Trade Name (Agent): Lenti-D
Sponsor:
Date of BT Designation Disclosure: 5/23/2018
Indication: Cerebral adrenoleukodystrophy
Category: Rare Inherited Disorders
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 March 31, 2023, the FDA has approved 278* breakthrough therapy designated products and lists that there have been 1342 total requests for the designation with 519** designations granted.
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
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): 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): Trumenba (Meningococcal Group B Vaccine)
Sponsor:
Date of BT Designation Disclosure: 3/20/2014
Approval Date: 10/29/2014
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age
Category: Infectious Disease
Trade Name (Agent): Bexsero (Meningococcal Group B Vaccine)
Sponsor:
Date of BT Designation Disclosure: 4/07/2014
Approval Date: 1/23/2015
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age
Category: Infectious Disease
Trade Name (Agent): Kalydeco (ivacaftor)
Sponsor: Vertex, Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 2013
Approval Date: 2/21/2014
Indication: Patients age 6 years and older with cystic fibrosis who have mutations in the CTFR gene
Category: Rare Inherited Disorders
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 4/24/2013
Approval Date: 12/18/2015
Indication: Patients with unresectable or metastatic melanoma
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 9/02/2015
Approval Date: 10/09/2015
Indication: Patients with metastatic non-squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 9/26/2014
Approval Date: 12/22/2014
Indication: Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600+, a BRAF inhibitor
Category: Cancer
Trade Name (Agent): Kymriah (tisagenlecleucel)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 7/07/2014
Approval Date: 8/30/2017
Indication: Pediatric and young adult patients (age 3-25 years) with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse
Category: Cancer
Trade Name (Agent): Yescarta (axicabtagene ciloleucel)
Sponsor: Kite Pharma Inc.
Date of BT Designation Disclosure: 12/07/2015
Approval Date: 10/18/2017
Indication: Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy
Category: Cancer
Trade Name (Agent): Rapamune (sirolimus)
Sponsor:
Date of BT Designation Disclosure: 2015
Approval Date: 5/28/2015
Indication: Patients with lymphangioleiomyomatosis (LAM)
Category: Other
Trade Name (Agent): Xalkori (crizotinib)
Sponsor:
Date of BT Designation Disclosure: 4/21/2015
Approval Date: 3/11/2016
Indication: Patients with non-small cell lung cancer whose tumors are ROS-1 positive
Category: Cancer
Trade Name (Agent): Zykadia (ceritinib)
Sponsor:
Date of BT Designation Disclosure: 3/15/2013
Approval Date: 4/29/2014
Indication: Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib
Category: Cancer
Trade Name (Agent): Imbruvica (ibrutinib)
Sponsor:
Date of BT Designation Disclosure: 2/12/2013
Approval Date: 1/29/2015
Indication: Patients with Waldenstrom's macroglobulinemia (WM)
Category: Cancer
Trade Name (Agent): Imbruvica (ibrutinib)
Sponsor:
Date of BT Designation Disclosure: 2/12/2013
Approval Date: 11/13/2013
Indication: Patients with mantle cell lymphoma (MCL)
Category: Cancer
Trade Name (Agent): Ibrance (palbociclib)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 2016
Approval Date: 2/19/2016
Indication: Patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer in combination with fulvestrant in women with disease progression following endocrine therapy
Category: Cancer
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): 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): 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): 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): Ribaxamase (SYN-004)
Sponsor:
Date of BT Designation Disclosure: 5/11/2017
Indication: prevention of Clostridium difficile infection
Category: Infectious Disease
Trade Name (Agent): Ketamine
Sponsor:
Date of BT Designation Disclosure: 4/16/2018
Indication: Patients with depression
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): 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): 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): bb2121
Sponsor:
Date of BT Designation Disclosure: 11/04/2017
Indication: Patients with relapsed or refractory multiple myeloma
Category: Cancer
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): 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): 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): 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): 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): 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): 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): Nuplazid (pimavanserin)
Sponsor:
Date of BT Designation Disclosure: 2017
Indication: Patients with dementia-related psychosis (DRP)
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): SAGE-217
Sponsor:
Date of BT Designation Disclosure: 2/07/2018
Indication: Patients with major depressive disorder (MDD)
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): 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): 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): 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): Gavreto (Pralsentinib)
Sponsor: Blueprint Medicines Corporation
Date of BT Designation Disclosure: 2/26/2019
Approval Date: 9/04/2020
Indication: Patients with RET fusion-positive NSCLC that has progressed on platinum-based chemotherapy
Category: Cancer
Trade Name (Agent): LN-145
Sponsor: Iovance Biotherapeutics, Inc.
Date of BT Designation Disclosure: 5/22/2019
Indication: Patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy
Category: Cancer
Trade Name (Agent): Zolgensma (onasemnogene abeparvovec-xioi)
Sponsor: AveXis
Date of BT Designation Disclosure: 7/20/2016
Approval Date: 5/24/2019
Indication: Spinal muscular atrophy (SMA) Type 1 in children less than two years old
Category: Other
Trade Name (Agent): enfortumab vedotin
Sponsor: Astellas Pharma US, Inc. and Seattle Genetics, Inc.
Date of BT Designation Disclosure: 3/26/2018
Indication: Patients with locally advanced or metastatic urothelial carcinoma
Category: Cancer
Trade Name (Agent): Breyanzi (Lisocabtagene maraleucel)
Sponsor:
Date of BT Designation Disclosure: 12/20/2016
Approval Date: 2/05/2021
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
Trade Name (Agent): Myrcludex
Sponsor: MYR Pharma
Date of BT Designation Disclosure: 10/23/2018
Indication: Chronic hepatitis delta (HDV) infection
Category: Infectious Disease
Trade Name (Agent): LOXO-292
Sponsor: Loxo Oncology, Inc.
Date of BT Designation Disclosure: 10/16/2018
Indication: Patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment, and have no acceptable alternative treatment options
Category: Cancer
Trade Name (Agent): Poteligeo (mogamulizumab-kpkc)
Sponsor: Kyowa Kirin Inc.
Date of BT Designation Disclosure: 10/25/2017
Approval Date: 8/08/2018
Indication: Adult patients Sézary syndrome (SS) after at least one prior systemic therapy
Category: Cancer
Trade Name (Agent): Danyelza (naxitamab-gqgk)
Sponsor: Y-mAbs Therapeutics, Inc.
Date of BT Designation Disclosure: 8/23/2018
Approval Date: 11/25/2020
Indication: In combination with GM-CSF for patients with high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow
Category: Cancer
Trade Name (Agent): LOXO-292
Sponsor: Loxo Oncology, Inc.
Date of BT Designation Disclosure: 9/05/2018
Indication: Patients with metastatic RET-fusion-positive non-small cell lung cancer; patients with RET-mutant medullary thyroid cancer
Category: Cancer
Trade Name (Agent): PF-06651600
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 9/06/2018
Indication: Patients with alopecia areata
Category: Other
Trade Name (Agent): Tezepelumab
Sponsor: AstraZeneca, AstraZeneca & Amgen Inc.
Date of BT Designation Disclosure: 9/07/2018
Indication: Patients with severe asthma, without an eosinophilic phenotype, who are receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers
Category: Other
Trade Name (Agent): CA-008
Sponsor: Concentric Analgesics, Inc.
Date of BT Designation Disclosure: 9/11/2018
Indication: Patients needing long-lasting, non-opioid pain relief
Category: Other
Trade Name (Agent): Prevnar 20 (PF-06482077)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 9/20/2018
Approval Date: 6/08/2021
Indication: Prevention of pneumonia and invasive disease caused by Streptococcus pneumoniae serotypes
Category: Infectious Disease
Trade Name (Agent): Psilocybin therapy
Sponsor: COMPASS Pathways
Date of BT Designation Disclosure: 10/23/2018
Indication: Treatment-resistant Depression
Category: Other
Trade Name (Agent): AT-GAA (ATB200 + AT2221)
Sponsor: Amicus Therapeutics
Date of BT Designation Disclosure: 2/26/2019
Indication: Patients with late onset Pompe disease
Category: Rare Inherited Disorders
Trade Name (Agent): CP101
Sponsor: Finch Therapeutics Group, Inc.
Date of BT Designation Disclosure: 2/09/2019
Indication: Patients with recurrent Clostridium difficile infection
Category: Infectious Disease
Trade Name (Agent): Promacta (eltrombopag)
Sponsor:
Date of BT Designation Disclosure: 11/16/2018
Indication: Low platelet counts in people exposed to radiation
Category: Other
Trade Name (Agent): NGX-101
Sponsor:
Date of BT Designation Disclosure: 11/13/2018
Indication: Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy
Category: Other
Trade Name (Agent): Xolair (omalizumab)
Sponsor:
Date of BT Designation Disclosure: 8/13/2018
Approval Date: 9/28/2018
Indication: Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies
Category: Other
Trade Name (Agent): Andexxa (andexanet alfa)
Sponsor:
Date of BT Designation Disclosure: 11/25/2013
Approval Date: 5/03/2018
Indication: patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding
Category: Cardiovascular
Trade Name (Agent): Olipudase Alfa
Sponsor:
Date of BT Designation Disclosure: 11/29/2018
Indication: Niemann-Pick disease Type B (NPB)
Category: Rare Inherited Disorders
Trade Name (Agent): lonafarnib
Sponsor: Eiger BioPharmaceuticals
Date of BT Designation Disclosure: 11/19/2018
Approval Date: 11/20/2020
Indication: Patients with Hutchinson-Gilford progeria syndrome (HGPS_
Category: Rare Inherited Disorders
Trade Name (Agent): MEDI8897
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 2/06/2019
Indication: Prevention of lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV)
Category: Other
Trade Name (Agent): Vaxneuvance (V114)
Sponsor: Merck & Co., Inc.
Date of BT Designation Disclosure: 1/28/2019
Approval Date: 7/16/2021
Indication: Prevention of invasive pneumococcal disease in adults 18 years and older caused by 15 serotypes.
Category: Infectious Disease
Trade Name (Agent): UKONIQ (umbralisib)
Sponsor: TG Therapeutics
Date of BT Designation Disclosure: 1/28/2019
Approval Date: 2/05/2021
Indication: Adult patients with marginal zone lymphoma (MZL) who did not respond to at least one anti-CD20 therapy
Category: Cancer
Trade Name (Agent): Luxturna (voretigene neparvovec-rzyl)
Sponsor: Spark Therapeutics Inc.
Date of BT Designation Disclosure: 9/24/2014
Approval Date: 12/19/2017
Indication: Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy
Category: Rare Inherited Disorders
Trade Name (Agent): Rubraca (rucaparib)
Sponsor: Clovis Oncology
Date of BT Designation Disclosure: 10/03/2018
Indication: BRCA1/BRCA2-mutated, metastatic, castration-resistant prostate cancer
Category: Cancer
Trade Name (Agent): Trikafta (elexacaftor/ivacaftor/tezacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 5/01/2018
Approval Date: 10/21/2019
Indication: Patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
Category: Rare Inherited Disorders
Trade Name (Agent): Givlaari (givosiran)
Sponsor: Alnylam Pharmaceuticals Inc.
Date of BT Designation Disclosure: 5/30/2017
Approval Date: 11/20/2019
Indication: Adult patients with acute hepatic porphyria (AHP)
Category: Rare Inherited Disorders
Trade Name (Agent): Jakafi (Ruxolitinib)
Sponsor: Incyte
Date of BT Designation Disclosure: 6/23/2016
Approval Date: 5/28/2019
Indication: Patients aged 12 years and older with steroid-refractory acute graft versus-host disease (GVHD)
Category: Other
Trade Name (Agent): Aliqopa (copanlisib)
Sponsor: Bayer
Date of BT Designation Disclosure: 5/30/2019
Indication: Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies
Category: Cancer
Trade Name (Agent): Oliceridine (TRV130)
Sponsor: Trevena, Inc.
Date of BT Designation Disclosure: 2/22/2016
Indication: Moderate-to-Severe Acute Pain
Category: Other
Trade Name (Agent): AXS-05
Sponsor: Axsome Therapeutics, Inc.
Date of BT Designation Disclosure: 3/27/2019
Indication: Major Depressive Disorder (MDD)
Category: Rare Inherited Disorders
Trade Name (Agent): PB2452
Sponsor: PhaseBio Pharmaceuticals
Date of BT Designation Disclosure: 4/08/2019
Indication: Reversal agent for the antiplatelet drug ticagrelor
Category: Other
Trade Name (Agent): SER-109
Sponsor: Seres Therapeutics, Inc.
Date of BT Designation Disclosure: 4/29/2019
Indication: Recurrent C. difficile infection
Category: Rare Inherited Disorders
Trade Name (Agent): elafibranor
Sponsor: Genfit
Date of BT Designation Disclosure: 4/19/2019
Indication: Patients with primary biliary cholangitis (PBC)
Category: Other
Trade Name (Agent): LJPC-0118
Sponsor: La Jolla Pharmaceutical Company
Date of BT Designation Disclosure: 4/24/2019
Indication: Patients with severe malaria
Category: Infectious Disease
Trade Name (Agent): Zejula (niraparib)
Sponsor: Janssen Pharmaceutica
Date of BT Designation Disclosure: 10/03/2019
Indication: Patients with BRCA1/2-mutated metastatic castration-resistant prostate cancer (CRPC) who have previously received taxane chemotherapy and androgen receptor (AR)–targeted therapy
Category: Cancer
Trade Name (Agent): Cotellic (cobimetinib)
Sponsor: Memorial Sloan Kettering Cancer Center
Date of BT Designation Disclosure: 10/03/2019
Indication: Adult patients with histiocytic neoplasms who do not harbor a BRAF V600 mutation
Category: Rare Inherited Disorders
Trade Name (Agent): Hemlibra (emicizumab-kxwh)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 4/17/2018
Approval Date: 10/04/2018
Indication: Patients with hemophilia A without factor VIII inhibitors
Category: Rare Inherited Disorders
Trade Name (Agent): Rezurock (belumosudil)
Sponsor: Kadmon Holdings, Inc.
Date of BT Designation Disclosure: 10/17/2018
Approval Date: 7/16/2021
Indication: Patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy
Category: Other
Trade Name (Agent): Braftovi (encorafenib) + Mektovi (binimetinib) + Erbitux (cetuximab)
Sponsor: Array BioPharma Inc., Eli Lilly and Company
Date of BT Designation Disclosure: 8/07/2018
Indication: Patients with BRAF V600E-mutation metastatic colorectal caner following 1 or 2 prior lines of treatment in the metastatic setting
Category: Cancer
Trade Name (Agent): seladelpar
Sponsor: CymaBay Therapeutics
Date of BT Designation Disclosure: 2/20/2019
Indication: Patients with early stage primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA)
Category: Other
Trade Name (Agent): Avexitide
Sponsor: Eiger BioPharmaceuticals Inc.
Date of BT Designation Disclosure: 6/17/2019
Indication: Post-bariatric hypoglycemia (PBH)
Category: Other
Trade Name (Agent): DCR-PHXC
Sponsor: Dicerna Pharmaceuticals
Date of BT Designation Disclosure: 7/15/2019
Indication: Primary hyperoxaluria type 1 (caused by a mutation in the AGXT gene)
Category: Other
Trade Name (Agent): Peginterferon Lambda
Sponsor: Eiger Pharmaceuticals
Date of BT Designation Disclosure: 8/20/2019
Indication: Hepatitis delta virus (HDV) infection
Category: Infectious Disease
Trade Name (Agent): NKTR-214 (bempegaldesleukin) with Opdivo (nivolumab)
Sponsor: Nektar Therapeutics
Date of BT Designation Disclosure: 8/23/2019
Indication: Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery
Category: Cancer
Trade Name (Agent): Nirogacestat
Sponsor: SpringWorks Therapeutics, Inc.
Date of BT Designation Disclosure: 8/29/2019
Indication: Adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis
Category: Cancer
Trade Name (Agent): Tepotinib (Tepmetko)
Sponsor: Merck
Date of BT Designation Disclosure: 9/11/2019
Approval Date: 2/03/2021
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) harboring a MET exon 14 skipping alteration who have progressed following platinum-based chemotherapy
Category: Cancer
Trade Name (Agent): Kisqali (ribociclib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 6/20/2018
Approval Date: 7/18/2018
Indication: In combination with an aromatase inhibitor for pre/perimenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy
Category: Cancer
Trade Name (Agent): Yervoy (ipilimumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 2/10/2018
Approval Date: 7/10/2018
Indication: Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 2/10/2018
Approval Date: 6/10/2018
Indication: Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan
Category: Cancer
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 2017
Approval Date: 6/13/2018
Indication: Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after two or more prior lines of therapy
Category: Cancer
Trade Name (Agent): Krintafel (tafenoquine)
Sponsor: GlaxoSmithKline Intellectual Property Development LTD England
Date of BT Designation Disclosure: 12/18/2013
Approval Date: 7/20/2018
Indication: For the radical cure (prevention of relapse) of plasmodium vivax malaria in patients aged 16 years and older who are receiving appropriate antimalarial therapy for acute P. vivax
Category: Infectious Disease
Trade Name (Agent): Azedra (iobenguane I 131)
Sponsor: Progenics Pharmaceuticals Inc.
Date of BT Designation Disclosure: 7/28/2015
Approval Date: 7/30/2018
Indication: Adult and pediatric patients (12 years and older) with iobenguane scan-positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) who require systemic anticancer therapy
Category: Cancer
Trade Name (Agent): Poteligeo (mogamulizumab-kpkc)
Sponsor: Kyowa Kirin Inc.
Date of BT Designation Disclosure: 8/25/2017
Approval Date: 8/08/2018
Indication: Adult patients with relapsed or refractory mycosis fungoides (MF) or Sezary syndrome after at least one prior systemic therapy
Category: Cancer
Trade Name (Agent): Orkambi (lumacaftor/ivacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 3/12/2018
Approval Date: 8/07/2018
Indication: Treatment of cystic fibrosis (CF) in patients 2 years and older, homozygous for the F508del-CFTR mutation in the CFTR gene
Category: Rare Inherited Disorders
