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Rare Diseases

ChemoCentryx Rare Disease Drug as Effective as Standard of Care in Mid-Stage Study

MOUNTAIN VIEW, Calif., Jan. 06, 2016 (GLOBE NEWSWIRE) — ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company developing orally-administered therapeutics to treat autoimmune diseases, inflammatory disorders, and cancer, today announced positive top-line data from its’ Phase II CLEAR trial with CCX168 in patients with anti-neutrophil cytoplasmic antibody (ANCA) -associated vasculitis, or …

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Raptor Announces Plans to Advance Development of Huntington’s Disease Drug

NOVATO, Calif., Dec. 10, 2015 /PRNewswire/ — Raptor Pharmaceutical Corp. (Nasdaq: RPTP) today announced 36-month efficacy results from a Phase 2/3 clinical trial evaluating RP103 for the potential treatment of Huntington’s disease, called CYST-HD, conducted in collaboration with the Centre Hospitalier Universitaire d’Angers (CHU d’Angers), France. Data from the study revealed …

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OPKO’s GeneDx Study Reveals Usefulness of Whole Exome Sequencing in Diagnosing Rare Disorders

MIAMI–(BUSINESS WIRE)–OPKO Health, Inc. (NYSE:OPK) announced today through its subsidiary GeneDx, results from a retrospective review of molecular diagnostic testing by Whole Exome Sequencing (WES). The peer-reviewed study, “Clinical Application of Whole Exome Sequencing across Clinical Indications,” was published in the December 2015 issue of Genetics in Medicine. It is …

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Baxalta Wins FDA Approval for Vonvendi for Treatment of Patients with Von Willebrand Disease

BANNOCKBURN, Ill.–(BUSINESS WIRE)– Baxalta Incorporated (NYSE: BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today that the U.S. Food and Drug Administration (FDA) has approved VONVENDI [von Willebrand factor (Recombinant)]. VONVENDI is the first and only recombinant treatment for …

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FDA Approves Alexion’s Kanuma for Patients with a Rare Enzyme Disease

CHESHIRE, Conn.–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced today that the U.S. Food and Drug Administration (FDA) has approved Kanuma™(sebelipase alfa) for the treatment of patients of all ages with a diagnosis of lysosomal acid lipase deficiency (LAL-D). Kanuma, an innovative enzyme replacement therapy (ERT), is the first therapy approved …

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Pfizer and Merck KGaA’s MCC Drug Receives Breakthrough Therapy Designation

Merck KGaA, Darmstadt, Germany, and Pfizer today announced that the US Food and Drug Administration (FDA) has granted avelumab*, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, Breakthrough Therapy designation for the treatment of patients with metastatic Merkel cell carcinoma (MCC) who have progressed after at least one previous chemotherapy …

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FDA Approves Baxalta’s Adynovate for Treatment of Hemophilia A

BANNOCKBURN, Ill.–(BUSINESS WIRE)– Baxalta Incorporated (NYSE: BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today that the U.S. Food and Drug Administration (FDA) has approved ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment …

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Teva’s Investigational Tardive Dyskinesia Drug Granted FDA Breakthrough Therapy Designation

JERUSALEM–(BUSINESS WIRE)–Teva Pharmaceutical Industries Ltd. (NYSE and TASE:TEVA) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to SD-809 (deutetrabenazine) for the treatment of patients with moderate to severe tardive dyskinesia, a hyperkinetic movement disorder affecting about 500,000 people in the United States. …

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Agilis Biotherapeutics’ Investigational Angelman Syndrome Drug Granted Orphan Drug Status

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AGIL-AS, the Company’s gene therapy product candidate being developed for …

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