Sunday , June 25 2017
Home / Rare Diseases

Rare Diseases

Sarepta and Genethon Enter Gene Therapy Research Collaboration for Duchenne Muscular Dystrophy Treatments

CAMBRIDGE, Mass. and EVRY, France, June 21, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, a non-profit R&D organization dedicated to the development of biotherapies for …

Read More »

FDA Grants Orphan Drug Designation to RASRx’s Duchenne Muscular Dystrophy Drug

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 for the treatment of Duchenne Muscular Dystrophy. RASRx1902 is an oral therapy that has shown positive effects on muscle function in animal models of …

Read More »

FDA Grants Breakthrough Therapy Designation to Omeros’ MASP-2 Inhibitor for the Treatment of IgA Nephropathy

SEATTLE–(BUSINESS WIRE)–Omeros Corporation (NASDAQ: OMER) today announced that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation to OMS721 for the treatment of Immunoglobulin A (IgA) nephropathy. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway …

Read More »

Pfizer’s Tafamidis Receives Fast Track Designation for Treatment of Transthyretin Cardiomyopathy

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to tafamidis, the company’s investigational treatment for transthyretin cardiomyopathy (TTR-CM). This rare disease is associated with progressive heart failure and is universally fatal.1,2,3Currently in Phase 3 clinical development for TTR-CM, tafamidis …

Read More »

FDA Grants Breakthrough Therapy Designation to Alnylam’s Acute Hepatic Porphyria Drug

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the prophylaxis of attacks in patients with acute hepatic porphyria (AHP). …

Read More »

GW Pharma’s Epidiolex Reduces Seizures in Children with Dravet Syndrome

London, UK, May 24, 2017 – GW Pharmaceuticals plc (Nasdaq: GWPH, “GW,” “the Company” or “the Group”), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, along with its U.S. subsidiary Greenwich Biosciences, announced today that The New England Journal of Medicine …

Read More »

Bioverativ to Acquire Rare Disease Biotech True North for $400 Million Upfront

WALTHAM, Mass.–(BUSINESS WIRE)–Bioverativ Inc. (NASDAQ: BIVV), a global biotechnology company focused on the discovery, development and commercialization of innovative therapies for hemophilia and other rare blood disorders, today announced that it has entered into a definitive agreement to acquire South San Francisco-based True North Therapeutics, a privately-held, clinical-stage rare disease …

Read More »

Sangamo’s Hemophilia A Gene Therapy Candidate Receives Fast Track Status from the FDA

RICHMOND, Calif., May 16, 2017 /PRNewswire/ — Sangamo Therapeutics, Inc. (NASDAQ: SGMO) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SB-525, the Company’s clinical stage cDNA gene therapy candidate for hemophilia A, which is being developed as part of an exclusive, global …

Read More »

Alexion and Rady Children’s Institute for Genomic Medicine Partner to Speed Diagnosis of Rare Genetic Disorders in Newborns

SAN DIEGO & NEW HAVEN, Conn.–(BUSINESS WIRE)–The Rady Children’s Institute for Genomic Medicine (RCIGM) and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced a strategic partnership to accelerate the diagnosis of critically-ill newborns with rare genetic disorders. The collaboration combines the Institute’s genomic research expertise with Alexion data science and bioinformatics capabilities …

Read More »

Ionis Pharmaceuticals’ Inotersen Meets Both Primary Endpoints in Phase 3 Study in Familial Amyloid Polyneuropathy

CARLSBAD, Calif., May 15, 2017 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the Phase 3 NEURO-TTR study of inotersen (IONIS-TTRRx) in patients with familial amyloid polyneuropathy (FAP) met both primary endpoints. Over the 15-month period of the study, inotersen-treated patients achieved statistically significant benefit compared to placebo …

Read More »