In August there were 4 new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 2 supplemental drug approvals. The FDA had acted on assignment of 5 new product reviews.
Drugs included in this review are generally regarded by the industry as specialty drugs. Qualifiers may include anticipated administration by a professional, distribution by a specialty pharmacy or submission and reimbursement under the medical claim or Medicare Part B.
New Specialty Drug Approvals – New FDA drug approvals in the previous month
| Drug Name | Manufacturer | Route of Administration | Indication | Approval Date |
| TURALIO™ (pexidartinib) | Daiichi | Oral | Treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. | 08/02/2019 |
| Rozlytrek™ (entrectinib) | Genentech | Oral | Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion with a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. | 08/15/2019 |
| Rinvoq™ (upadacitinib) | AbbVie | Oral | treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate. | 08/16/2019 |
| INREBIC® (fedratinib) | Celgene | Oral | Treatment of adult patients with intermediate-2 or high-risk primary or secondary (postpolycythemia vera or post-essential thrombocythemia) myelofibrosis (MF). | 08/16/2019 |
New Specialty Supplemental Indication Updates – Approved updated supplemental indications on currently marketed drugs in the previous month
| Drug Name | Manufacturer | Route of Administration | Indication | Approval Date |
| Myobloc® (rimabotulinumtoxinB) | Solstice Neurosciences | Intramuscular | Treatment of sialorrhea in adults. | 08/20/2019 |
| Taltz® (Ixekizumab) | Eli Lilly | Subcutaneous | Treatment of adults with ankylosing spondylitis | 08/23/2019 |
Specialty Drug FDA Actions – New and supplemental FDA filings in the previous month
| Drug Name | Manufacturer | Type of Application | Indication | Route | PDUFA Date |
| Givosiran | Alnylam Pharmaceuticals | NDA | Treatment of acute hepatic porphyria (AHP) | Subcutaneous | 02/04/2020 |
| Avapritinib | Blueprint Medicines | NDA | Treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. | Oral | 02/14/2020 |
| VX-445 (elexacaftor) | Vertex | NDA | In combination with tezacaftor and ivacaftor for the treatment of cystic fibrosis. | Oral | 03/19/2020 |
| XTANDI® (enzalutamide) | Astellas | sNDA | Treatment of men with metastatic hormone-sensitive prostate cancer (mHSPC). | Oral | Q4 2019 |
| Inebilizumab | Viela Bio | BLA | Treatment of patients with neuromyelitis optica spectrum disorder (NMOSD) | Intravenous | Not Reported |
Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q2-2020
| Drug Name | Manufacturer | Indication | Route | PDUFA Date |
| Inrebic® (fedratinib) | Impact Biomedicines | Post-Polycythemia Vera Myelofibrosis (PPV-MF) | Oral | 9/3/2019 |
| Emgality® (galcanezumab) | Eli Lilly | Cluster Headache Syndrome (Cluster Headache) | Subcutaneous | 9/5/2019 |
| Botox® (onabotulinumtoxinA) | Allergan | Upper Limb Muscle Spasticity | Intramuscular | 9/8/2019 |
| Darzalex® (daratumumab) | Genmab | Multiple Myeloma (Kahler Disease) | Intravenous | 9/26/2019 |
| Brolucizumab | Novartis | Wet (Neovascular / Exudative) Macular Degeneration | Intravitreal | 10/15/2019 |
| Ultomiris™ (ravulizumab) | Alexion Pharmaceuticals | Atypical Hemolytic Uremic Syndrome | Intravenous | 10/19/2019 |
| Zejula® (niraparib) | TESARO | Epithelial Ovarian Cancer | Oral | 10/24/2019 |
| Nubeqa™ (darolutamide) | Bayer | Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer | Oral | 10/28/2019 |
| Ofev® (Ofev) | Boehringer Ingelheim | Interstitial Lung Diseases (Diffuse Parenchymal Lung Disease) | Oral | 11/20/2019 |
| Exservan (riluzole) | Aquestive Therapeutics | Amyotrophic Lateral Sclerosis | Oral | 11/30/2019 |
| Luspatercept | Celgene | Thalassemia | Subcutaneous | 12/4/2019 |
| RVT-802 | Enzyvant Sciences | DiGeorge Syndrome | Intravenous | 12/5/2019 |
| Rituxan® (rituximab) | Genentech | Granulomatosis with Polyangiitis (Wegener’s Granulomatosis) | Intravenous | 12/11/2019 |
| Infliximab biosimilar | Amgen | Rheumatoid Arthritis | Intravenous | 12/14/2019 |
| Vumerity™ (diroximel fumarate) | Alkermes | Relapsing Remitting Multiple Sclerosis (RRMS) | Oral | 12/25/2019 |
| Tazemetostat | Epizyme | Soft Tissue Sarcoma | Oral | 1/23/2020 |
| Avapritinib | Blueprint Medicines | Gastrointestinal Stromal Tumor (GIST) | Oral | 2/14/2020 |
| Keytruda® (pembrolizumab) | Merck | Hepatocellular Carcinoma | Intravenous | 2/18/2020 |
| Eptinezumab | Alder Biopharmaceuticals | Migraine | Intramuscular | 2/21/2020 |
| Zanubrutinib | BeiGene | Mantle Cell Lymphoma | Oral | 2/27/2020 |
| Elexacaftor | Vertex | Cystic Fibrosis | Oral | 3/19/2020 |
| Ozanimod | Celgene | Relapsing Multiple Sclerosis (RMS) | Oral | 3/25/2020 |
| Isatuximab | Sanofi | Refractory Multiple Myeloma | Intravenous | 4/30/2020 |
Terms & Abbreviations:
- aNDA: Abbreviated New Drug Application
- BLA: Biologics License Application
- FDA: Food and Drug Administration
- NDA: New Drug Application
- sBLA: supplemental Biologics License Application
- sNDA: supplemental New Drug Application
- PDUFA: Prescription Drug User Fee Act: a law passed in 1992 which allowed the Food and Drug Administration (FDA) to collect fees from drug manufacturers to fund the new drug approval process. By collecting the fee, the FDA is required to meet benchmarks related to the speed of the NDA/BLA review process
- PDUFA Date: deadlines for the FDA to approve new drugs, typically 10 months from NDA/BLA completions, drugs selected for priority review may be allotted 6 months
- sBLA: supplemental Biologics License Application
- sNDA: supplemental New Drug Application
References:
U.S. Food and Drug Administration. Center for Drug Evaluation and Research Advancing Health Through Innovation 2017 New Drug Therapy Approvals. Downloaded June 29, 2018. https://www.fda.gov/downloads/…/CDER/ReportsBudgets/UCM591976.pdf
Pharma Intelligence Center Web Site. Accessed September 1, 2019. https://pharma.globaldata.com
U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed September 1, 2019. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm
U.S. Food and Drug Administration. Novel Drug Approvals for 2019. Accessed September 1, 2019. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm629491.htm
