FDA News

FDA Approves Genentech’s Enspryng for Treatment of Neuromyelitis Optica Spectrum Disorder

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Enspryng™ (satralizumab-mwge) as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD …

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Scholar Rock’s Investigational Spinal Muscular Atrophy Drug Receives Rare Pediatric Disease Designation from the FDA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular …

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FDA Approves NS Pharma’s Viltepso Injection for Treatment of Duchenne Muscular Dystrophy Patients Amenable to Exon 53 Skipping Therapy

PARAMUS, N.J., Aug. 12, 2020 /PRNewswire/ — NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President Shigenobu Maekawa), announced today that the U.S. Food & Drug Administration (FDA) has approved VILTEPSO™ (viltolarsen) injection for patients with Duchenne muscular dystrophy …

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FDA Accepts and Grants Priority Review to Regeneron’s Evinacumab BLA for Treatment of HoFH, an Ultra-rare Inherited Form of High Cholesterol

Homozygous familial hypercholesterolemia affects approximately 1,300 patients in the U.S., the vast majority of whom are unable to reach target LDL-C levels with currently available therapies Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review a Biologics License Application (BLA) for evinacumab as …

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Mersana Receives FDA Fast Track Designation for XMT-1536 for the Treatment of Patients with Platinum-resistant Ovarian Cancer

CAMBRIDGE, Mass., Aug. 11, 2020 (GLOBE NEWSWIRE) — Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track …

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FDA Accepts and Grants Priority Review to Pegunigalsidase Alfa BLA for Treatment of Fabry Disease

CARMIEL, Israel, Aug. 11, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, or the Company, together with its development and commercialization partner Chiesi …

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FDA Rejects Fennec’s NDA for Pedmark for Treatment of Ototoxicity Associated with Cisplatin in Pediatric Patients with Solid Tumors

RESEARCH TRIANGLE PARK, N.C., Aug. 11, 2020 (GLOBE NEWSWIRE) — Fennec Pharmaceuticals Inc., a specialty pharmaceutical company, today announced that it received a Complete Response Letter (CRL) on August 10, 2020 from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for PEDMARK™ (a unique formulation of sodium …

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Gilead Submits New Drug Application to the FDA for Veklury (Remdesivir) for the Treatment of COVID-19

FOSTER CITY, Calif.–(BUSINESS WIRE)–Gilead Sciences, Inc. (Nasdaq: GILD) announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Veklury® (remdesivir), an investigational antiviral for the treatment of patients with COVID-19. Veklury is currently available in the U.S. under an Emergency Use …

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FDA Approves Genentech’s Evrysdi for Treatment of Spinal Muscular Atrophy in Adults and Children 2 Months and Older

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Evrysdi showed clinically-meaningful …

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FDA Approves Trevena’s Olinvyk, a New Opioid for Intravenous Use in Hospitals, Other Controlled Clinical Settings

CHESTERBROOK, Pa., Aug. 10, 2020 (GLOBE NEWSWIRE) — Trevena, Inc. (Nasdaq: TRVN), a biopharmaceutical company focused on the development and commercialization of novel medicines for patients with central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has approved OLINVYK in adults for the management of …

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