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Rare Diseases

Rocket Pharmaceuticals and REGENXBIO Announce New License Agreement for the Treatment of Danon Disease Using NAV AAV9 Vector

NEW YORK & ROCKVILLE, Md.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT), a leading U.S.-based multi-platform gene therapy company, and REGENXBIO Inc. (Nasdaq:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced an exclusive, worldwide license agreement for …

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GW Pharma Announces Second Positive Phase 3 Trial for Epidiolex Oral Solution CV in Patients with Dravet Syndrome

LONDON, Nov. 26, 2018 (GLOBE NEWSWIRE) — GW Pharmaceuticals plc (Nasdaq: GWPH, AIM: “GWP,” “GW,” “the Company” or “the Group”), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, announces positive top-line results of the second randomized, double-blind, placebo-controlled Phase 3 clinical …

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FDA Accepts Biohaven’s NDA for BHV-0223, a Sublingual Delivery Form of Riluzole for Treatment of Amyotrophic Lateral Sclerosis

NEW HAVEN, Conn., Nov. 26, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced that its 505(b)(2) new drug application (NDA) for BHV-0223, an investigational sublingual form of riluzole for the treatment of people living with amyotrophic lateral sclerosis (ALS), has been accepted for review by the U.S. Food and …

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FDA Approves Gamifant, the First and Only Treatment Indicated for Primary Hemophagocytic Lymphohistiocytosis

WALTHAM, Mass.–(BUSINESS WIRE)–Sobi, an international biopharmaceutical company dedicated to rare diseases, and Novimmune SA, a Swiss biotech company, today announced that the U.S. Food and Drug Administration (FDA) has approved Gamifant®(emapalumab-lzsg), an interferon gamma (IFNγ) blocking antibody for the treatment of pediatric (newborn and older) and adult patients with primary hemophagocytic …

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LEO Pharma and PellePharm Announce $760 Million Collaboration to Advance Innovative Therapies for Rare Skin Diseases

SAN FRANCISCO & BALLERUP, Denmark–(BUSINESS WIRE)–Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm today announced a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such …

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FDA Grants Orphan Drug Designation to Pliant’s PLN-74809 for Treatment of Primary Sclerosing Cholangitis

SOUTH SAN FRANCISCO, Calif., Nov. 20, 2018 /PRNewswire/ — Pliant Therapeutics, Inc., a biotechnology company focused on discovering, developing and commercializing treatments for fibrotic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the company’s anti-fibrotic lead compound, PLN-74809, for the treatment …

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Catabasis and the University of Texas Southwestern Partner to Explore the Potential Benefits of Edasalonexent on Cardiac Function in Duchenne and Becker Muscular Dystrophies

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced a collaboration with Pradeep Mammen, MD, FACC, FAHA, founder and Medical Director of the Neuromuscular Cardiomyopathy Clinic at the University of Texas Southwestern (UT Southwestern) Medical Center as well as Co-Director of the National Institute of Health Sponsored UT …

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FDA Accepts Shire’s Supplemental New Drug Application for Gattex for Children with Short Bowel Syndrome

Cambridge, Mass. – November 13, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG) the global leader in rare diseases, announced today the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental new drug application to extend the indication of GATTEX® (teduglutide [rDNA origin]) for Injection to pediatric patients (aged 1-17 years …

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Alnylam Presents Updated Phase 1/2 Open-Label Extension Results for Givosiran for the Treatment of Acute Hepatic Porphyria

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company presented updated results from the ongoing Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic, targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP). The new …

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FDA Approves Aquestive’s Sympazan Oral Film for Treatment of Seizures Associated with Lennox-Gastaut Syndrome

WARREN, N.J., Nov. 2, 2018 /PRNewswire/ — Aquestive Therapeutics, Inc. (NASDAQ: AQST), a specialty pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) approved SYMPAZAN™ (clobazam) oral film for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients 2 years of age or older.1 SYMPAZAN is the first and only oral film FDA-approved …

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