Trade Name (Agent): Venetoclax
Sponsor: AbbVie Inc., AbbVie/Roche
Date of BT Designation Disclosure: 1/28/2016
Indication: AML combination therapy
Category: Cancer
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 August 31, 2021, the FDA has approved 251* breakthrough therapy designated products and lists that there have been 1182 total requests for the designation with 458** designations granted.
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): Rapastinel (GLYX-13)
Sponsor: Allergan
Date of BT Designation Disclosure: 1/31/2016
Indication: Major Depressive Disorder (MDD)
Category: Other
Trade Name (Agent): Affinity enhanced T-cell therapy
Sponsor: Adaptimmune
Date of BT Designation Disclosure: 2/09/2016
Indication: NY-ESO in synovial sarcoma
Category: Cancer
Trade Name (Agent): Durvalumab (MEDI4736)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 2/17/2016
Indication: PD-L1+ inoperable/metastatic urothelial bladder cancer
Category: Cancer
Trade Name (Agent): PKC412 (midostaurin)
Sponsor: Novartis
Date of BT Designation Disclosure: 2/19/2016
Indication: Newly-diagnosed FLT3-mutated AML
Category: Cancer
Trade Name (Agent): NI-0501
Sponsor: Novimmune
Date of BT Designation Disclosure: 3/16/2016
Indication: Primary Hemophagocytic Lymphohistiocytosis (HLH)
Category: Rare Inherited Disorders
Trade Name (Agent): SER-109
Sponsor: Seres
Date of BT Designation Disclosure: 6/12/2015
Indication: Clostridium difficile infection (CDI)
Category: Infectious Disease
Trade Name (Agent): ACTEMRA (tocilizumab)
Sponsor: Genentech/Roche
Date of BT Designation Disclosure: 6/10/2015
Approval Date: 3/04/2021
Indication: Systemic scerlosis
Category: Other
Trade Name (Agent): MYDICAR
Sponsor: Celladon
Date of BT Designation Disclosure: 4/10/2014
Indication: Heart failure
Category: Cardiovascular
Trade Name (Agent): Rociletinib (CO-1686)
Sponsor: Clovis Oncology
Date of BT Designation Disclosure: 5/19/2014
Indication: NSCLC
Category: Cancer
Trade Name (Agent): CRS-207 and GVAX
Sponsor: Aduro
Date of BT Designation Disclosure: 7/21/2014
Indication: Pancreatic cancer
Category: Cancer
Trade Name (Agent): AP26113
Sponsor: Ariad Pharmaceuticals Inc.
Date of BT Designation Disclosure: 10/02/2014
Indication: ALK-positive NSCLC
Category: Cancer
Trade Name (Agent): SPK-RPE65
Sponsor: Spark Therapeutics
Date of BT Designation Disclosure: 11/06/2014
Indication: Inherited retinal dystrophy (IRD)
Category: Rare Inherited Disorders
Trade Name (Agent): JCAR015
Sponsor: Juno Therapeutics
Date of BT Designation Disclosure: 11/24/2014
Indication: ALL
Category: Cancer
Trade Name (Agent): Ixazomib
Sponsor: Takeda
Date of BT Designation Disclosure: 12/02/2014
Indication: Systemic light-chain (AL) amyloidosis
Category: Other
Trade Name (Agent): Obeticholic acid (OCA)
Sponsor: Intercept
Date of BT Designation Disclosure: 1/29/2015
Indication: Nonalcoholic steatohepatitis (NASH)
Category: Other
Trade Name (Agent): Olipudase alfa
Sponsor: Sanofi/Genzyme
Date of BT Designation Disclosure: 6/04/2015
Indication: Niemann-Pick disease Type B
Category: Rare Inherited Disorders
Trade Name (Agent): Viaskin Peanut
Sponsor: DBV
Date of BT Designation Disclosure: 4/09/2015
Indication: Peanut allergy in children
Category: Other
Trade Name (Agent): Rucaparib
Sponsor: Clovis
Date of BT Designation Disclosure: 4/06/2015
Indication: Ovarian cancer
Category: Cancer
Trade Name (Agent): EBV-CTL
Sponsor: Atara and MSKCC
Date of BT Designation Disclosure: 3/02/2015
Indication: EBV-associated lymphoproliferative disease
Category: Cancer
Trade Name (Agent): Rindopepimut (Rintega)
Sponsor: Celldex
Date of BT Designation Disclosure: 2/23/2015
Indication: Glioblastoma (GBM)
Category: Cancer
Trade Name (Agent): MPDL3280A
Sponsor: Genentech/Roche
Date of BT Designation Disclosure: 2/01/2015
Indication: Non-small cell lung cancer
Category: Cancer
Trade Name (Agent): Ivacaftor (Kalydeco)
Sponsor: Vertex
Date of BT Designation Disclosure: 12/15/2012
Indication: CF combination therapy
Category: Rare Inherited Disorders
Trade Name (Agent): PVS-RIPO
Sponsor: Duke Medicine
Date of BT Designation Disclosure: 5/15/2016
Indication: Recurrent glioblastoma multiforme (GBM)
Category: Cancer
Trade Name (Agent): SHP621 (budesonide oral suspension, or BOS)
Sponsor: Shire
Date of BT Designation Disclosure: 6/13/2016
Indication: Eosinophilic esophagitis (EoE)
Category: Rare Inherited Disorders
Trade Name (Agent): Ivacaftor (Kalydeco)
Sponsor: Vertex
Date of BT Designation Disclosure: 2014
Approval Date: 3/17/2015
Indication: CF monotherapy, patients 2 years and older with one of the following CFTR gene mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H
Category: Rare Inherited Disorders
Trade Name (Agent): Ivacaftor (Kalydeco)
Sponsor: Vertex
Date of BT Designation Disclosure: 2014
Approval Date: 12/29/2014
Indication: CF monotherapy, patients 6 years or older with R117H mutationin CFTR gene
Category: Rare Inherited Disorders
Trade Name (Agent): SPK-9001
Sponsor: Pfizer/Spark
Date of BT Designation Disclosure: 7/21/2016
Indication: Hemophilia B.
Category: Rare Inherited Disorders
Trade Name (Agent): V920
Sponsor: Merck
Date of BT Designation Disclosure: 7/25/2016
Indication: Ebola Zaire
Category: Infectious Disease
Trade Name (Agent): Pracinostat
Sponsor: MEI Pharma
Date of BT Designation Disclosure: 8/01/2016
Indication: In combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy
Category: Cancer
Trade Name (Agent): Esketamine
Sponsor: Janssen
Date of BT Designation Disclosure: 8/16/2016
Approval Date: 7/31/2020
Indication: Major depressive disorder with imminent risk for suicide
Category: Other
Trade Name (Agent): SL-401
Sponsor: Stemline Therapeutics
Date of BT Designation Disclosure: 8/23/2016
Indication: Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123)
Category: Rare Inherited Disorders
Trade Name (Agent): Daclatasvir
Sponsor: BMS
Date of BT Designation Disclosure: 4/25/2013
Indication: Hepatitis C combination therapy
Category: Infectious Disease
Trade Name (Agent): SD101
Sponsor: Scioderm
Date of BT Designation Disclosure: 4/29/2013
Indication: Epidermolysis bullosa
Category: Other
Trade Name (Agent): LIVMARLI (maralixibat)
Sponsor: Shire
Date of BT Designation Disclosure: 6/13/2016
Approval Date: 9/29/2021
Indication: Progressive familial intrahepatic cholestasis type 2 (PFIC2)
Category: Rare Inherited Disorders
Trade Name (Agent): Daclatasvir/asunaprevir combination
Sponsor: BMS
Date of BT Designation Disclosure: 2/24/2014
Indication: Hepatitis C
Category: Infectious Disease
Trade Name (Agent): Esketamine
Sponsor: J&J
Date of BT Designation Disclosure: 1/21/2014
Indication: Treatment resistant depression
Category: Other
Trade Name (Agent): Dabrafenib
Sponsor: GlaxoSmithKline
Date of BT Designation Disclosure: 1/13/2014
Indication: NSCLC w/ BRAF mutation
Category: Cancer
Trade Name (Agent): cPMP (ALXN1011)
Sponsor: Alexion Pharmaceuticals Inc.
Date of BT Designation Disclosure: 10/24/2013
Indication: Molybdenum cofactor deficiency (MoCD) type A
Category: Rare Inherited Disorders
Trade Name (Agent): Grazoprevir/Elbasvir
Sponsor: Merck
Date of BT Designation Disclosure: 10/22/2013
Indication: Hepatitis C
Category: Infectious Disease
Trade Name (Agent): Volasertib
Sponsor: Boehringer Ingelheim
Date of BT Designation Disclosure: 9/17/2013
Indication: AML combination therapy
Category: Cancer
Trade Name (Agent): Entinostat
Sponsor: Syndax
Date of BT Designation Disclosure: 9/11/2013
Indication: ER-positive metastatic breast cancer combination
Category: Cancer
Trade Name (Agent): BYM338 (Bimagrumab)
Sponsor: Novartis
Date of BT Designation Disclosure: 8/20/2013
Indication: Sporadic inclusion body myositis (sIBM)
Category: Other
Trade Name (Agent): Drisapersen
Sponsor: GlaxoSmithKline/Prosensa
Date of BT Designation Disclosure: 6/27/2013
Indication: Duchenne muscular dystrophy (DMD)
Category: Rare Inherited Disorders
Trade Name (Agent): Serelaxin
Sponsor: Novartis
Date of BT Designation Disclosure: 6/21/2013
Indication: Acute heart failure
Category: Cardiovascular
Trade Name (Agent): Alecensa (alectinib)
Sponsor: Roche
Date of BT Designation Disclosure: 10/04/2016
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
Trade Name (Agent): Elbit Imaging
Sponsor: Gamida Cell
Date of BT Designation Disclosure: 10/10/2016
Indication: Nicord
Category: Cancer
Trade Name (Agent): Acalabrutinib
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/01/2017
Indication: Mantle cell lymphoma for patients who have received at least one prior line of therapy
Category: Cancer
Trade Name (Agent): Venetoclax
Sponsor: AbbVie Inc., AbbVie/Roche
Date of BT Designation Disclosure: 8/01/2017
Indication: Elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy
Category: Cancer
Trade Name (Agent): Methylenedioxymethamphetamine (MDMA)
Sponsor: MAPS
Date of BT Designation Disclosure: 8/28/2017
Indication: PTSD
Category: Other
Trade Name (Agent): EB-101
Sponsor: Abeona Therapeutics Inc
Date of BT Designation Disclosure: 8/30/2017
Indication: Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Category: Other
Trade Name (Agent): DAS181
Sponsor: Ansun BioPharma
Date of BT Designation Disclosure: 10/10/2017
Indication: Treatment of lower respiratory tract parainfluenza virus (PIV) infection in immunocompromised patients
Category: Infectious Disease
Trade Name (Agent): Valoctocogene Roxaparvovec
Sponsor: BioMarin
Date of BT Designation Disclosure: 10/26/2017
Indication: Hemophilia A
Category: Rare Inherited Disorders
Trade Name (Agent): Monjuvi
Sponsor: MorpoSys
Date of BT Designation Disclosure: 10/23/2017
Approval Date: 7/31/2020
Indication: Relapsed/Refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation
Category: Cancer
Trade Name (Agent): Avelumab in combination with Inlyta (axitinib)
Sponsor: EMD Serono and Pfizer
Date of BT Designation Disclosure: 12/21/2017
Indication: Treatment-naïve patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): Lenvima (lenvatinib)
Sponsor: Merck and Eisai
Date of BT Designation Disclosure: 1/09/2018
Approval Date: 8/10/2021
Indication: In combination with pembrolizumab for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
Category: Cancer
Trade Name (Agent): Upadacitinib (ABT-494)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 1/09/2018
Indication: Adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy
Category: Other
Trade Name (Agent): Livtencity (Maribavir)
Sponsor: Takeda Pharmaceutical Company Ltd.
Date of BT Designation Disclosure: 1/08/2018
Approval Date: 11/23/2021
Indication: Treatment of adults and pediatric patients 12 years of age and older and weighing at least 35 kg with posttransplant cytomegalovirus (CMV) infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir or foscarnet.
Category: Infectious Disease
Trade Name (Agent): Imcivree (setmelanotide)
Sponsor: Rhythm Pharmaceutical, Inc.
Date of BT Designation Disclosure: 1/7/2016
Approval Date: 11/27/2020
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
Trade Name (Agent): Tonmya (TNX-102 SL)
Sponsor: Tonix Pharmaceuticals
Date of BT Designation Disclosure: 12/19/2016
Indication: PTSD
Category: Other
Trade Name (Agent): ABT-530
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 9/30/2016
Indication: genotype 1 HCV patients who have failed to respond to a previous therapy of direct acting antivirals (or DAAs)
Category: Infectious Disease
Trade Name (Agent): Neridronic acid
Sponsor: Grünenthal and Abiogen Pharma
Date of BT Designation Disclosure: 12/16/2016
Indication: Complex regional pain syndrome (CRPS)
Category: Rare Inherited Disorders
Trade Name (Agent): AMT-060
Sponsor: uniQure
Date of BT Designation Disclosure: 1/20/2017
Indication: Hemophilia B
Category: Rare Inherited Disorders
Trade Name (Agent): Toca 511 & Toca FC
Sponsor: Tocagen
Date of BT Designation Disclosure: 2/23/2017
Indication: Recurrent high grade glioma
Category: Cancer
Trade Name (Agent): Evkeeza (evinacumab-dgnb)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 4/07/2017
Approval Date: 2/11/2021
Indication: Homozygous familial hypercholesterolemia (HOFH)
Category: Rare Inherited Disorders
Trade Name (Agent): Rethymic (RVT-802)
Sponsor: Enzyvant
Date of BT Designation Disclosure: 4/14/2017
Approval Date: 10/8/2021
Indication: Treatment of pediatric patients with congenital athymia.
Category: Rare Inherited Disorders
Trade Name (Agent): Vonapanitase
Sponsor: Proteon Therapeutics, Inc.
Date of BT Designation Disclosure: 5/10/2017
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
Trade Name (Agent): GMI-1271
Sponsor: GlycoMimetics
Date of BT Designation Disclosure: 5/17/2017
Indication: Adult Relapsed/Refractory Acute Myeloid Leukemia
Category: Cancer
Trade Name (Agent): Balovaptan
Sponsor: Roche
Date of BT Designation Disclosure: 1/29/2018
Indication: Autism spectrum disorder (ASD)
Category: Rare Inherited Disorders
Trade Name (Agent): Burtomab
Sponsor: Y-mAbs Therapeutics, Inc.
Date of BT Designation Disclosure: 6/07/2017
Indication: Pediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis
Category: Cancer
Trade Name (Agent): OMS721
Sponsor: Omeros
Date of BT Designation Disclosure: 6/13/2017
Indication: Immunoglobulin A (IgA) nephropathy
Category: Other
Trade Name (Agent): PTI-428
Sponsor:
Date of BT Designation Disclosure: 3/16/2018
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
Trade Name (Agent): tab-cel
Sponsor:
Date of BT Designation Disclosure: 2/15/2015
Indication: Patients with EBV+ PTLD following allogeneic hematopoietic stem cell transplant
Category: Other
Trade Name (Agent): bb2121
Sponsor:
Date of BT Designation Disclosure: 11/04/2017
Indication: Patients with relapsed or refractory multiple myeloma
Category: Cancer
Trade Name (Agent): ALIS (amikacin liposome inhalation suspension)
Sponsor:
Date of BT Designation Disclosure: 3/29/2018
Indication: Adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex
Category: Other
Trade Name (Agent): Pedmark (sodium thiosulfate)
Sponsor:
Date of BT Designation Disclosure: 3/27/2018
Indication: Prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB)
Category: Other
Trade Name (Agent): SPK-8011
Sponsor:
Date of BT Designation Disclosure: 2/15/2018
Indication: Patients with hemophilia A.
Category: Rare Inherited Disorders
Trade Name (Agent): Ketamine
Sponsor:
Date of BT Designation Disclosure: 4/16/2018
Indication: Patients with depression
Category: Other
Trade Name (Agent): Ribaxamase (SYN-004)
Sponsor:
Date of BT Designation Disclosure: 5/11/2017
Indication: prevention of Clostridium difficile infection
Category: Infectious Disease
Trade Name (Agent): OMS721
Sponsor:
Date of BT Designation Disclosure: 4/26/2018
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
Trade Name (Agent): Trumenba (Meningococcal Group B Vaccine)
Sponsor:
Date of BT Designation Disclosure: 4/23/2018
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 1 through 9 years of age
Category:
Trade Name (Agent): Kymriah (tisagenlecleucel)
Sponsor:
Date of BT Designation Disclosure: 4/18/2017
Approval Date: 5/1/2018
Indication: Treatment of adults with relapsed/refractory diffuse large B-cell lymphoma (DLBCL).
Category: Cancer
Trade Name (Agent): Oxlumo (lumasiran)
Sponsor:
Date of BT Designation Disclosure: 3/12/2018
Approval Date: 11/23/2020
Indication: Primary Hyperoxaluria Type 1 (PH1)
Category: Infectious Disease
Trade Name (Agent): Trastuzumab Deruxtecan (DS-8201)
Sponsor:
Date of BT Designation Disclosure: 8/29/2017
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
Trade Name (Agent): Balovaptan (RG7314)
Sponsor:
Date of BT Designation Disclosure: 1/28/2018
Indication: A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD)
Category: Other
Trade Name (Agent): Kymriah (CTL019)
Sponsor:
Date of BT Designation Disclosure: 4/18/2017
Indication: Adult patients with relapsed and refractory diffuse large B-cell lymphoma, who have failed two or more prior therapies
Category: Cancer
Trade Name (Agent): Korsuva (difelikefalin)
Sponsor:
Date of BT Designation Disclosure: 6/22/2017
Approval Date: 8/23/2021
Indication: Treatment of moderate-to-severe pruritus associated with chronic kidney disease (CKD-aP) in adults undergoing hemodialysis (HD).
Category: Other
Trade Name (Agent): ZX008 (fenfluramine)
Sponsor: Zogenix, Inc.
Date of BT Designation Disclosure: 2/06/2018
Indication: Patients who have seizures associated with Dravet syndrome
Category: Other
Trade Name (Agent): SAGE-217
Sponsor:
Date of BT Designation Disclosure: 2/07/2018
Indication: Patients with major depressive disorder (MDD)
Category: Other
Trade Name (Agent): PF-04965842
Sponsor:
Date of BT Designation Disclosure: 2/14/2018
Indication: Patients with moderate-to-severe atopic dermatitis (AD)
Category: Other
Trade Name (Agent): Nuplazid (pimavanserin)
Sponsor:
Date of BT Designation Disclosure: 2017
Indication: Patients with dementia-related psychosis (DRP)
Category: Other
Trade Name (Agent): Larotrectinib
Sponsor:
Date of BT Designation Disclosure: 7/13/2016
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
Trade Name (Agent): Advantage Arrest™ Silver Diamine Fluoride 38%
Sponsor:
Date of BT Designation Disclosure: 11/03/2016
Indication: Arrest of tooth decay in children and adults
Category: Other
Trade Name (Agent): GC4419
Sponsor:
Date of BT Designation Disclosure: 2/28/2018
Indication: Reduction of the duration, severity, and incidence of severe oral mucositis induced by radiation therapy
Category: Other
Trade Name (Agent): Enjaymo (sutimlimab-jome)
Sponsor:
Date of BT Designation Disclosure: 3/06/2018
Approval Date: 2/4/2022
Indication: Indicated to decrease the need for red blood cell (RBC) transfusion due to hemolysis in adults with cold agglutinin disease (CAD).
Category: Other
Trade Name (Agent): Verzenio (abemaciclib)
Sponsor:
Date of BT Designation Disclosure: 10/08/2015
Approval Date: 2/26/2018
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
Trade Name (Agent): Quizartinib
Sponsor:
Date of BT Designation Disclosure: 8/01/2018
Indication: Adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML)
Category: Cancer
Trade Name (Agent): Pitolisant
Sponsor:
Date of BT Designation Disclosure: 5/21/2018
Indication: Excessive daytime sleepiness and cataplexy in patients with narcolepsy
Category: Other
Trade Name (Agent): Lenti-D
Sponsor:
Date of BT Designation Disclosure: 5/23/2018
Indication: Cerebral adrenoleukodystrophy
Category: Rare Inherited Disorders
Trade Name (Agent): Tafamidis
Sponsor:
Date of BT Designation Disclosure: 5/23/2018
Indication: Transthyretin Cardiomyopathy (ATTR-ACT)
Category: Cardiovascular
