Tag: FDA

A total of 103 posts are filed under FDA
RARECast: An FDA Perspective on Rare Diseases
  The FDA Office of Orphan Products Development seeks to advance the evaluation of drugs and diagnostics to treat rare diseases. A growing… Continue Reading
Girl With Duchenne Waits for Life-Changing Drug, Eteplirsen, To Gain FDA Approval
Susie Arroyo knows she is an anomaly. The 25-year-old Pendleton woman has a genetic mutation called Duchenne Muscular Dystrophy, a… Continue Reading
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Kids (Six and Under) With Rare Blood Disorder, Chronic ITP, Get FDA Approval on New Novartis Drug, Promacta
Promacta® (eltrombopag) significantly increased and sustained platelet counts in two studies, including the largest Phase III clinical… Continue Reading
Today: Last Chance to Register for 21st Century Cures Initiative Briefing
Briefing on the 21st Century Cures Initiative: Second Discussion Draft takes place today, May 12, 2015 from 12:00PM – 1:30 PM. PhRMA… Continue Reading
Horizon Pharma’s Friedreich’s Ataxia Drug Gets Fast-Track Designation
Horizon Pharma PLC said Friday that its experimental Friedreich’s ataxia treatment has received fast-track designation from the U.S. Food… Continue Reading
Rare Disease Report: FDA Approves Unituxin (dinutuximab) for Neuroblastoma in Children
The U.S. Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) as part of first-line therapy for pediatric patients with… Continue Reading
RAREcast: How the Orphan Drug Act Reshaped the Rare Disease Landscape
The U.S. Food and Drug Administration approved more than 230 new drugs to treat rare diseases in the past decade and there are currently… Continue Reading