May 2018 Specialty Drug FDA Actions
In May there were a total of five new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were eight supplemental drug approvals. The FDA had assigned eight new products for review and assigned five potential specialty products with June 2018 PDUFA dates. Finally, the FDA shifted the PDUFA dates for two products to later this year.
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 – May 2018 FDA drug approvals
Drug Name | Manufacturer | Route of Administration | Indication | Approval Date |
AndexXa®
(andexanet alfa) |
Portola Pharmaceuticals Inc | Intravenous | Antidote indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. | May 3, 2018 |
Retacrit™
(epoetin alfa-epbx) Biosimilar for Epogen/Procrit |
Hospira | Injection |
Treatment of anemia due to: Chronic Kidney Disease (CKD) in patients on dialysis and not on dialysis, Zidovudine in HIV-infected patients, the effects of concomitant myelosuppressive chemotherapy, and upon initiation, there is a minimum of two additional months of planned chemotherapy, Reduction of allogeneic red blood cell (RBC) transfusions in patients undergoing elective, noncardiac, nonvascular surgery. |
May 15, 2018 |
Aimovig™
(erenumab-aooe) |
Amgen | Subcutaneous | Preventive treatment of migraine in adults. | May 17, 2018 |
YONSA® (abiraterone acetate) |
Sun Pharma | Oral |
In combination with methylprednisolone for the treatment of patients with metastatic castration resistant prostate cancer (CRPC) |
May 22, 2018 |
PALYNZIQ™ (pegvaliase-pqpz) | BioMarin Pharmaceutical | Subcutaneous |
Treatment of adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. |
May 24, 2018 |
May 2018 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 |
KYMRIAH™ (tisagenlecleucel) | Novartis | Intravenous | Treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. | May 1, 2018 |
Tafinlar®
(dabrafenib) |
GlaxoSmithKline | Oral | Administered in combination with Mekinist (trametinib) for the treatment of anaplastic thyroid cancer (ATC) that cannot be removed by surgery or has spread to other parts of the body (metastatic), and has a type of abnormal gene, BRAF V600E (BRAF V600E mutation-positive). | May 4, 2018 |
Mekinist® (trametinib) | Novartis | Oral | Administered in combination with Tafinlar (dabrafenib) for the treatment of anaplastic thyroid cancer (ATC) that cannot be removed by surgery or has spread to other parts of the body (metastatic), and has a type of abnormal gene, BRAF V600E (BRAF V600E mutation-positive). | May 4, 2018 |
DARZALEX® (daratumumab) | Genmab / Janssen Biotech | Intravenous | In combination with bortezomib, melphalan and prednisone (VMP) for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). | May 7, 2018 |
Gilenya®
(fingolimod) |
Novartis | Oral | Treatment of children and adolescents 10 to less than 18 years of age with relapsing forms of multiple sclerosis (RMS). | May 11, 2018 |
Actemra® (tocilizumab) | Genentech | Subcutaneous | Treatment of active polyarticular juvenile idiopathic arthritis (PJIA) in patients two years of age and older with the subcutaneous (SC) formulation of Actemra (tocilizumab). | May 14, 2018 |
Prolia®
(denosumab) |
Amgen | Subcutaneous | Treatment of glucocorticoid-induced osteoporosis (GIOP) in men and women at high risk of fracture | May 18, 2018 |
XELJANZ® (tofacitinib) | Pfizer | Oral | Treatment of adult patients in the U.S. with moderately to severely active ulcerative colitis (UC). | May 30, 2018 |
Specialty Drug FDA Actions – New and supplemental FDA filings in the previous month
Drug Name | Manufacturer | Type of Application | Indication | Route | PDUFA Date |
TECENTRIQ® (atezolizumab) | Genentech | sBLA | In combination with Avastin (bevacizumab), paclitaxel and carboplatin (chemotherapy), for the initial (first-line) treatment of people with metastatic non-squamous non-small cell lung cancer (NSCLC). | Intravenous | September 5, 2018 |
Emapalumab | Novimmune | BLA | Treat patients with primary Hemophagocytic Lymphohistiocytosis (HLH), | Injectable | November 20, 2018 |
Larotrectinib | Bayer | NDA | Treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion | Oral | November 26, 2018 |
Amifampridine phosphate | Catalyst Pharmaceuticals, Inc. | NDA | Treatment of Lambert-Eaton myasthenic syndrome (LEMS) | Oral | November 28, 2018 |
Gilteritinib fumarate | Astellas Pharma Inc. | NDA | Treatment of adult patients who have relapsed or refractory (resistant to treatment) Acute Myeloid Leukemia (AML) with a FLT3 mutation. | Oral | November 29, 2018 |
Rituximab biosimilar1 | Celltrion | BLA | Follicular Lymphoma, Rheumatoid Arthritis, Non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia (CLL), Granulomatosis with Polyangiitis (Wegener Polyangiitis), Microscopic Polyangiitis (MPA) | Intravenous | 4th Quarter 2018 |
Coagadex® (Coagulation Factor X, Human) | Bio Products Laboratory Limited | sBLA | Treatment of Hereditary Factor X Deficiency | Intravenous | 4th Quarter 2018 |
CABOMETYX® (cabozantinib) | Exelixis, Inc. | NDA | Treatment for patients with previously treated advanced hepatocellular carcinoma (HCC) | Oral | January 14, 2019 |
- Resubmission to FDA completed and is expected to have a determination within 6 months.
Looking Ahead – Anticipated specialty drug FDA actions for 2018 and other significant changes.
Drug Name | Manufacturer | Indication | Route | PDUFA Date |
Pegfilgrastim biosimilar | Biocon Ltd | Chemotherapy Induced Neutropenia | Subcutaneous | June 4, 2018 |
CINRYZE®
(C1 esterase inhibitor [human]) |
Shire | Supplemental Biologics License Application (sBLA) to expand the currently approved indication to include children aged 6 years and older with hereditary angioedema (HAE). | Intravenous | June 20, 2018 |
Keytruda®
(pembrolizumab) |
Merck | Supplemental indication for cervical cancer | Intravenous | June 28, 2018 |
Xeljanz®
(tofacitinib citrate) |
Pfizer | Supplemental indication for
Ulcerative Colitis |
Oral | June 29, 2018 |
COMBO450
(binimetinib and encorafenib) |
Array BioPharma Inc | Treatment of patients with BRAF-mutant advanced, unresectable or metastatic melanoma. | Oral | June 30, 2018 |
Xtandi®
(enzalutamide) |
Pfizer | Supplemental indication for Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer | Oral | July 2, 2018 |
Keytruda®
(pembrolizumab) |
Merck | Supplemental indication for Primary Mediastinal B-Cell Lymphoma | Intravenous | July 3, 2018 |
Poteligeo
(mogamulizumab)1 |
Kyowa Hakko Kirin Co Ltd | Cutaneous T-Cell Lymphoma | Intravenous | September 4, 2018 |
Fremanezumab2 | Teva | Migraine | Intravenous | September 16, 2018 |
- FDA extended review period from June 4, 2018 to September 4, 2018
- Review delayed by FDA from June 16, 2018 to September 16, 2018.
Review the April specialty drug FDA actions newsletter here.
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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 February 16,2018. https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ReportsBudgets/UCM591976.pdf?utm_campaign=Advancing%20Health%20through%20Innovation%3A%20New%20Drug%20Approvals%20and%20Other%20Drug%20Therapy%20Advances%20of%202017&utm_medium=email&utm_source=Eloqua
Pharma Intelligence Center Web Site. Accessed February 28, 2018. https://pharma.globaldata.com
U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed February 28, 2018. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm
U.S. Food and Drug Administration. Novel Drug Approvals for 2017. Accessed February 28, 2018. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm