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