# List of drugs granted breakthrough therapy designation

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.

## 2024

| Drug | Manufacturer | Indication |
| Adagrasib | | In combination with cetuximab for the treatment of adults with KRAS G12C mutated locally advanced or metastatic colorectal cancer who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy |
| Arimoclomol | | In combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in people aged two years of age and older |
| Asciminib | Novartis | Treatment of adults with newly diagnosed Philadelphia chromosomepositive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP) |
| Blinatumomab | | Treatment of CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in people aged one month of age and older |
| Budesonide | | For twelve weeks of treatment in people aged eleven years of age and older with eosinophilic esophagitis |
| Ciltacabtagene autoleucel | Janssen Biotech | Treatment of adults with relapsed or refractory multiple myeloma, who previously received a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 antibody |
| Crinecerfont (capsules) | Neurocrine Biosciences | Adjunctive treatment to glucocorticoid replacement to control androgens in people aged four years of age and older with classic congenital adrenal hyperplasia (CAH) |
| Crinecerfont (solution) | Neurocrine Biosciences | Adjunctive treatment to glucocorticoid replacement to control androgens in people aged four years of age and older with classic congenital adrenal hyperplasia (CAH) |
| Danicopan | | As add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) |
| Donanemab | | Treatment of Alzheimer’s disease |
| Dupilumab | | As an add-on maintenance treatment in adults with inadequately controlled chronic obstructive pulmonary disease (COPD) and an eosinophilic phenotype |
| Durvalumab | Astrazeneca | Treatment of adults with limited stage small cell lung cancer (LS-SCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy (cCRT) |
| Elafibranor | | Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in people unable to tolerate UDCA |
| Epkinly | | Treatment of adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy |
| Fidanacogene elaparvovec | Pfizer | Treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who are receiving routine prophylaxis and without pre-existing neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid detected by an FDA-approved test. |
| Inavolisib | Genentech | In combination with palbociclib and fulvestrant for the treatment of adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth-factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy |
| Nemolizumab | | Treatment of adults with Prurigo Nodularis |
| Nogapendekin alfa inbakicept | | Indicated with Bacillus Calmette-Guérin (BCG) for the treatment of adults with BCG-unresponsive nonmuscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors |
| Olezarsen | Ionis Pharmaceuticals | Adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS) |
| Omalizumab | | Treatment of IgE-mediated food allergy in people aged one year of age and older for the reduction of allergic reactions (Type I), including anaphylaxis, that may occur with accidental exposure to one or more foods. To be used in conjunction with food allergen avoidance |
| Osimertinib | | In combination with pemetrexed and platinum-based chemotherapy for the first-line treatment of adults with locally advanced or metastatic non-small cell lung cancer whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test |
| Osimertinib | | Treatment of adults with locally advanced, unresectable (stage III) non-small cell lung cancer (NSCLC) whose disease has not progressed during or following concurrent or sequential platinum-based chemoradiation therapy and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test |
| Repotrectinib | | Treatment of people aged twelve years of age and older with solid tumors that: have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy |
| Resmetirom | | In conjunction with diet and exercise, for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH) with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis) |
| Revumenib | | Treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in people aged one year of age and older |
| Seladelpar | | Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in people unable to tolerate UDCA |
| Sotatercept | | Treatment of adults with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group 1) to increase exercise capacity, improve WHO functional class (FC), and reduce the risk of clinical worsening events |
| Spesolimab | | Treatment of generalized pustular psoriasis (GPP) in people aged twelve years of age and older and weighing at least 40 kg |
| Tarlatamab | | Treatment of adults with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy |
| Tirzepatide | Eli Lilly | Treatment of moderate to severe obstructive sleep apnea (OSA) in adults with obesity |
| Tovorafenib | | Treatment of people aged six months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation |
| Trastuzumab deruxtecan | | Treatment of adults with unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options |
| Vorasidenib | | Treatment of people aged twelve years of age and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, subtotal resection, or gross total resection |
| Zanidatamab | Jazz Pharmaceuticals | Treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC3+) biliary tract cancer as detected by an FDA approved test |
| Zenocutuzumab | | Treatment of adults with advanced, unresectable or metastatic non-small cell lung cancer harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy; and adults with advanced, unresectable or metastatic pancreatic adenocarcinoma harboring an NRG1 gene fusion with disease progression on or after prior systemic therapy |

## 2023

| Drug | Indication |
| Avacincaptad pegol | To treat geographic atrophy secondary to age-related macular degeneration |
| Cipaglucosidase alfa | To treat late-onset Pompe disease with miglustat |
| Dabrafenib | |
| Dostarlimab | |
| Eflornithine | |
| Elranatamab | To treat relapsed or refractory multiple myeloma after at least four lines of therapy |
| Enfortumab vedotin | |
| Iptacopan | To treat paroxysmal nocturnal hemoglobinuria |
| Ivosidenib | |
| Lecanemab | To treat Alzheimer's disease |
| Miglustat | |
| Nedosiran | To lower urinary oxalate levels in primary hyperoxaluria type 1 and relatively preserved kidney function |
| Nirogacestat | To treat desmoid tumors |
| Pembrolizumab | |
| Sutimlimab | |
| Talquetamab | To treat relapsed or refractory multiple myeloma after at least four therapies |
| Toripalimab | To treat recurrent or metastatic nasopharyngeal carcinoma with or following other therapies |
| Tucatinib | |

## 2022

| 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-resistant prostate 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 Company | 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 |

## 2021

| Drug | Manufacturer | Indication |
| 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 |

## 2020

| Drug | Manufacturer | Indication |
| Avapritinib | Blueprint Medicines Corp | metastatic gastrointestinal stromal tumor |
| Teprotumumab | 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 FGFR2 fusion |
| Sacituzumab govitecan | 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 |
| Pomalidomide | Celgene Corp | AIDS-related Kaposi sarcoma |
| Rucaparib | Clovis Oncology | metastatic castration-resistant prostate 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 | multidrug-resistant HIV-1 |
| Tafasitamab | MorphoSys | relapsed or refractory diffuse large B-cell lymphoma |
| Esketamine | Janssen | major depressive disorder with acute suicidal ideation or behavior |
| belantamab mafodotin | GlaxoSmithKline | refractory multiple myeloma |
| Satralizumab | Genentech | neuromyelitis optica spectrum disorder |
| Pralsetinib | Blueprint Medicines Corp | RET fusion-positive non-small cell lung cancer |
| Pitolisant | Harmony Biosciences | cataplexy in adults with narcolepsy |
| Atoltivimab/maftivimab/odesivimab | Regeneron | Ebola Zaire |
| Lonafarnib | Eiger BioPharmaceuticals | Hutchinson-Gilford progeria syndrome (HGPS), processing-deficient progeroid laminopathies with LMNA or ZMPSTE24 mutations |
| Lumasiran | Alnylam Pharmaceuticals | primary hyperoxaluria type 1 |
| Naxitamab | Y-mAbs Therapeutics | relapsed or refractory high-risk neuroblastoma, used in combination with GM-CSF |
| Setmelanotide | Rhythm Pharmaceuticals | medical obesity caused by proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency |
| Pralsetinib | Blueprint Medicines Corp | RET-mutant medullary thyroid cancer or advanced or metastatic RET fusion-positive thyroid cancer, in radioactive iodine-refractory patients |
| Belimumab | GlaxoSmithKline | active lupus nephritis |
| Osimertinib | AstraZeneca | adjuvant therapy after tumor resection in non-small cell lung cancer with EGFR exon 19 deletions or exon 21 L858R mutations |
| Ansuvimab | Ridgeback Biotherapeutics | Ebola Zaire |

## 2019

| Drug | Manufacturer | Indication |
| Letrozole/ribociclib | Novartis Pharmaceuticals | HR-positive, HER2-negative advanced or metastatic breast cancer |
| Esketamine | Janssen | treatment-resistant depression |
| Dupilumab | Regeneron Pharmaceuticals | atopic dermatitis |
| Brexanolone | Sage Therapeutics | postpartum depression |
| Palbociclib | Pfizer | metastatic breast cancer |
| Erdafitinib | Janssen | urothelial carcinoma |
| Pembrolizumab | Merck Sharp & Dohme | advanced renal cell carcinoma |
| Ado-trastuzumab emtansine | Genentech | HER2-positive early breast cancer |
| Tafamidis meglumine | Foldrx Pharmaceuticals | cardiomyopathy |
| Avelumab | EMD Serono | advanced renal cell carcinoma |
| Venetoclax | AbbVie | chronic lymphocytic leukemiaor small lymphocytic lymphoma |
| Galcanezumab | Eli Lilly | episodic cluster headache |
| Polatuzumab vedotin | Genentech | diffuse large B-cell lymphoma |
| Pexidartinib | Daiichi Sankyo | tenosynovial giant cell tumor |
| Entrectinib | Genentech | solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion |
| Pembrolizumab | Merck | endometrial carcinoma |
| Lenvatinib | Eisai | endometrial carcinoma |
| Elexacaftor/tezacaftor/ivacaftor | Vertex Pharmaceuticals | cystic fibrosis |
| Zanubrutinib | BeiGene | mantle cell lymphoma |
| Crizanlizumab | Novartis | sickle cell disease |
| Givosiran | Alnylam Pharmaceuticals | acute hepatic porphyria |
| Acalabrutinib | AstraZeneca | relapsed or refractory chronic lymphocytic leukemia |
| Acalabrutinib | AstraZeneca | untreated chronic lymphocytic leukemia |
| Voxelotor | Global Blood Therapeutics | sickle cell disease |
| Enfortumab vedotin | Astellas Pharma | metastatic urothelial carcinoma |
| Fam-trastuzumab deruxtecan | Daiichi Sankyo | HER2-positive breast cancer |

## 2018

| Drug | Manufacturer | Indication |
| Tezacaftor/ivacaftor | Vertex Pharmaceuticals | cystic fibrosis |
| Durvalumab | AstraZeneca UK | unresectable Stage III non-small cell lung cancer |
| Ibalizumab | 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 | 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 | Kyowa Hakko Kirin | mycosis fungoides or Sézary syndrome |
| Patisiran | Alnylam Pharmaceuticals | hereditary transthyretin-mediated amyloidosis |
| Cenegermin | Dompe Farmaceutici | neurotrophic keratitis |
| Lanadelumab | Takeda Pharmaceuticals | hereditary angioedema |
| Cemiplimab | Regeneron Pharmaceuticals | cutaneous squamous cell carcinoma |
| Amikacin liposome inhalation suspension | Insmed | mycobacterial lung disease |
| Emicizumab | 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 | 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 | Stemline Therapeutics | blastic plasmacytoid dendritic cell neoplasm |
| Balovaptan | Roche | Autism spectrum |

## 2017

| Drug | Manufacturer | Indication |
| Nivolumab | Bristol-Myers Squibb | urothelial carcinoma |
| Ribociclib | Novartis Pharmaceuticals | HR-positive, HER2-negative breast cancer |
| Pembrolizumab | Merck Sharp & Dohme | Hodgkin lymphoma |
| Avelumab | EMD Serono | metastatic Merkel cell carcinoma |
| Niraparib | GlaxoSmithKline | 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 |
| Ranibizumab | Genentech | diabetic retinopathy |
| Cerliponase alfa | BioMarin Pharmaceutical | tripeptidyl peptidase 1 (TPP1) deficiency |
| Midostaurin | Novartis Pharmaceuticals | FLT3-positive acute myeloid leukemia |
| Brigatinib | Takeda Pharmaceuticals | ALK-positive non-small cell lung cancer |
| Durvalumab | AstraZeneca UK | metastatic urothelial carcinoma |
| Letrozole/ribociclib | Novartis Pharmaceuticals | breast cancer |
| Pembrolizumab | Merck Sharp & Dohme | urothelial carcinoma |
| Tocilizumab | Genentech | giant cell arteritis |
| Pembrolizumab | Merck Sharp & Dohme | microsatellite instability-high tumors |
| Ceritinib | Novartis Pharmaceuticals | ALK-positive, metastatic non-small cell lung cancer |
| Dabrafenib | Novartis Pharmaceuticals | metastatic non-small cell lung cancer with BRAF V600E mutation |
| Trametinib | Novartis Pharmaceuticals | metastatic non-small cell lung cancer with BRAF V600E mutation |
| Ibrutinib | Pharmacyclics | chronic graft-versus-host disease |
| Glecaprevir/pibrentasvir | AbbVie | hepatitis C |
| Cytarabine/daunorubicin | Celator Pharmaceuticals | acute myeloid leukemia |
| Inotuzumab ozogamicin | Wyeth Pharmaceuticals | B-cell precursor acute lymphoblastic leukemia |
| Deutetrabenazine | Teva Pharmaceuticals | tardive dyskinesia |
| Abemaciclib | Eli Lilly | breast cancer |
| Acalabrutinib | AstraZeneca UK | mantle cell lymphoma |
| Vemurafenib | Hoffmann-La Roche | Erdheim-Chester disease with BRAF V600 mutation |
| Alectinib | Hoffmann-La Roche | ALK-positive, metastatic non-small cell lung cancer |
| Letermovir | Merck Sharp & Dohme | cytomegalovirus infection |
| Brentuximab vedotin | Seattle Genetics | anaplastic large-cell lymphoma |
| Emicizumab | Genentech | hemophilia |
| Nivolumab | Bristol-Myers Squibb | melanoma |

## 2016

| Drug | Manufacturer | Indication |
| Elbasvir/grazoprevir | Merck Sharp & Dohme | Hepatitis C |
| Palbociclib | Pfizer | HR-positive, HER2-negative breast cancer |
| Crizotinib | PF Prism | ROS1-positive NSCLC |
| Venetoclax | AbbVie | CLL with 17p deletion |
| Cabozantinib | Exelixis | renal cell carcinoma |
| Pimavanserin | Acadia Pharmaceuticals | Parkinson's disease-related psychosis |
| Ibrutinib | Pharmacyclics | small lymphocytic lymphoma (SLL) with 17p deletion |
| Lenvatinib | Eisai | renal cell carcinoma |
| Nivolumab | Bristol-Myers Squibb | Hodgkin lymphoma |
| Atezolizumab | Genentech | urothelial carcinoma |
| Sofosbuvir/velpatasvir | Gilead Sciences | chronic hepatitis C virus genotypes 1, 2, 3, 4, 5 or 6 infection |
| Canakinumab | Novartis | Familial mediterranean fever |
| Canakinumab | Novartis | Hyper-IgD syndrome |
| Canakinumab | Novartis | TNF receptor associated periodic syndrome |
| Lumacaftor/ivacaftor | Vertex Pharmaceuticals | cystic fibrosis with CFTR F508del |
| Atezolizumab | Genentech | metastatic NSCLC |
| Olaratumab | Eli Lilly | soft-tissue sarcoma |
| Nivolumab | Bristol-Myers Squibb | head and neck cancer |
| Daratumumab | Janssen | multiple myeloma |
| Rucaparib | Clovis Oncology | BRCA-mutated ovarian cancer |

## 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 |
| Sirolimus | PF Prism | lymphangioleiomyomatosis |
| Lumacaftor/ivacaftor | Vertex Pharmaceuticals | cystic fibrosis with CFTR F508del mutation |
| Ombitasvir/paritaprevir/ritonavir | AbbVie | Hepatitis C |
| Uridine triacetate | Wellstat Therapeutics | hereditary orotic aciduria |
| Pembrolizumab | Merck Sharp & Dohme | PDL-1 positive NSCLC |
| Nivolumab | Bristol-Myers Squibb | metastatic NSCLC |
| Idarucizumab | Boehringer Ingelheim | reversal of dabigatran |
| Asfotase alfa | Alexion Pharmaceuticals | perinatal/infantile/childhood hypophosphatasia |
| Osimertinib | AstraZeneca | EGFR-positive NSCLC |
| Daratumumab | Janssen Biotech | multiple myeloma |
| Nivolumab | Bristol-Myers Squibb | advanced renal cell carcinoma |
| Elotuzumab | Bristol-Myers Squibb | multiple myeloma |
| Sebelipase alfa | Alexion Pharmaceuticals | lysosomal acid lipase deficiency |
| Alectinib | Hoffmann-La Roche | ALK-mutated NSCLC |
| Pembrolizumab | Merck Sharp & Dohme | metastatic melanoma |

## 2014

| Drug | Manufacturer | Indication |
| Ivacaftor | Vertex Pharmaceuticals | cystic fibrosis with CFTR mutation |
| Ofatumumab | Novartis Pharmaceuticals | chronic lymphocytic leukemia |
| Ceritinib | Novartis Pharmaceuticals | ALK-positive metastatic non-small cell lung cancer |
| Idelalisib | Gilead Sciences | relapsed chronic lymphocytic leukemia |
| Ibrutinib | Pharmacyclics | chronic lymphocytic leukemia (with 17p deletion |
| Eltrombopag | Novartis Pharmaceuticals | aplastic anemia |
| Pembrolizumab | Merck | metastatic melanoma |
| Ledipasvir/sofosbuvir | Gilead Sciences | Hepatitis C |
| Pirfenidone | Genentech | idiopathic pulmonary fibrosis |
| Nintedanib | Boehringer Ingelheim | idiopathic pulmonary fibrosis |
| Blinatumomab | Amgen | acute lymphoblastic leukemia |
| Ombitasvir/paritaprevir/ritonavir/dasabuvir | 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 Sciences | Hepatitis C |