In October there were 7 new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 7 supplemental drug approvals. The FDA had acted on assignment of 3 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 |
| BEOVU® (brolucizumab-dbll) | Novartis | Intravitreal | Treatment of neovascular age-related macular degeneration. | 10/07/2019 |
| Scenesse® (afamelanotide) | Clinuvel | Subcutaneous | To increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP) | 10/07/2019 |
| Fluorodopa (FDOPA) | The Feinstein Institute for Medical Research | Intravenous | A radioactive diagnostic agent indicated for use in positron emission tomography (PET) to visualize dopaminergic nerve terminals in the striatum for the evaluation of adult patients with suspected Parkinsonian syndromes (PS). | 10/10/2019 |
| VUMERITY™ (diroximel fumarate) | Alkermes | Oral | Treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. | 10/11/2019 |
| Trikafta™ (elexacaftor, tezacaftor and ivacaftor) | Vertex | Oral | treatment of cystic fibrosis in patients 12 years and older who have at least one F508del mutation in the CFTR gene. | 10/21/2019 |
| VUMERITY™ (diroximel fumarate) | Alkermes | Oral | Treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. | 10/29/2019 |
| OXERVATE™ (cenegermin-bkbj) | Dompé farmaceutici | Ophthalmic | Treatment of neurotrophic keratitis. | 10/30/2019 |
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 |
| Fasenra® (benralizumab) | AstraZeneca | Subcutaneous | The addition of a single use auto-injector presentation for patient and/or caregiver administration in a non-clinical setting. | 10/03/2019 |
| WILATE (von Willebrand Factor/ Coagulation Factor VIII Complex (Human)) | Octapharma | Intravenous | Treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on demand treatment and control of bleeding episodes. | 10/08/2019 |
| Botox® (onabotulinumtoxinA) | Allergan | Intramuscular | Treatment of lower limb spasticity in pediatric patients 2 to 17 years of age, excluding spasticity caused by cerebral palsy. | 10/18/2019 |
| Erelzi™ (etanercept-szzs) | Sandoz | Subcutaneous | Addition of psoriatic arthritis and adult plaque psoriasis indications. | 10/18/2019 |
| STELARA® (ustekinumab) | Janssen | Subcutaneous | Addition of the indication of treatment of adults with moderately to severely active ulcerative colitis | 10/18/2019 |
| ULTOMIRIS® (ravulizumab-cwvz) | Alexion | Intravenous | Treatment of adults and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). | 10/18/2019 |
| Zejula® (niraparib) | TESARO | Oral | Treatment of adult patients with advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with three or more prior chemotherapy regimens and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either: a deleterious or suspected deleterious BRCA mutation, or genomic instability and who have progressed more than six months after response to the last platinum-based chemotherapy.
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10/23/2019 |
Specialty Drug FDA Actions – New and supplemental FDA filings in the previous month
| Drug Name | Manufacturer | Type of Application | Indication | Route | PDUFA Date |
| UX007 (triheptanoin) | Ultragenyx Pharmaceutical | NDA | Treatment of long-chain fatty acid oxidation disorders (LC-FAOD) | Oral | 07/31/2020 |
| Mirena® (levonorgestrel-releasing intrauterine system) | Bayer | sNDA | Extend the indication to up to six years of pregnancy prevention based on results from a Phase 3 extension trial evaluating the efficacy and safety of Mirena. | Intrauterine | Not Reported |
| ROLONTIS (eflapegrastim) | Spectrum | BLA | Treatment of neutropenia due to myelosuppressive chemotherapy | Subcutaneous | Not Reported |
Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q3-2020
| Drug Name | Manufacturer | Indication | Route | PDUFA Date |
| Ofev® (Nintedanib) | Boehringer Ingelheim | Interstitial Lung Diseases (Diffuse Parenchymal Lung Disease) | Oral | 11/20/2019 |
| Exservan (riluzole) | Aquestive Therapeutics | Amyotrophic Lateral Sclerosis | Oral | 11/30/2019 |
| RediTrex (methotrexate) | Cumberland Pharmaceuticals | Juvenile Rheumatoid Arthritis; Psoriasis; Psoriatic Arthritis; Rheumatoid Arthritis | Intramuscular | 12/1/2019 |
| luspatercept | Celgene | Thalassemia | Subcutaneous | 12/4/2019 |
| RVT-802 | Enzyvant Sciences | DiGeorge Syndrome | Injection | 12/5/2019 |
| Rituxan® (rituximab) | Genentech | Microscopic Polyangiitis (MPA) | Intravenous | 12/11/2019 |
| infliximab biosimilar | Amgen | Rheumatoid Arthritis | Intravenous | 12/14/2019 |
| AV-001 | Avadel Pharmaceuticals | Unspecified | Parenteral | 12/15/2019 |
| Vumerity™ (diroximel fumarate DR) | Biogen | Relapsing Remitting Multiple Sclerosis (RRMS) | Oral | 12/25/2019 |
| Xtandi® (enzalutamide) | Astellas | Hormone-Sensitive Prostate Cancer | Oral | 12/31/2019 |
| Botox® (onabotulinumtoxinA) | Allergan | Lower Limb Muscle Spasticity | Intramuscular | 1/7/2020 |
| crizanlizumab | Novartis | Vaso-Occlusive Crisis Associated With Sickle Cell Disease | Intravenous | 1/15/2020 |
| tazemetostat hydrobromide | Epizyme | Soft Tissue Sarcoma | Oral | 1/23/2020 |
| givosiran | Alnylam Pharmaceuticals | Acute Intermittent Porphyria | Subcutaneous | 2/4/2020 |
| avapritinib | Blueprint Medicines | Gastrointestinal Stromal Tumor (GIST) | Oral | 2/14/2020 |
| eptinezumab | Alder Biopharmaceuticals | Migraine | Subcutaneous | 2/21/2020 |
| voxelotor | Global Blood Therapeutics | Sickle Cell Disease | Oral | 2/26/2020 |
| zanubrutinib | BeiGene | Mantle Cell Lymphoma | Oral | 2/27/2020 |
| teprotumumab | Horizon Therapeutics | Graves’ Ophthalmopathy | Intravenous | 3/8/2020 |
| enfortumab vedotin | Astellas Pharma | Metastatic Transitional (Urothelial) Tract Cancer | Intravenous | 3/15/2020 |
| Trikafta™ ((elexacaftor + ivacaftor + tezacaftor) + ivacaftor) | Vertex Pharmaceuticals | Cystic Fibrosis | Oral | 3/19/2020 |
| ozanimod | Celgene | Relapsing Multiple Sclerosis (RMS) | Oral | 3/25/2020 |
| trastuzumab deruxtecan | AstraZeneca | Metastatic Breast Cancer | Intravenous | 3/29/2020 |
| neratinib | Puma Biotechnology | Metastatic Breast Cancer | Oral | 4/30/2020 |
| isatuximab | Sanofi | Refractory Multiple Myeloma; Relapsed Multiple Myeloma | Intravenous | 4/30/2020 |
| Keytruda® (pembrolizumab) | Merck | Squamous Cell Carcinoma | Intravenous | 6/29/2020 |
| abicipar pegol | Allergan | Wet (Neovascular / Exudative) Macular Degeneration | Intravitreal | 7/9/2020 |
| triheptanoin | Ultragenyx | Fatty Acid Biosynthesis Disorders | Oral | 7/31/2020 |
| satralizumab | F. Hoffmann-La Roche | Neuromyelitis Optica (Devic’s Syndrome) | Subcutaneous | 8/28/2020 |
View our full pipeline library here:
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 November 1, 2019. https://www.fda.gov/downloads/…/CDER/ReportsBudgets/UCM591976.pdf
Pharma Intelligence Center Web Site. Accessed November 1, 2019. https://pharma.globaldata.com
U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed November 1, 2019. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm
U.S. Food and Drug Administration. Novel Drug Approvals for 2019. Accessed November 1, 2019. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm629491.htm
