List of drugs granted breakthrough therapy designation

From Wikipedia, the free encyclopedia

Below is a list of drugs granted breakthrough therapy designation (BTD) by the FDA.

Drugs may be listed more than once as BTD can be awarded for multiple indications.

2020[]

Drug Manufacturer Indication
Avapritinib Blueprint Medicines Corp metastatic gastrointestinal stromal tumor
Tepezza Horizon Therapeutics thyroid eye disease
Nintedanib Boehringer Ingelheim chronic fibrosing interstitial lung diseases
Ipilimumab Bristol-Myers Squibb hepatocellular carcinoma
Nivolumab Bristol-Myers Squibb hepatocellular carcinoma
Selumetinib AstraZeneca neurofibromatosis type 1
Mitomycin Urogen Pharma upper tract urothelial cancer
Tucatinib Seattle Genetics advanced unresectable or metastatic HER2-positive breast cancer
Pemigatinib Incyte Corp metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion
Trodelvy Immunomedics metastatic triple-negative breast cancer
Capmatinib Novartis metastatic non-small cell lung cancer
Selpercatinib Loxo Oncology metastatic RET fusion-positive non-small cell lung cancer
Ripretinib Deciphera Pharmaceuticals advanced gastrointestinal stromal tumor
Artesunate Amivas LLC severe malaria
Bevacizumab Genentech metastatic hepatocellular carcinoma
Atezolizumab Genentech metastatic hepatocellular carcinoma
Inebilizumab Viela Bio neuromyelitis optica spectrum disorder
Fostemsavir Viiv Healthcare Co multidrug-resistant HIV-1
Monjuvi MorphoSys relapsed or refractory diffuse large B-cell lymphoma
Esketamine Janssen major depressive disorder with acute suicidal ideation or behavior
Blenrep GlaxoSmithKline refractory multiple myeloma
Enspryng Genentech neuromyelitis optica spectrum disorder
Pralsetinib Blueprint Medicines Corp RET fusion-positive non-small cell lung cancer

|| ||

2019[]

Drug Manufacturer Indication
Psilocybin Usona Institute major depressive disorder[1]
B38M (JNJ‐4528) Legend Biotech/Janssen multiple myeloma
Rilonacept Kiniska Pharmaceuticals recurrent pericarditis
Inmazeb Regeneron Ebola Virus
Olorofim F2G invasive mold infections
Nintedanib Boehringer Ingelheim interstitial lung disease
Niraparib Janssen castration-resistant prostate cancer
Cobimetinib Memorial Sloan Kettering histiocytic neoplasms
Tepotinib Merck metastatic non-small cell lung cancer
Ansuvimab Ridgeback Biotherapeutics Ebola Virus
Capmatinib Novartis metastatic non-small cell lung cancer
Nirogacestat SpringWorks Therapeutics refractory desmoid tumors
Bempegaldesleukin Nektar Therapeutics melanoma
Peginterferon Lambda Eiger Pharmaceuticals hepatitis delta virus infection
Acalabrutinib AstraZeneca chronic lymphocytic leukemia
Trilaciclib G1 Therapeutics small-cell lung cancer
Nedosiran Dicerna Pharmaceuticals primary hyperoxaluria
Pembrolizumab Merck hepatocellular carcinoma
Avexitide Eiger BioPharmaceuticals post-bariatric hypoglycemia
Copanlisib Bayer marginal zone lymphoma
LN-145 Iovance Biotherapeutics cervical cancer
Trastuzumab deruxtecan Daiichi Sankyo HER2-positive breast cancer
SER-109 Seres Therapeutics Clostridioides difficile infection
I.V. Artesunate La Jolla Pharmaceutical malaria
Elafibranor Genfit primary biliary cholangitis
Inebilizumab Viela Bio neuromyelitis optica spectrum disorder
Bentracimab PhaseBio Pharmaceuticals reversal agent for ticagrelor
Selumetinib AstraZeneca/Merck pediatric neurofibromatosis type 1
AXS-05 Axsome Therapeutics major depressive disorder
ivosidenib Agios Pharmaceuticals acute myeloid leukemia
Venetoclax Abbvie chronic lymphocytic leukemia
AT-GAA Amicus Therapeutics Pompe disease
Pralsentinib Blueprint Medicines Non-small cell lung cancer
Seladelpar CymaBay Therapeutics primary biliary cholangitis
CP101 Finch Therapeutics Clostridioides difficile infection
Nirsevimab AstraZeneca respiratory syncytial virus
Kadcyla Genentech HER2-positive breast cancer
Umbralisib TG Therapeutics marginal-zone lymphoma
V114 Merck pneumococcal disease
Zanubrutinib BeiGene USA mantle cell lymphoma
Crizanlizumab Novartis sickle cell disease
Encorafenib Array BioPharma metastatic colorectal cancer

[2]

2018[]

Drug Manufacturer Indication
Psilocybin COMPASS Pathways treatment resistant depression[3][4]
Satralizumab Genentech neuromyelitis optica spectrum disorder
Olipudase Alfa Sanofi/Genzyme rare inherited disorders
Lonafarib Eiger BioPharmaceuticals Hutchinson-Gilford progeria syndrome
Eltrombopag Novartis thrombocytopenia
Galcanezumab Eli Lilly episodic cluster headache
Brentuximab Seattle Genetics anaplastic large-cell lymphoma
NGX-101 NeuroRx bipolar depression
Mitomycin C UroGen Pharma urothelial cancer
Myrcludex MYR Pharma hepatitis delta
Belumosudil Kadmon Holdings graft-versus-host disease
Selpercatinib Loxo Oncology thyroid cancer/
Selpercatinib Loxo Oncology Non-small cell lung cancer
Rucaparib Clovis Oncology castration-resistant prostate cancer
PF-06482077 Pfizer pneumonia
Vocacapsaicin Concentric Analgesics pain relief
Tezepelumab AstraZeneca/Amgen asthma
Ritlecitinib Pfizer alopecia areata
Danyelza Y-mAbs Therapeutics neuroblastoma
Omalizumab Genentech severe allergic reactions
Encorafenib Array BioPharma/Eli Lilly colorectal cancer
Quizartinib Daiichi Sankyo acute myeloid leukemia
Pembrolizumab Merck endometrial carcinoma
Lenvatinib Eisai endometrial carcinoma
Bevacizumab Genentech hepatocellular carcinoma
Atezolizumab Genentech hepatocellular carcinoma
AMT-601 uniQure hemophilia B
HTX-011 Heron Therapeutics Postoperative Pain
Ribociclib Novartis breast cancer
Tagraxofusp Stemline Therapeutics blastic plasmacytoid dendritic cell neoplasm
Crizotinib Pfizer anaplastic large cell lymphoma
Crizotinib Pfizer small-cell lung cancer
Tafamidis meglumine Foldrx Pharmaceuticals cardiomyopathy
Lenti-D Bluebird bio cerebral adrenoleukodystrophy
Tafamidis Pfizer transthyretin cardiomyopathy
Pitolisant Harmony Biosciences narcolepsy
Trikafta Vertex Pharmaceuticals cystic fibrosis
Narsoplimab Omeros Corporation thrombotic microangiopathy
Trumenba Pfizer meningococcal disease
Emicizumab Genentech hemophilia
Ketamine Johnson & Johnson depression
Amikacin Insmed mycobacterial lung disease
Sodium thiosulfate Fennec Pharmaceuticals hepatoblastoma
Enfortumab vedotin Astellas Pharma metastatic urothelial carcinoma
Nesolicaftor Proteostasis Therapeutics cystic fibrosis
Erdafitinib Janssen urothelial carcinoma
Lumasiran Alnylam Pharmaceuticals primary hyperoxaluria
Lumacaftor/ivacaftor Vertex Pharmaceuticals cystic fibrosis
Sutimlimab Bioverativ cold agglutinin disease
Avasopasem manganese Galera Therapeutics oral mucositis
Nivolumab Bristol Myers Squibb renal cell carcinoma
Ipilimumab Bristol Myers Squibb renal cell carcinoma
SPK-8011 Spark Therapeutics hemophilia A
Abrocitinib Pfizer atopic dermatitis
Ipilimumab Bristol Myers Squibb metastatic colorectal cancer
Nivolumab Bristom Myers Squibb metastatic colorectal cancer
Zuranolone Sage Therapeutics major depressive disorder
Fenfluramine Zogenix seizures associated with Dravet syndrome
Patisiran Alnylam Pharmaceuticals hereditary transthyretin-mediated amyloidosis
Balovaptan Hoffman-La Roche autism spectrum disorder
Kisquali Femara Co-Pack Novartis HR-positive, HER2- negative breast cancer
Upadacitinib AbbVie atopic dermatitis
Lenvatinib + Pembrolizumab Merck/Eisai metastatic renal cell carcinoma
Voxelotor Global Blood Therapeutics sickle cell disease
Maribavir Shire Cytomegalovirus infection
Eltrombopag Novartis severe aplastic anemia
Emapalumab Swedish Orphan Biovitrum hemophagocytic lymphohistiocytosis
Venetoclax AbbVie chronic lymphocytic leukemia

[5]


2017[]

[6]

Drug Manufacturer Indication
Nivolumab Bristol-Myers Squibb urothelial carcinoma
Ribociclib Novartis HR-positive, HER2-negative breast cancer
Pembrolizumab Merck Hodgkin Lymphoma
Avelumab EMD Serono metastatic Merkel cell carcinoma
Niraparib Tesaro recurrent ovarian cancer responsive to platinum-based chemotherapy
Ocrelizumab Genentech multiple sclerosis
Dupilumab Regeneron Pharmaceuticals atopic dermatitis
Palbociclib Pfizer HR-positive, HER2-negative breast cancer
Valbenazine Neurocrine Biosciences tardive dyskinesia
Cerliponase alpha BioMarin tripeptidyl peptidase 1 (TPP1) deficiency
Midostaurin Novartis FLT3-positive acute myeloid leukemia
Brigatinib Ariad Pharmaceuticals ALK-positive non-small cell lung cancer
Durvalumab AstraZeneca metastatic urothelial carcinoma
Kisqali-Femara Co-pack Novartis breast cancer
Ribaxamase Synthetic Biologics antibiotic resistance / Clostridioides difficile infection
Midomafetamine (Ecstasy) Multidisciplinary Association for Psychedelic Studies posttraumatic stress disorder[7][8]


2016[]

Drug Manufacturer Indication
Elbasvir/grazoprevir Merck Hepatitis C
Palbociclib Pfizer HR-positive, HER2-negative breast cancer
Crizotinib PF Prism ROS-1-positive NSCLC
Venetoclax AbbVie CLL with 17p deletion
Cabozantinib Exelixis renal cell carcinoma
Pimavanserin Acadia Pharmaceuticals Parkinson's disease-related psychosis
Lenvatinib mesylate Eisai renal cell carcinoma
Nivolumab Bristol-Myers Squibb Hodgkin lymphoma
Atezolizumab Genentech urothelial carcinoma
Lumacaftor/ivacaftor Vertex Pharmaceuticals cystic fibrosis with CFTR F508del
Canakinumab Novartis Familial mediterranean fever
Canakinumab Novartis Hyper-IgD syndrome
Canakinumab Novartis TNF receptor associated periodic syndrome
Atezolizumab Genentech metastatic NSCLC
Olaratumab Eli Lilly soft-tissue sarcoma
Pembrolizumab Merck metastatic NSCLC
Nivolumab Bristol-Myers Squibb head and neck cancer
Daratumumab Janssen multiple myeloma
Oliceridine Trevena Analgesia and pain management
Rucaparib Clovis Oncology BRCA-mutated ovarian cancer
Silver diamine fluoride Elevate Oral Care early childhood caries


2015[]

Drug Manufacturer Indication
Ibrutinib Pharmacyclics Waldenstrom’s macroglobulinemia
Palbociclib Pfizer ER-positive, HER2-negative breast cancer
Ranibizumab Genentech diabetic retinopathy
ivacaftor Vertex Pharmaceuticals cystic fibrosis with a variety of CFTR mutations
Aflibercept Regeneron Pharmaceuticals diabetic retinopathy
Sirolimus PF Prism lymphangioleiomyomatosis
Lumacaftor/ivacaftor Vertex Pharmaceuticals cystic fibrosis with CFTR F508del mutation
Ombitasvir/paritaprevir/ritonavir AbbVie Hepatitis C
Uridine triacetate Wellstat hereditary orotic aciduria
Pembrolizumab Merck PDL-1 positive NSCLC
Nivolumab Bristol-Myers Squibb metastatic NSCLC
Idarucizumab Boehringer Ingelheim reversal of dabigatran
Asfotase alfa Alexion perinatal/infantile/childhood hypophosphatasia
Osimertinib AstraZeneca EGFR-positive NSCLC
Daratumumab Janssen multiple myeloma
Nivolumab Bristol-Myers Squibb advanced renal cell carcinoma
Elotuzumab Bristol-Myers Squibb multiple myeloma
Sebelipase alfa Synageva lysosomal acid lipase deficiency
Alectinib Roche ALK-mutated NSCLC
Pembrolizumab Merck metastatic melanoma
Ibalizumab TaiMed Biologics HIV-1 infection


2014[]

Drug Manufacturer Indication
Ivacaftor Vertex Pharmaceuticals cystic fibrosis with CFTR mutation
Ofatumumab GlaxoSmithKline chronic lymphocytic leukemia (CLL)
Ceritinib Novartis ALK-positive NSCLC
Idelalisib Gilead relapsed CLL
Ibrutinib Pharmacyclics CLL with 17p deletion
Eltrombopag GlaxoSmithKline aplastic anemia
Pembrolizumab Merck metastatic melanoma
Ledipasvir/sofosbuvir Gilead Hepatitis C
Nintedanib Boehringer Ingelheim idiopathic pulmonary fibrosis
Pirfenidone Genentech idiopathic pulmonary fibrosis
Blinatumomab Amgen acute lymphoblastic leukemia
Ombitasvir/paritaprevir/ritonavir AbbVie Hepatitis C
Nivolumab Bristol-Myers Squibb metastatic melanoma
Ivacaftor Vertex Pharmaceuticals cystic fibrosis with CFTR R117H mutation

2013[]

Drug Manufacturer Indication
Obinutuzumab Genentech chronic lymphocytic leukemia
Ibrutinib Pharmacyclics mantle cell lymphoma
Sofosbuvir Gilead Hepatitis C




References[]

  1. ^ "FDA Grants Psilocybin Second Breakthrough Therapy Designation". Medscape. Retrieved 5 January 2020.
  2. ^ Research, Center for Drug Evaluation and (2020-10-08). "Breakthrough Therapy Approvals". FDA.
  3. ^ "COMPASS Pathways Receives FDA Breakthrough Therapy Designation for Psilocybin Therapy for Treatment-resistant Depression". www.compasspathways.com. October 23, 2018. Retrieved April 2, 2019.
  4. ^ "FDA Puts 'Magic Mushroom' Ingredient on Fast Track for Depression Treatment". www.healthline.com. November 7, 2018. Retrieved April 2, 2019.
  5. ^ Research, Center for Drug Evaluation and (2020-10-08). "Breakthrough Therapy Approvals". FDA.
  6. ^ "Approval report" (PDF). www.fda.gov. Retrieved 2019-06-12.
  7. ^ "FDA Grants Breakthrough Therapy Designation for MDMA-Assisted Psychotherapy for PTSD". maps.org. August 26, 2017. Retrieved April 2, 2019.
  8. ^ "Ecstasy as a Remedy for PTSD? You Probably Have Some Questions". www.nytimes.com. May 1, 2018. Retrieved April 2, 2019.
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