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Rare Diseases

FDA Grants Orphan Drug Designation to Ocugen’s OCU300 for Ocular Graft Versus Host Disease

MALVERN, Pa., Aug. 9, 2017 /PRNewswire/ — Ocugen, Inc., a clinical stage biopharmaceutical company developing novel treatments for sight-threatening diseases, today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for OCU300 (brimonidine tartrate) for the treatment of ocular graft versus host disease (oGVHD). Ocular …

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FDA Grants Priority Review and Breakthrough Therapy Designation for Zelboraf in Erdheim-Chester Disease with BRAF V600 Mutation

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 accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Zelboraf® (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD …

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Flex Pharma’s FLX-787 Granted FDA Fast Track Status for Treatment of Severe Muscle Cramps Associated with ALS

Flex Pharma, Inc. (NASDAQ: FLKS), a clinical-stage biotechnology company that is developing innovative and proprietary treatments for cramps and spasticity associated with serious neurological diseases including amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and Charcot-Marie-Tooth (CMT), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for …

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CSL Behring’s Haegarda Receives Orphan-Drug Exclusivity for Prevention of Hereditary Angioedema Attacks

KING OF PRUSSIA, Pa., July 21, 2017 /PRNewswire/ — Global biotherapeutics leader CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted the Company seven years of orphan-drug exclusivity for HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous treatment option for prevention of …

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New Data Network for Huntington’s Disease Research

Huntington’s disease is an hereditary disorder of the nervous system caused by a faulty gene on chromosome four. The faulty gene leads to cell death in neurons in the brain resulting in gradual physical, mental and emotional changes, and ultimately death. Those born to a parent with Huntington’s disease have …

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FDA Approves Endari for the Treatment of Patients with Sickle Cell Disease

The U.S. Food and Drug Administration today approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder. “Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” said Richard …

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FDA Grants Orphan Drug Designation to Sobi’s Treatment for MPS IIIA

STOCKHOLM–(BUSINESS WIRE)–Swedish Orphan Biovitrum AB (publ) (Sobi™) has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the company’s development candidate SOBI003, a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, a …

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FDA Approves Triptodur for Treatment of Patients 2 Years and Older with Central Precocious Puberty

ATLANTA and LAUSANNE, Switzerland, June 30, 2017 /PRNewswire/ — Arbor Pharmaceuticals, LLC, a U.S.-based specialty pharmaceutical company, and Debiopharm International SA, part of Debiopharm Group™, a Swiss-based global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has approved Triptodur™ for the treatment of pediatric patients 2 …

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FDA Unveils Plan to Eliminate Orphan Designation Backlog

Today, the U.S. Food and Drug Administration unveiled a strategic plan to eliminate the agency’s existing orphan designation request backlog and ensure continued timely response to all new requests for designation with firm deadlines. The agency’s Orphan Drug Modernization Plan comes a week after FDA Commissioner Scott Gottlieb committed to …

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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 …

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