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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 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.

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Trade Name (Agent)

Sponsor

Date of BT Designation Disclosure

Approval Date

Indication

Category

Trade Name (Agent): Lenti-D

Sponsor:

Date of BT Designation Disclosure: 5/23/2018

Indication: Cerebral adrenoleukodystrophy

Category: Rare Inherited Disorders

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