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Rare Diseases

FDA Grants Orphan Drug Designation to Processa’s PCS499 in Necrobiosis Lipoidica

HANOVER, Md., June 22, 2018 /PRNewswire/ — Processa Pharmaceuticals, Inc. (OTC: PCSA) a clinical stage biopharmaceutical company developing products to improve the survival and/or quality of life for patients who have a high unmet medical need, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation …

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FDA Approves Expanded Label for Shire’s Cinryze for Prevention of Attacks in Pediatric Hereditary Angioedema

Dublin, Ireland – Thursday, June 21, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for CINRYZE® (C1 esterase inhibitor [human]), making it available to help prevent angioedema attacks …

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Sarepta Announces Positive Preliminary Results from Phase 1/2a Gene Therapy Trial in Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., June 19, 2018 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from …

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FDA Lifts Clinical Hold on Solid Biosciences’ Clinical Trial for its Investigational Duchenne Muscular Dystrophy Treatment

CAMBRIDGE, Mass., June 18, 2018 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (NASDAQ:SLDB) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). …

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Blueprint Avapritinib Demonstrates Profound and Durable Clinical Activity in Advanced Systemic Mastocytosis Trial

CAMBRIDGE, Mass., June 15, 2018 /PRNewswire/ — Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, today announced updated data from its ongoing Phase 1 EXPLORER clinical trial of avapritinib, a potent and highly selective KIT and PDGFRA inhibitor in development for patients with …

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Novartis Survey Uncovers Real-World Impact of Immune Thrombocytopenia on Patients’ Quality of Life

Basel, June 15, 2018 – Many patients with the rare blood disorder immune thrombocytopenia (ITP) find the disease has a negative impact on their everyday quality of life, according to interim results of a Novartis survey, called I-WISh, presented today at the 23rd Congress of the European Hematology Association (EHA) in Stockholm, Sweden …

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FDA Approves Genentech’s Rituxan (Rituximab) for Pemphigus Vulgaris

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Rituxan®(rituximab) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), a rare, serious, potentially life-threatening condition characterized by progressive painful blistering of the …

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Alnylam Reports Updated Positive Results from Phase 1/2 Study of Lumasiran in Primary Hyperoxaluria Type 1

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1). Results were presented at the OxalEurope, European Hyperoxaluria Consortium, taking …

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FDA Grants Orphan Drug Designation to Ironwood’s Olinciguat for the Treatment of Sickle Cell Disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Ironwood Pharmaceuticals, Inc. (NASDAQ: IRWD), a commercial biotech company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to olinciguat (IW-1701) for the treatment of patients with sickle cell disease. Olinciguat is an orally administered soluble guanylate cyclase (sGC) stimulator. “There is an …

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FDA Grants Orphan Drug Designation to Alnylam’s Investigational RNAi Therapeutic for Treatment of Transthyretin-Mediated Amyloidosis

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALN-TTRsc02, an investigational RNAi therapeutic for the treatment of transthyretin-mediated amyloidosis. ALN-TTRsc02 has the potential to be a once-quarterly, low volume, subcutaneously …

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