Cyclodextrin Drug Trial Update November 14, 2012 From Dr. Forbes Porter, Clinical Director of the Division of Intramural Research at the National Institute of Child Health and Human Development, National Institutes of Health in Bethesda, Maryland. The Therapies for Rare and Neglected Diseases (TRND) team is pleased to announce that the Investigational New Drug (IND) …
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FDA rejects Zavesca panel recommendation.
Unfortunately, in mid-March the FDA advised pharmaceutical company Actelion, which markets Zavesca that they would not yet approve Zavesca for NPC; that more definitive proof is required by the law. We are hopeful that further study efforts will lead to future approval. The consequence of the FDA’s decision is that too many children with NPC …