Tag Archives: Niemann-Pick disease

January, 2016

6 January
FDA Grants Breakthrough Therapy Designation to Vtesse’s Drug for Treatment of a Rare Genetic Disease

GAITHERSBURG, Md., Jan. 6, 2016 /PRNewswire/ — Vtesse, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted its drug candidate, VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC), Breakthrough Therapy designation status. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status …
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8 June
Breakthrough Status Granted to Genzyme’s Rare Enzyme Replacement Drug

Genzyme, a Sanofi company, recently announced that its investigational enzyme-replacement therapy was granted Breakthrough Therapy designation from US health regulators. The company said that the agency granted the status to its olipudase alfa for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick …
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