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Rare Diseases

FDA Accepts and Grants Priority Review to Pfizer’s Tafamidis for Treatment of Transthyretin Amyloid Cardiomyopathy

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) announced today that the US Food and Drug Administration (FDA) accepted for filing the company’s New Drug Applications (NDAs) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Pfizer has submitted two NDAs based on two forms of tafamidis: meglumine salt and free …

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Acceleron Announces Publication of Luspatercept Phase 2 Beta-Thalassemia Study Results in Blood

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today announced that the journal Bloodhas published results from the Phase 2 study of luspatercept in patients with red blood cell (RBC) transfusion-dependent and non-transfusion-dependent beta-thalassemia. Luspatercept …

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Sanofi Announces NEJM Publishes Positive Results of Pivotal Trial of Cablivi for Treatment of Rare Blood Clotting Disorder

PARIS – January 9, 2019 – The New England Journal of Medicine (NEJM) today published positive results of the Phase 3 trial of Cablivi® (caplacizumab) in adults with acquired thrombotic thrombocytopenic purpura (aTTP).aTTP is a rare, life-threatening autoimmune blood disorder characterized by extensive clot formation in small blood vessels throughout the body, leading to …

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Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

NOVATO, Calif., Jan. 04, 2019 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced positive topline safety and efficacy data from the first, lowest dose cohort of the ongoing Phase 1/2 study …

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Catabasis Pharmaceuticals Phase 3 PolarisDMD Clinical Trial for Edasalonexent in Duchenne Muscular Dystrophy Progress Update

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared an update on clinical trial sites in the United States and recent international clinical trial application approvals to perform the Phase 3 PolarisDMD trial for edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD). PolarisDMD clinical trial sites in the United …

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ProQR’s QR-421a Receives Fast Track Designation from the FDA for Treatment of Usher Syndrome Type 2

LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan. 02, 2019 (GLOBE NEWSWIRE) — ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that it received Fast Track designation from the Food and Drug Administration …

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FDA Expands Approval of Horizon’s Ravicti Oral Liquid for Use in Newborns Living with Urea Cycle Disorder

DUBLIN–(BUSINESS WIRE)–Horizon Pharma plc (NASDAQ: HZNP) today announced the U.S. Food and Drug Administration (FDA) has approved a supplemental new drug application (sNDA) to expand the age range for RAVICTI® (glycerol phenylbutyrate) Oral Liquid to include infants younger than two months of age living with a urea cycle disorder (UCD). …

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Alexion Receives Early FDA Approval for Ultomiris in Adults with Paroxysmal Nocturnal Hemoglobinuria

BOSTON–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the U.S. Food and Drug Administration (FDA) has approved ULTOMIRIS™ (ravulizumab-cwvz), the first and only long-acting C5 complement inhibitor administered every eight weeks, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating ultra-rare blood disorder characterized by complement-mediated destruction …

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FDA Approves Stemline’s Elzonris, the First Treatment for Blastic Plasmacytoid Dendritic Cell Neoplasm, a Rare Blood Disease

The U.S. Food and Drug Administration today approved Elzonris (tagraxofusp-erzs) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients, two years of age and older. “Prior to today’s approval, there had been no FDA approved therapies for BPDCN. The standard of care …

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Sarepta Completes Submission of NDA Seeking Approval of Golodirsen in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53

CAMBRIDGE, Mass., Dec. 20, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat those …

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