Rare Diseases

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 Grants Axsome Therapeutics’ AXS-12 Breakthrough Therapy Designation for Treatment of Narcolepsy

NEW YORK, Aug. 05, 2020 (GLOBE NEWSWIRE) — Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for AXS-12 for the treatment of cataplexy …

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FDA Approves GW’s Epidiolex Oral Solution to Treat Seizures Associated with Tuberous Sclerosis Complex

CARLSBAD, Calif., Aug. 03, 2020 (GLOBE NEWSWIRE) — GW Pharmaceuticals plc (Nasdaq: GWPH), the world leader in the science, development, and commercialization of cannabinoid prescription medicines, along with its U.S. subsidiary Greenwich Biosciences, Inc., today announced the U.S. Food and Drug Administration (FDA) has approved EPIDIOLEX® (cannabidiol) oral solution to treat …

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FDA Grants Fast Track Designation to BioCryst’s BCX9930 for Treatment of Paroxysmal Nocturnal Hemoglobinuria

RESEARCH TRIANGLE PARK, N.C., Aug. 03, 2020 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). According to the FDA, the purpose of the …

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New Topline Tepezza Data Underscore its Efficacy in Longer Disease Duration, Long-Term Durability and Potential for Retreatment in Patients with Thyroid Eye Disease

DUBLIN–(BUSINESS WIRE)–Horizon Therapeutics plc (Nasdaq: HZNP) today announced positive topline data from two clinical trials that add to the growing body of evidence supporting the efficacy and safety of TEPEZZA® (teprotumumab-trbw) for the treatment of Thyroid Eye Disease (TED). TEPEZZA is the first and only medicine approved by the U.S. Food …

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Imara’s IMR-687 Granted Fast Track Designation and Rare Pediatric Disease Designation for Treatment of Beta-Thalassemia

BOSTON, July 30, 2020 (GLOBE NEWSWIRE) — IMARA Inc. (Nasdaq: IMRA), a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Rare Pediatric …

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Momenta Pharmaceuticals Announces FDA Rare Pediatric Disease Designation for Nipocalimab in HDFN

CAMBRIDGE, Mass., July 28, 2020 (GLOBE NEWSWIRE) — Momenta Pharmaceuticals, Inc. (NASDAQ: MNTA) today announced that its novel drug candidate, nipocalimab, has received rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for the prevention of hemolytic disease of the fetus and newborn (HDFN). Additionally, FDA granted nipocalimab orphan …

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FDA Requests More Data Prior to Lifting Clinical Hold on Solid Biosciences’ Phase I/II Gene Therapy Trial in DMD

CAMBRIDGE, Mass., July 24, 2020 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy, today announced that it received written communication from the U.S. Food and Drug Administration (FDA) that the Company’s IGNITE DMD Phase I/II clinical trial …

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Sarepta Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy for Treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., July 24, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene …

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Ovid Therapeutics and University of Connecticut Partner to Accelerate the Development of Next-Generation Genetic Therapy for Angelman Syndrome

NEW YORK and FARMINGTON, Conn., July 23, 2020 (GLOBE NEWSWIRE) — Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, and the University of Connecticut School of Medicine (“UConn”), today announced a research collaboration and license agreement to …

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