In June there were four new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 12 supplemental drug approvals. The FDA had acted on assignment of six 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 |
| NUCALA (mepolizumab) | GlaxoSmithKline | Subcutaneous | Add-on maintenance treatment of patients with severe asthma aged 12 years and older, and with an eosinophilic phenotype; 2)treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA) | 06/06/2019 |
| POLIVY™ (polatuzumab vedotin-piiq) | Genentech | Intravenous | Indicated in combination with bendamustine and a rituximab product for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified, after at least two prior therapies. | 06/10/2019 |
| KANJINTI™ (trastuzumab-anns) – Biosimilar to Herceptin | Amgen | Intravenous | – Adjuvant treatment of HER2 overexpressing node positive or node negative (ER/PR negative or with one high risk feature) breast cancer.
– In combination with paclitaxel for first-line treatment of HER2-overexpressing metastatic breast cancer. – In combination with cisplatin and capecitabine or 5-fluorouracil, for the treatment of patients with HER2 overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma who have not received prior treatment for metastatic disease. |
06/13/2019 |
| Zirabev™ (bevacizumab-bvzr) | Pfizer | Intravenous | Treatment of metastatic colorectal cancer, locally advanced, recurrent or metastatic non-squamous non-small cell lung cancer, recurrent glioblastoma in adults, metastatic renal cell carcinoma, and persistent, recurrent, or metastatic cervical cancer. | 06/27/2019 |
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 |
| EMGALITY® (galcanezumab-gnlm) | Eli Lilly | Subcutaneous | Treatment of episodic cluster headache in adults. | 06/04/2019 |
| KEYTRUDA® (pembrolizumab) | Merck | Intravenous | Treatment of patients with recurrent or metastatic head and
neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum containing chemotherapy |
06/10/2019 |
| KEYTRUDA® (pembrolizumab) | Merck | Intravenous | The treatment of patients with metastatic small cell lung cancer with disease progression
on or after platinum-based chemotherapy and at least one other prior line of therapy. |
06/17/2019 |
| INFLECTRA® (infliximab-dyyb) | Celltrion | Intravenous | For reducing signs and symptoms and inducing and maintaining
clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy. |
06/18/2019 |
| Botox® (onabotulinumtoxinA) | Allergan | Intramuscular | Treatment of upper limb spasticity in pediatric patients 2 to 17
years of age. |
06/20/2019 |
| DOPTELET® (avatrombopag) | Dova Pharmaceuticals | Oral | Treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia
who have had an insufficient response to a previous treatment. |
06/26/2019 |
| Renflexis® (infliximab-abda) | Samsung Bioepis | Intravenous | Reducing signs and symptoms and inducing and maintaining clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy. | 06/26/2019 |
| Dupixent® (dupilumab) | Regeneron | Subcutaneous | Use of dupilumab as add-on maintenance treatment in adult patients with inadequately
controlled chronic rhinosinusitis with nasal polyposis. |
06/26/2019 |
| SOLIRIS® (eculizumab) | Alexion | Intravenous | The treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive | 06/27/2019 |
| DARZALEX® (daratumumab) | Janssen | Intravenous | Treatment of adult patients with multiple myeloma in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant. | 06/27/2019 |
Specialty Drug FDA Actions – New and supplemental FDA filings in the previous month
| Drug Name | Manufacturer | Type of Application | Indication | Route | PDUFA Date |
| EDSIVO™ (celiprolol) | Acer | NDA | vascular Ehlers-Danlos syndrome (vEDS) | Oral | Complete Response Letter |
| ZEJULA® (niraparib) | GlaxoSmithKline | sNDA | Treatment of advanced ovarian, fallopian tube, or primary peritoneal cancer | Oral | 10/24/2019 |
| ULTOMIRIS® (ravulizumab-cwvz) | Alexion | sBLA | Treatment of people with atypical hemolytic uremic syndrome (aHUS) in order to inhibit complement-mediated thrombotic microangiopathy (TMA). | Intravenous | 10/19/2019 |
| Rituxan® (rituximab) | Genentech | sBLA | In combination with glucocorticoids (GCC), for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children two years of age and older. | Intravenous | No Date Provided |
| RVT-802 | Enzyvant | BLA | Treatment of pediatric congenital athymia | Intravenous | 12/2019 |
| SCENESSE® (afamelanotide) | Clinuvel | NDA | Prevention of phototoxicity and anaphylactoid reactions in adult patients with erythropoietic protoporphyria (EPP) | Injectable | 10/06/2019 |
Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q1-2020
| Drug Name | Manufacturer | Indication | Route | PDUFA Date |
| Vyndaqel (tafamidis meglumine) | Pfizer | Familial Amyloid Cardiomyopathy | Oral | 7/1/2019 |
| rituximab biosimilar | Pfizer | Follicular Lymphoma | Intravenous | 7/1/2019 |
| Emflaza (deflazacort) | PTC Therapeutics | Duchenne Muscular Dystrophy | Oral | 7/4/2019 |
| Xpovio (selinexor) | Karyopharm Therapeutics | Refractory Multiple Myeloma | Oral | 7/7/2019 |
| Otezla (apremilast) | Celgene | Behcet Disease | Oral | 7/21/2019 |
| riluzole | Biohaven | Amyotrophic Lateral Sclerosis | Sublingual | 7/21/2019 |
| pexidartinib hydrochloride | Plexxikon | Tenosynovial Giant Cell Tumor | Oral | 8/3/2019 |
| entrectinib | F. Hoffmann-La Roche | Non-Small Cell Lung Cancer | Oral | 8/18/2019 |
| upadacitinib tartrate | AbbVie | Rheumatoid Arthritis | Oral | 8/19/2019 |
| Polivy (polatuzumab vedotin) | Genentech | Diffuse Large B-Cell Lymphoma | Intravenous | 8/19/2019 |
| golodirsen | Sarepta Therapeutics | Duchenne Muscular Dystrophy | Intravenous | 8/19/2019 |
| quizartinib | Daiichi Sankyo | Refractory Acute Myeloid Leukemia | Oral | 8/25/2019 |
| fedratinib | Celgene | Myelofibrosis | Oral | 9/3/2019 |
| Emgality (galcanezumab) | Eli Lilly | Cluster Headache Syndrome | Subcutaneous | 9/5/2019 |
| Botox (onabotulinumtoxinA) | Allergan | Upper Limb Muscle Spasticity | Intramuscular | 9/8/2019 |
| AV-001 | Avadel Pharmaceuticals | Unspecified | Parenteral | 9/15/2019 |
| Darzalex (daratumumab) | Genmab | Multiple Myeloma (Kahler Disease) | Intravenous | 9/26/2019 |
| Scenesse (afamelanotide acetate) | Clinuvel Pharmaceuticals | Porphyria (Erythropoietic Protoporphyri) | Subcutaneous | 10/6/2019 |
| teriparatide | Pfenex | Osteoporosis | Subcutaneous | 10/7/2019 |
| brolucizumab | Novartis | Wet (Neovascular / Exudative) Macular Degeneration | Intravitreal | 10/15/2019 |
| Ultomiris (ravulizumab) | Alexion | Atypical Hemolytic Uremic Syndrome | Intravenous | 10/19/2019 |
| Zejula (niraparib) | TESARO | Epithelial Ovarian Cancer; Fallopian Tube Cancer; Peritoneal Cancer | Oral | 10/24/2019 |
| darolutamide | Orion Corp | Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer | Oral | 10/29/2019 |
| Ofev (nintedanib) | Boehringer Ingelheim | Interstitial Lung Diseases | 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 |
| Vumerity (diroximel fumarate) | Alkerme | Relapsing Remitting Multiple Sclerosis (RRMS) | Oral | 12/25/2019 |
| eptinezumab | Alder Biopharmaceuticals | Migraine | Subcutaneous | 2/21/2020 |
| ozanimod | Celgene | Relapsing Multiple Sclerosis (RMS) | Oral | 3/25/2020 |
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 July 1, 2019. https://pharma.globaldata.com
U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed July 1, 2019. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm
U.S. Food and Drug Administration. Novel Drug Approvals for 2019. Accessed July 1, 2019. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm629491.htm
