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Rare Diseases

FDA Grants Breakthrough Therapy Designation to Eiger’s Lonafarnib for Treatment of Progeria and Progeroid Laminopathies

PALO ALTO, Calif., December 19, 2018 — Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for rare and ultra-rare diseases, today announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome (HGPS or Progeria) …

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Ligand Acquires Milestone and Royalty Rights to PTX-022 from Palvella Therapeutics

SAN DIEGO–(BUSINESS WIRE)–Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announces the acquisition of economic rights to PTX-022 from Palvella Therapeutics. PTX-022 is a novel, topical formulation of rapamycin currently in Phase 2/3 development for the treatment of pachyonychia congenita (PC), a rare skin disorder for which no FDA-approved treatment exists. Ligand will pay …

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Biogen Exercises Option with Ionis to Develop and Commercialize Investigational Treatment BIIB067 for a Subtype of Familial Amyotrophic Lateral Sclerosis

CAMBRIDGE, Mass. and CARLSBAD, Calif., Dec. 06, 2018 (GLOBE NEWSWIRE) — Biogen Inc (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB067 (IONIS-SOD1RX), an investigational treatment for amyotrophic lateral sclerosis (ALS) with …

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Zogenix Announces Positive Phase 3 Trial Results on the Efficacy and Safety of Fintepla in Dravet Syndrome

EMERYVILLE, Calif., Dec. 03, 2018 (GLOBE NEWSWIRE) —  Zogenix, Inc. (NASDAQ:ZGNX), a pharmaceutical company developing therapies for the treatment of rare diseases, announced today that late-breaking data will be presented on the use of its investigational drug, FINTEPLA® (ZX008; low-dose fenfluramine), in children and young adults with Dravet syndrome. These three …

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FDA Grants Priority Review to Novartis’ AVXS-101, a One-Time Treatment Designed to Address the Genetic Root Cause of SMA Type 1

Basel, December 3, 2018 – Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx)[1], an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the …

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FDA Accepts Editas Medicine’s IND Application for its CRISPR Genome Editing Medicine Being Investigated for Leber Congenital Amaurosis type 10

CAMBRIDGE, Mass., Nov. 30, 2018 (GLOBE NEWSWIRE) —  Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of …

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FDA Approves Catalyst’s Firdapse for the Treatment of Lambert-Eaton Myasthenic Syndrome

CORAL GABLES, Fla., Nov. 29, 2018 (GLOBE NEWSWIRE) — Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Firdapse® (amifampridine) 10 mg tablets for the treatment of …

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Shire Announces Publication of Complete Results of Phase 3 Study of Lanadelumab as Preventive Treatment for Hereditary Angioedema

Dublin, Ireland – 27 November 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, today announced the Journal of the American Medical Association (JAMA) publication of complete results from the Phase 3 HELP Study™, a randomised, placebo-controlled trial evaluating the efficacy and safety of subcutaneously …

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Two Phase 3 Studies of the Triple Combination of VX-659, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function in Cystic Fibrosis Patients

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that treatment with the triple combination of the next-generation corrector VX-659, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis (CF). …

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Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy and Fast Track Designations for RP-L102 Gene Therapy for Fanconi Anemia

NEW YORK–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announces that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations to RP-L102, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia …

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