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Regeneron Announces Positive Topline Phase 2 Data with Anti-C5 Antibody Pozelimab in Patients with a Rare Blood Disorder

TARRYTOWN, N.Y., Dec. 5, 2019 /PRNewswire/ — Results from initial 6-patient cohort show pozelimab reduced lactate dehydrogenase (LDH) to normal levels at week 8 in patients with paroxysmal nocturnal hemoglobinuria (PNH), utilizing a weekly subcutaneous dosing regimen Second part of Phase 2 trial initiated; plans for Phase 3 program underway …

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FDA Grants Regenerative Advanced Therapy Designation to Adaptimmune’s Investigational Synovial Sarcoma Treatment

PHILADELPHIA and OXFORDSHIRE, United Kingdom, Dec. 03, 2019 (GLOBE NEWSWIRE) — Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, today announced that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for ADP-A2M4 for the treatment of synovial sarcoma. …

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Positive Phase 3 Results Published for Genentech’s Satralizumab in Neuromyelitis Optica Spectrum Disorder

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that data from SAkuraSky, a pivotal Phase III study of the investigational medicine satralizumab for the treatment of neuromyelitis optica spectrum disorder (NMOSD), were published in the November 27, 2019 online issue …

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FDA Approves Oxbryta, the First Drug Specifically Targeting the Root Cause of Sickle Cell Disease

SOUTH SAN FRANCISCO, Calif., Nov. 25, 2019 (GLOBE NEWSWIRE) —  Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Oxbryta™ (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older.1 Oxbryta, an oral therapy …

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FDA Approves Alnylam’s Givlaari for Treatment of Adults with Acute Hepatic Porphyria

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) approved GIVLAARI™ (givosiran) injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening …

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FDA Grants Newron’s Sarizotan Rare Pediatric Disease Designation for Treatment of Rett Syndrome

MILAN & MORRISTOWN, N.J.–(BUSINESS WIRE)–Newron Pharmaceuticals S.p.A. (“Newron”) (SIX: NWRN, XETRA: NP5), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, announced today that the U.S. Food and Drug Administration (FDA) has granted the Rare Pediatric Disease designation for …

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CRISPR Therapeutics and Vertex Announce Positive Early Results for its Investigational CTX001 for Severe Hemoglobinopathies

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 19, 2019 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. One patient with transfusion-dependent …

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FDA Grants Breakthrough Therapy Designation to Orphazyme’s Arimoclomol for Treatment of Niemann-Pick Disease Type C

Copenhagen, Denmark, November 19, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announces that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for arimoclomol, an investigational drug for the treatment of Niemann-Pick Disease Type C (NPC). Kim Stratton, Chief Executive …

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Santhera Announces Publication of Long-term Idebenone Data from SYROS Study in Duchenne Muscular Dystrophy

Pratteln, Switzerland, November 19, 2019 – Santhera Pharmaceuticals (SIX: SANN) announces publication of the previously reported SYROS study data in prominent peer-reviewed medical journal Neuromuscular Disorders [1], demonstrating long-term efficacy with idebenone in slowing respiratory function loss in patients with Duchenne muscular dystrophy (DMD) under routine clinical care. The publication reports data collected …

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CENTOGENE Collaborates with PTC Therapeutics for Global Diagnostic Testing Program for AADC Deficiency

CAMBRIDGE, Ma. and ROSTOCK, Germany, Nov. 18, 2019 (GLOBE NEWSWIRE) — Centogene N.V. (NASDAQ: CNTG), a commercial-stage company focused on rare diseases that transforms real-world clinical and genetic data into actionable information for patients, physicians and pharmaceutical companies, announced that it has signed an agreement with PTC Therapeutics, Inc. (PTC) …

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