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 |
| 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 | |
|---|---|---|---|
| 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 |