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Alexion to Acquire Wilson Therapeutics and its Rare Liver Disorder Drug

NEW HAVEN, Conn.–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Wilson Therapeutics AB (publ) announced today that Alexion has made a recommended public cash offer to the shareholders in Wilson Therapeutics to acquire all outstanding shares in Wilson Therapeutics by way of a tender offer, through a wholly-owned subsidiary. Wilson Therapeutics is …

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GW Pharma Announces Publication of Landmark Epidiolex (cannabidiol) Study in The Lancet

London, UK, Carlsbad, CA, January 24, 2018 – 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 Lancet has published results from a Phase 3 …

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FDA Grants Fast Track Status to Sobi’s Investigational MPS IIIA Drug

Swedish Orphan Biovitrum AB (publ) (Sobi™) announces that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug (IND) application for the drug candidate SOBI003. In addition, SOBI003 was granted Fast Track status by the FDA. SOBI003 is a …

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Orphazyme’s Arimoclomol Receives FDA Rare Pediatric Disease Designation for Niemann-Pick disease Type C

Copenhagen, Denmark, January 19, 2018 – Orphazyme A/S, a Danish biotech company listed on Nasdaq Copenhagen (TICKER: ORPHA.CO), with a late-stage, orphan-drug pipeline, today announced that arimoclomol has been granted rare pediatric disease designation by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease Type C (NPC). …

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Biohaven Announces Positive Results from Bioequivalence Study with its Amyotrophic Lateral Sclerosis Drug

NEW HAVEN, Conn., Jan. 9, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) (“Biohaven” or the “Company”) announced positive results today from its bioequivalence study with BHV-0223, an innovative sublingual formulation of riluzole. The study was designed to demonstrate pharmacokinetic equivalence of sublingual BHV-0223 compared to the reference …

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FDA Grants Breakthrough Therapy Designation to Novartis’ Promacta for Treatment of Severe Aplastic Anemia

Basel, January 4, 2018 – Novartis today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Promacta®(eltrombopag) for use in combination with standard immunosuppressive therapy for the treatment of patients with severe aplastic anemia (SAA) as a first-line therapy. Promacta, which is marketed as Revolade® in most …

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Patients Treated with Sarepta’s Eteplirsen Experienced a Reduction in Pulmonary Decline in Duchenne Muscular Dystrophy Study

CAMBRIDGE, Mass., Dec. 27, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced that the pulmonary function results from eteplirsen-treated Duchenne muscular dystrophy (DMD) patients (N=12) in Study 201/202 compared …

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INSYS’ Cannabidiol Solution Receives FDA Fast Track Designation as Treatment for Prader-Willi Syndrome

PHOENIX, Dec. 26, 2017 (GLOBE NEWSWIRE) — INSYS Therapeutics, Inc. (NASDAQ:INSY), announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company’s cannabidiol (CBD) oral solution for the treatment of Prader-Willi syndrome, a rare and complex genetic disorder characterized by insatiable appetite in children that …

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FDA Grants Orphan Drug Designation to Organovo’s 3D Bioprinted Therapeutic Liver Tissue Treatment of Alpha-1 Antitrypsin Deficiency

SAN DIEGO, Dec. 26, 2017 (GLOBE NEWSWIRE) — Organovo Holdings, Inc. (NASDAQ:ONVO) (“Organovo”) today announced that the U.S. Food and Drug Administration (“FDA”) granted orphan drug designation for the Company’s treatment of alpha-1 antitrypsin deficiency (“A1AT”) with its 3D bioprinted liver therapeutic tissue. “We are extremely pleased to receive orphan …

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FDA Approves Novel Gene Therapy to Treat Patients with a Rare Form of Inherited Vision Loss

The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused …

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