Drugs granted breakthrough therapy designation (BTD) by the US Food and Drug Administration (FDA). Drugs may be listed more than once since breakthrough therapy can be awarded for multiple indications.
| Brand name | Manufacturer | Indication | |
|---|---|---|---|
| Elrexfio | To treat relapsed or refractory multiple myeloma after at least four lines of therapy[1] | ||
| Fabhalta | To treat paroxysmal nocturnal hemoglobinuria | ||
| Izervay | To treat geographic atrophy secondary to age-related macular degeneration | ||
| Leqembi | To treat Alzheimer's disease | ||
| Loqtorzi | To treat recurrent or metastatic nasopharyngeal carcinoma with or following other therapies | ||
| Ogsiveo | To treat desmoid tumors | ||
| Pombiliti | To treat late-onset Pompe disease with miglustat | ||
| Rivfloza | To lower urinary oxalate levels in primary hyperoxaluria type 1 and relatively preserved kidney function | ||
| Talvey | To treat relapsed or refractory multiple myeloma after at least four therapies |
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| Abrocitinib | Pfizer | atopic dermatitis | |
| Tebentafusp | Immunocore | HLA-A*02:01–positive uveal melanoma | |
| Sutimlimab | Bioverativ | cold agglutinin disease | |
| Lutetium (177Lu) vipivotide tetraxetan | Novartis | prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistantprostate cancer (mCRPC) | |
| Alpelisib | Novartis | PIK3CA-related overgrowth spectrum conditions | |
| Mavacamten | Myokardia Inc | class II-III obstructive hypertrophic cardiomyopathy | |
| Trastuzumab deruxtecan | Daiichi Sankyo | granted for three indications: HER2-positive breast cancer with prior anti-HER2-based treatment; HER2 low (IHC 1+ or IHC 2+/ISH) breast cancer; and non-small cell lung cancer with an activating HER2 mutation | |
| Dupilumab | Regeneron Pharmaceuticals | granted for two indications: eosinophilic esophagitis; and atopic dermatitis | |
| Ivosidenib | Servier Pharmaceuticals | acute myeloid leukemia with a susceptible IDH1 mutation | |
| Baricitinib | Eli Lilly and Co | severe alopecia areata | |
| Setmelanotide | Rhythm Pharmaceuticals | Bardet–Biedl syndrome | |
| Dextromethorphan/bupropion | Axsome Therapeutics | major depressive disorder | |
| Pemigatinib | Incyte | myeloid/lymphoid neoplasms with FGFR1 rearrangement | |
| Olipudase alfa | Genzyme | non–central nervous system manifestations of acid sphingomyelinase deficiency | |
| Spesolimab | Boehringer Ingelheim | generalized pustular psoriasis | |
| Futibatinib | Taiho Oncology | intrahepatic cholangiocarcinoma with FGFR2 gene fusions or other rearrangements | |
| Teclistamab | Janssen | multiple myeloma | |
| Teplizumab | Provention Bio | delay the onset of Stage 3 type 1 diabetes (T1D) in Stage 2 T1D | |
| Atezolizumab | Genentech | alveolar soft part sarcoma | |
| Adagrasib | Mirati Therapeutics | KRAS G12C-mutated non-small cell lung cancer | |
| Mosunetuzumab | Genentech | follicular lymphoma | |
| Lenacapavir | Gilead Sciences | HIV-1 |
| Crizotinib | PF Prism | ALK-positive systemic anaplastic large cell lymphoma | |
| Trastuzumab deruxtecan | Daiichi Sankyo | HER2-positive gastric or gastroesophageal junction adenocarcinoma, previously treated with trastuzumab | |
| Umbralisib | TG Therapeutics | marginal zone lymphoma, previously treated with an anti-CD20-based regimen | |
| Evinacumab | Regeneron Pharmaceuticals | homozygous familial hypercholesterolemia | |
| Trilaciclib | G1 Therapeutics | myelosuppression, when administered prior to a platinum/etoposide or topotecan containing regimen for small cell lung cancer | |
| Fosdenopterin | Origin Biosciences | molybdenum cofactor deficiency, Type A | |
| Tocilizumab | Genentech | systemic sclerosis-associated interstitial lung disease | |
| Rilonacept | Kiniksa Pharmaceuticals | recurrent pericarditis | |
| Dostarlimab | GSK | mismatch repair deficient recurrent or advanced endometrial cancer | |
| Dapagliflozin | AstraZeneca | chronic kidney disease | |
| Bupivacaine/meloxicam | Heron Therapeutics | postsurgical analgesia | |
| Amivantamab | Janssen Biotech | non-small cell lung cancer with EGFR exon 20 insertion mutations | |
| Sotorasib | Amgen | non-small cell lung cancer with KRAS G12C mutation | |
| Avapritinib | Blueprint Medicines Corp | granted for two indications: mast cell leukemia and advanced systemic mastocytosis | |
| Belumosudil | Kadmon Pharmaceuticals | chronic graft-versus-host disease | |
| Pembrolizumab | Merck | high risk, early-stage triple-negative breast cancer | |
| Avalglucosidase alfa | Genzyme | late-onset Pompe disease | |
| Lenvatinib | Eisai Inc | advanced renal cell carcinoma, used in combination with pembrolizumab | |
| Difelikefalin | Cara Therapeutics | pruritis related to hemodialysis in chronic kidney disease | |
| Mobocertinib | Takeda Pharmaceuticals | non-small cell lung cancer with EGFR exon 20 insertion mutations | |
| Cabozantinib | Exelixis | thyroid cancer | |
| Maralixibat | Mirum Pharmaceuticals | cholestatic pruritus in Alagille syndrome | |
| Asciminib | Novartis | granted for two indications: Philadelphia chromosome-positive chronic myeloid leukemia previously treated with tyrosine kinase inhibitors, and Philadelphia chromosome-positive chronic myeloid leukemia with T315I mutation | |
| Sirolimus | AADI Bioscience | metastatic or unresectable perivascular epithelioid cell tumor | |
| Maribavir | Takeda | refractory post-transplant cytomegalovirus infection | |
| Abatacept | Bristol Myers Squibb | acute graft-versus-host disease, in combination with a calcineurin inhibitor and methotrexate, undergoing hematopoietic stem cell transplantation from a matched or 1 allele-mismatched unrelated donor | |
| Voxelotor | Global Blood Therapeutics Inc | sickle cell disease | |
| Cabotegravir | ViiV Healthcare Co | pre-exposure prophylaxis for HIV |
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| Blueprint Medicines Corp | metastatic gastrointestinal stromal tumor | ||
| Horizon Therapeutics | thyroid eye disease | ||
| Boehringer Ingelheim | chronic fibrosing interstitial lung diseases | ||
| Bristol-Myers Squibb | hepatocellular carcinoma | ||
| Bristol-Myers Squibb | hepatocellular carcinoma | ||
| AstraZeneca | neurofibromatosis type 1 | ||
| Urogen Pharma | upper tract urothelial cancer | ||
| Seattle Genetics | advanced unresectable or metastatic HER2-positive breast cancer | ||
| Incyte Corp | metastatic cholangiocarcinoma with FGFR2 fusion | ||
| Immunomedics | metastatic triple-negative breast cancer | ||
| Novartis | metastatic non-small cell lung cancer | ||
| Loxo Oncology | metastatic RET fusion-positive non-small cell lung cancer | ||
| AIDS-related Kaposi sarcoma | |||
| metastatic castration-resistant prostate cancer | |||
| Deciphera Pharmaceuticals | advanced gastrointestinal stromal tumor | ||
| Amivas LLC | severe malaria | ||
| Genentech | metastatic hepatocellular carcinoma | ||
| Genentech | metastatic hepatocellular carcinoma | ||
| Inebilizumab-cdon | Viela Bio | neuromyelitis optica spectrum disorder | |
| multidrug-resistant HIV-1 | |||
| relapsed or refractory diffuse large B-cell lymphoma | |||
| major depressive disorder with acute suicidal ideation or behavior | |||
| GlaxoSmithKline | refractory multiple myeloma | ||
| Genentech | neuromyelitis optica spectrum disorder | ||
| Blueprint Medicines Corp | RET fusion-positive non-small cell lung cancer | ||
| Harmony Biosciences | cataplexy in adults with narcolepsy | ||
| Ebola Zaire | |||
| Eiger BioPharmaceuticals | Hutchinson-Gilford progeria syndrome (HGPS), processing-deficient progeroid laminopathies with LMNA or ZMPSTE24 mutations | ||
| primary hyperoxaluria type 1 | |||
| Y-mAbs Therapeutics | relapsed or refractory high-risk neuroblastoma, used in combination with GM-CSF | ||
| Rhythm Pharmaceuticals | medical obesity caused by proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency | ||
| Blueprint Medicines Corp | RET-mutant medullary thyroid cancer or advanced or metastatic RET fusion-positive thyroid cancer, in radioactive iodine-refractory patients | ||
| active lupus nephritis | |||
| adjuvant therapy after tumor resection in non-small cell lung cancer with EGFR exon 19 deletions or exon 21 L858R mutations | |||
| Ebola Zaire |
| Drug | Manufacturer | Indication |
|---|---|---|
| HR-positive, HER2-negative advanced or metastatic breast cancer | ||
| treatment-resistant depression | ||
| atopic dermatitis | ||
| Sage Therapeutics | postpartum depression | |
| metastatic breast cancer | ||
| urothelial carcinoma | ||
| advanced renal cell carcinoma | ||
| HER2-positive early breast cancer | ||
| Foldrx Pharmaceuticals | cardiomyopathy | |
| advanced renal cell carcinoma | ||
| chronic lymphocytic leukemiaor small lymphocytic lymphoma | ||
| episodic cluster headache | ||
| diffuse large B-cell lymphoma | ||
| tenosynovial giant cell tumor | ||
| solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion | ||
| endometrial carcinoma | ||
| endometrial carcinoma | ||
| cystic fibrosis | ||
| BeiGene | mantle cell lymphoma | |
| sickle cell disease | ||
| acute hepatic porphyria | ||
| relapsed or refractory chronic lymphocytic leukemia | ||
| untreated chronic lymphocytic leukemia | ||
| Global Blood Therapeutics | sickle cell disease | |
| metastatic urothelial carcinoma | ||
| HER2-positive breast cancer | ||
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| Tezacaftor/ivacaftor | Vertex Pharmaceuticals | cystic fibrosis | |
| Durvalumab | AstraZeneca UK | unresectable Stage III non-small cell lung cancer | |
| Ibalizumab-uiyk | Theratechnologies | human immunodeficiency virus type 1 (HIV-1) infection | |
| Brentuximab vedotin | Seattle Genetics | Hodgkin lymphoma | |
| Ipilimumab | Bristol-Myers Squibb | renal cell carcinoma | |
| Nivolumab | Bristol-Myers Squibb | renal cell carcinoma | |
| Burosumab-twza | Kyowa Hakko Kirin | X-linked hypophosphatemia | |
| Osimertinib | AstraZeneca | metastatic non-small cell lung cancer | |
| Dabrafenib | Novartis Pharmaceuticals | melanoma with BRAF V600E or V600K mutations | |
| Trametinib | Novartis Pharmaceuticals | melanoma with BRAF V600E or V600K mutations | |
| Dabrafenib | Novartis Pharmaceuticals | anaplastic thyroid cancer with BRAF V600E mutation | |
| Trametinib | Novartis Pharmaceuticals | anaplastic thyroid cancer with BRAF V600E mutation | |
| Fingolimod | Novartis Pharmaceuticals | multiple sclerosis | |
| Rituximab | Genentech | pemphigus vulgaris | |
| Venetoclax | AbbVie | chronic lymphocytic leukemia | |
| Pembrolizumab | Merck Sharp & Dohme | mediastinal large B-cell lymphoma | |
| Ipilimumab | Bristol-Myers Squibb | metastatic colorectal cancer | |
| Nivolumab | Bristol-Myers Squibb | metastatic colorectal cancer | |
| Ribociclib | Novartis Pharmaceuticals | breast cancer | |
| Tafenoquine | GlaxoSmithKline | Plasmodium vivax malaria | |
| Iobenguane I 131 | Progenics Pharmaceuticals | pheochromocytoma or paraganglioma | |
| Lumacaftor/ivacaftor | Vertex Pharmaceuticals | cystic fibrosis | |
| Mogamulizumab-kpkc | Kyowa Hakko Kirin | mycosis fungoides or Sézary syndrome | |
| Patisiran | Alnylam Pharmaceuticals | hereditary transthyretin-mediated amyloidosis | |
| Cenegermin-bkbj | Dompe Farmaceutici | neurotrophic keratitis | |
| Lanadelumab-flyo | Takeda Pharmaceuticals | hereditary angioedema | |
| Cemiplimab-rwlc | Regeneron Pharmaceuticals | cutaneous squamous cell carcinoma | |
| Amikacin liposome inhalation suspension | Insmed | mycobacterial lung disease | |
| Emicizumab-kxwh | Genentech | hemophilia | |
| Lorlatinib | Pfizer | ALK-positive, metastatic non-small cell lung cancer | |
| Brentuximab vedotin | Seattle Genetics | anaplastic large-cell lymphoma | |
| Eltrombopag | Novartis Pharmaceuticals | severe aplastic anemia | |
| Emapalumab-lzsg | Swedish Orphan Biovitrum | hemophagocytic lymphohistiocytosis | |
| Venetoclax | AbbVie | acute myeloid leukemia | |
| Larotrectinib | Bayer Healthcare | solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion | |
| Larotrectinib | Bayer Healthcare | solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion | |
| Amifampridine | Catalyst Pharmaceuticals | Lambert-Eaton myasthenic syndrome | |
| Pembrolizumab | Merck Sharp & Dohme | Merkel cell carcinoma | |
| Tagraxofusp-erzs | Stemline Therapeutics | blastic plasmacytoid dendritic cell neoplasm | |
| Balovaptan[3] | Roche | Autism spectrum |
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| urothelial carcinoma | |||
| HR-positive, HER2-negative breast cancer | |||
| Hodgkin Lymphoma | |||
| metastatic Merkel cell carcinoma | |||
| recurrent ovarian cancer responsive to platinum-based chemotherapy | |||
| multiple sclerosis | |||
| atopic dermatitis | |||
| HR-positive, HER2-negative breast cancer | |||
| tardive dyskinesia | |||
| diabetic retinopathy | |||
| tripeptidyl peptidase 1 (TPP1) deficiency | |||
| FLT3-positive acute myeloid leukemia | |||
| ALK-positive non-small cell lung cancer | |||
| metastatic urothelial carcinoma | |||
| breast cancer | |||
| urothelial carcinoma | |||
| giant cell arteritis | |||
| microsatellite instability-high tumors | |||
| ALK-positive, metastatic non-small cell lung cancer | |||
| metastatic non-small cell lung cancer with BRAF V600E mutation | |||
| metastatic non-small cell lung cancer with BRAF V600E mutation | |||
| chronic graft-versus-host disease | |||
| hepatitis C | |||
| Celator Pharmaceuticals | acute myeloid leukemia | ||
| B-cell precursor acute lymphoblastic leukemia | |||
| tardive dyskinesia | |||
| breast cancer | |||
| mantle cell lymphoma | |||
| Erdheim-Chester disease with BRAF V600 mutation | |||
| ALK-positive, metastatic non-small cell lung cancer | |||
| cytomegalovirus infection | |||
| anaplastic large-cell lymphoma | |||
| hemophilia | |||
| melanoma |
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| Hepatitis C | |||
| HR-positive, HER2-negative breast cancer | |||
| PF Prism | ROS1-positive NSCLC | ||
| CLL with 17p deletion | |||
| renal cell carcinoma | |||
| Parkinson's disease-related psychosis | |||
| small lymphocytic lymphoma (SLL) with 17p deletion | |||
| renal cell carcinoma | |||
| Hodgkin lymphoma | |||
| urothelial carcinoma | |||
| chronic hepatitis C virus genotypes 1, 2, 3, 4, 5 or 6 infection | |||
| Familial mediterranean fever | |||
| Hyper-IgD syndrome | |||
| TNF receptor associated periodic syndrome | |||
| cystic fibrosis with CFTR F508del | |||
| metastatic NSCLC | |||
| soft-tissue sarcoma | |||
| head and neck cancer | |||
| multiple myeloma | |||
| BRCA-mutated ovarian cancer |
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| Waldenstrom’s macroglobulinemia | |||
| ER-positive, HER2-negative breast cancer | |||
| diabetic retinopathy | |||
| cystic fibrosis with a variety of CFTR mutations | |||
| diabetic retinopathy | |||
| PF Prism | lymphangioleiomyomatosis | ||
| PF Prism | lymphangioleiomyomatosis | ||
| cystic fibrosis with CFTR F508del mutation | |||
| Hepatitis C | |||
| Wellstat Therapeutics | hereditary orotic aciduria | ||
| PDL-1 positive NSCLC | |||
| metastatic NSCLC | |||
| reversal of dabigatran | |||
| perinatal/infantile/childhood hypophosphatasia | |||
| EGFR-positive NSCLC | |||
| multiple myeloma | |||
| advanced renal cell carcinoma | |||
| multiple myeloma | |||
| lysosomal acid lipase deficiency | |||
| ALK-mutated NSCLC | |||
| metastatic melanoma |
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| cystic fibrosis with CFTR mutation | |||
| chronic lymphocytic leukemia | |||
| ALK-positive metastatic non-small cell lung cancer | |||
| relapsed chronic lymphocytic leukemia | |||
| chronic lymphocytic leukemia (with 17p deletion | |||
| aplastic anemia | |||
| metastatic melanoma | |||
| Hepatitis C | |||
| idiopathic pulmonary fibrosis | |||
| idiopathic pulmonary fibrosis | |||
| acute lymphoblastic leukemia | |||
| Hepatitis C | |||
| metastatic melanoma | |||
| cystic fibrosis with CFTR R117H mutation |
| Drug | Manufacturer | Indication | |
|---|---|---|---|
| chronic lymphocytic leukemia | |||
| mantle cell lymphoma | |||
| Sofosbuvir[5] | Hepatitis C |