphoma (NHL) Cancer
Date of BT Designation Disclosure: 12/7/2015
Indication: Refractory, Aggressive Non Hodgkin Lymphoma (NHL)
Category: Cancer
Avelumab Merck KGaA and Pfizer 11/18/2015 Metastatic Merkel cell carcinoma (MCC) Cancer
Date of BT Designation Disclosure: 11/18/2015
Indication: Metastatic Merkel cell carcinoma (MCC)
Category: Cancer
SD-809 (deutetrabenazine) Teva 11/9/2015 Tardive Dyskinesia Other
Date of BT Designation Disclosure: 11/9/2015
Indication: 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
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
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
Abemaciclib Eli Lilly 10/8/2015 HR+ Advanced Breast Cancer Cancer
Date of BT Designation Disclosure: 10/8/2015
Indication: HR+ Advanced Breast Cancer
Category: Cancer
Nivolumab (Opdivo) BMS 09/16/2015 Advanced renal cell carcinoma Cancer
Date of BT Designation Disclosure: 09/16/2015
Indication: Advanced renal cell carcinoma
Category: Cancer
ACE910 Roche/Genentech 09/4/2015 Hemophilia Rare Inherited Disorders
Date of BT Designation Disclosure: 09/4/2015
Indication: Hemophilia
Category: Rare Inherited Disorders
Nivolumab (Opdivo) BMS 09/2/2015 Nonsquamous NSCLC Cancer
Date of BT Designation Disclosure: 09/2/2015
Indication: Nonsquamous NSCLC
Category: Cancer
Cabozantinib Exelixis 08/24/2015 Renal cell carcinoma Cancer
Date of BT Designation Disclosure: 08/24/2015
Indication: Renal cell carcinoma
Category: Cancer
Lenvatinib (Lenvima) Eisai 07/29/2015 Advanced and/or metastatic renal cell carcinoma Cancer
Date of BT Designation Disclosure: 07/29/2015
Indication: Advanced and/or metastatic renal cell carcinoma
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
Tafinlar and Mekinist combination Novartis 07/24/2015 NSCLC w/ BRAF V600 mutation Cancer
Date of BT Designation Disclosure: 07/24/2015
Indication: NSCLC w/ BRAF V600 mutation
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
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
Siroliums (Rapamune) Wyeth, Pfizer 05/28/2015 LAM Other
Date of BT Designation Disclosure: 05/28/2015
Indication: LAM
Category: Other
Venetoclax AbbVie/Roche 05/6/2015 CLL monotherapy Cancer
Date of BT Designation Disclosure: 05/6/2015
Indication: CLL monotherapy
Category: Cancer
Xalkori (crizotinib) Pfizer 04/21/2015 ROS1-positive non-small cell lung cancer (NSCLC) Cancer
Date of BT Designation Disclosure: 04/21/2015
Indication: ROS1-positive non-small cell lung cancer (NSCLC)
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
Zepatier (Grazoprevir/Elbasvir) Merck 04/8/2015 Chronic hepatitis C genotype 1 in patients with end stage renal disease on hemodialysis Infectious Disease
Date of BT Designation Disclosure: 04/8/2015
Indication: Chronic hepatitis C genotype 1 in patients with end stage renal disease on hemodialysis
Category: Infectious Disease
Zepatier (Grazoprevir/Elbasvir) Merck 04/8/2015 Chronic hepatitis C genotype 4 Infectious Disease
Date of BT Designation Disclosure: 04/8/2015
Indication: Chronic hepatitis C genotype 4
Category: Infectious Disease
Rucaparib Clovis 04/6/2015 Ovarian cancer Cancer
Date of BT Designation Disclosure: 04/6/2015
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
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
Ranibizumab (Lucentis)  Genentech/Roche 12/15/2014 Diabetic retinopathy Other
Date of BT Designation Disclosure: 12/15/2014
Indication: Diabetic retinopathy
Category: Other
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
JCAR015 Juno Therapeutics 11/24/2014 ALL Cancer
Date of BT Designation Disclosure: 11/24/2014
Indication: ALL
Category: Cancer
Dupilumab Regeneron/Sanofi 11/20/2014 Atopic Dermatitis Other
Date of BT Designation Disclosure: 11/20/2014
Indication: Atopic Dermatitis
Category: Other
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
NBI-98854 Neurocrine Biosciences 10/30/2014 Tardive dyskinesia Other
Date of BT Designation Disclosure: 10/30/2014
Indication: Tardive dyskinesia
Category: Other
Pembrolizumab (Keytruda) Merck 10/27/2014 NSCLC Cancer
Date of BT Designation Disclosure: 10/27/2014
Indication: NSCLC
Category: Cancer
AP26113 Ariad 10/2/2014 ALK-positive NSCLC Cancer
Date of BT Designation Disclosure: 10/2/2014
Indication: ALK-positive NSCLC
Category: Cancer
Nivolumab (Opdivo) BMS 09/26/2014 Melanoma Cancer
Date of BT Designation Disclosure: 09/26/2014
Indication: Melanoma
Category: Cancer
Eylea (aflibercept) Regeneron 09/16/2014 Diabetic macular edema (DME) Other
Date of BT Designation Disclosure: 09/16/2014
Indication: Diabetic macular edema (DME)
Category: Other
Nuplazid (pimavanserin) Acadia 09/2/2014 Parkinson's disease psychosis Other
Date of BT Designation Disclosure: 09/2/2014
Indication: 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
Pirfenidone (Esbriet) InterMune 07/17/2014 idiopathic pulmonary fibrosis (IPF) Other
Date of BT Designation Disclosure: 07/17/2014
Indication: idiopathic pulmonary fibrosis (IPF)
Category: Other
Nintedanib (Ofev) Boehringer Ingelheim 07/16/2014 Idiopathic pulmonary fibrosis (IPF) Other
Date of BT Designation Disclosure: 07/16/2014
Indication: Idiopathic pulmonary fibrosis (IPF)
Category: Other
CTL019 Novartis 07/7/2014 ALL Cancer
Date of BT Designation Disclosure: 07/7/2014
Indication: ALL
Category: Cancer
Blinatumomab (Blincyto) Amgen 07/1/2014 ALL Cancer
Date of BT Designation Disclosure: 07/1/2014
Indication: ALL
Category: Cancer
Idarucizumab Boehringer Ingelheim 06/26/2014 Antidote for Praxada, an anticoagulant Other
Date of BT Designation Disclosure: 06/26/2014
Indication: Antidote for Praxada, an anticoagulant
Category: Other
Arikayce Insmed 06/17/2014 Infectious Disease
Date of BT Designation Disclosure: 06/17/2014
Indication:
Category: Infectious Disease
Atezolizumab (MPDL3280A) Roche 05/31/2014 Metastatic urothelial bladder cancer Cancer
Date of BT Designation Disclosure: 05/31/2014
Indication: Metastatic urothelial bladder cancer
Category: Cancer
Elotuzumab BMS 05/19/2014 Multiple myeloma Cancer
Date of BT Designation Disclosure: 05/19/2014
Indication: Multiple myeloma
Category: Cancer
Rociletinib (CO-1686) Clovis Oncology 05/19/2014 NSCLC Cancer
Date of BT Designation Disclosure: 05/19/2014
Indication: NSCLC
Category: Cancer
Nivolumab (Opdivo) BMS 05/14/2014 Hodgkin lymphoma Cancer
Date of BT Designation Disclosure: 05/14/2014
Indication: Hodgkin lymphoma
Category: Cancer
Uridine Triacetate (Xuriden) Wellstat Therapeutics 04/30/2014 Hereditary orotic aciduria Rare Inherited Disorders
Date of BT Designation Disclosure: 04/30/2014
Indication: Hereditary orotic aciduria
Category: Rare Inherited Disorders
Tagrisso (Osimertinib)  AstraZeneca 04/24/2014 NSCLC Cancer
Date of BT Designation Disclosure: 04/24/2014
Indication: NSCLC
Category: Cancer
MYDICAR Celladon  04/10/2014 Heart failure Cardiovascular
Date of BT Designation Disclosure: 04/10/2014
Indication: Heart failure
Category: Cardiovascular
Bexsero Novartis 04/7/2014 Meningitis B vaccine Infectious Disease
Date of BT Designation Disclosure: 04/7/2014
Indication: Meningitis B vaccine
Category: Infectious Disease
Trumenba Pfizer 03/20/2014 Meningitis B vaccine Infectious Disease
Date of BT Designation Disclosure: 03/20/2014
Indication: Meningitis B vaccine
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
Eltrombopag (Promacta) GlaxoSmithKline 02/3/2014 Severe aplastic anaemia (SAA)  Other
Date of BT Designation Disclosure: 02/3/2014
Indication: Severe aplastic anaemia (SAA) 
Category: Other
Orkambi (lumacaftor)/Kalydeco combination Vertex 01/29/2014 Cystic Fibrosis w/ F508del mutation Rare Inherited Disorders
Date of BT Designation Disclosure: 01/29/2014
Indication: Cystic Fibrosis w/ F508del mutation
Category: Rare Inherited Disorders
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
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
Idelalisib (Zydelig) Gilead 11/18/2013 CLL Cancer
Date of BT Designation Disclosure: 11/18/2013
Indication: CLL
Category: Cancer
Sofosbuvir (Sovaldi) Gilead 10/25/2013 Hepatitis C Infectious Disease
Date of BT Designation Disclosure: 10/25/2013
Indication: Hepatitis C
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
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
Alecensa (Alectinib) Genentech/Roche 09/23/2013 ALK-positive NSCLC monotherapy Cancer
Date of BT Designation Disclosure: 09/23/2013
Indication: ALK-positive NSCLC monotherapy
Category: Cancer
Volasertib Boehringer Ingelheim 09/17/2013 AML combination therapy Cancer
Date of BT Designation Disclosure: 09/17/2013
Indication: AML combination therapy
Category: Cancer
Ofatumumab (Arzerra) GlaxoSmithKline/Genmab 09/13/2013 First-line CLL combination therapy Cancer
Date of BT Designation Disclosure: 09/13/2013
Indication: First-line CLL 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
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
Sofosbuvir/ledipasvir combination (Harvoni) Gilead 07/25/2013 Hepatitis C Infectious Disease
Date of BT Designation Disclosure: 07/25/2013
Indication: Hepatitis C
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
Asfotase Alfa Alexion 05/28/2013 Hypophosphatasia Rare Inherited Disorders
Date of BT Designation Disclosure: 05/28/2013
Indication: Hypophosphatasia
Category: Rare Inherited Disorders
Sebelipase Alfa (Kanuma) Alexion 05/20/2013 LAL deficiency Rare Inherited Disorders
Date of BT Designation Disclosure: 05/20/2013
Indication: LAL deficiency
Category: Rare Inherited Disorders
Obinutuzumab (Gazyva) Genentech/Roche 05/15/2013 CLL Cancer
Date of BT Designation Disclosure: 05/15/2013
Indication: CLL
Category: Cancer
ABT-450 (Viekira Pak) AbbVie 05/6/2013 Hepatitis C  Infectious Disease
Date of BT Designation Disclosure: 05/6/2013
Indication: Hepatitis C 
Category: Infectious Disease
Daratumumab  Janssen 05/1/2013 Multiple myeloma Cancer
Date of BT Designation Disclosure: 05/1/2013
Indication: Multiple myeloma
Category: Cancer
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
Pembrolizumab (Keytruda) Merck 04/24/2013 Melanoma Cancer
Date of BT Designation Disclosure: 04/24/2013
Indication: Melanoma
Category: Cancer
Palbociclib (Ibrance) Pfizer 04/10/2013 ER+, HER2- breast cancer Cancer
Date of BT Designation Disclosure: 04/10/2013
Indication: ER+, HER2- breast cancer
Category: Cancer
Ibrutinib (Imbruvica) J&J/Pharmacyclics 04/8/2013 CLL monotherapy Cancer
Date of BT Designation Disclosure: 04/8/2013
Indication: CLL monotherapy
Category: Cancer
Ceritinib (Zykadia) Novartis 03/15/2013 NSCLC Cancer
Date of BT Designation Disclosure: 03/15/2013
Indication: NSCLC
Category: Cancer
Ibrutinib (Imbruvica ) J&J/Pharmacyclics 02/12/2013 Waldenstrom's macroglobulinemia (WM) Cancer
Date of BT Designation Disclosure: 02/12/2013
Indication: Waldenstrom's macroglobulinemia (WM)
Category: Cancer
Ibrutinib (Imbruvica ) J&J/Pharmacyclics 02/12/2013 Mantle cell lymphoma Cancer
Date of BT Designation Disclosure: 02/12/2013
Indication: Mantle cell lymphoma
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
Ivacaftor (Kalydeco) Vertex 12/15/2012 CF monotherapy Rare Inherited Disorders
Date of BT Designation Disclosure: 12/15/2012
Indication: CF monotherapy
Category: Rare Inherited Disorders
XLSX