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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 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.

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Trade Name (Agent)

Sponsor

Date of BT Designation Disclosure

Approval Date

Indication

Category

Trade Name (Agent): Venetoclax

Sponsor: AbbVie Inc., AbbVie/Roche

Date of BT Designation Disclosure: 1/28/2016

Indication: AML combination therapy

Category: Cancer

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