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 …
NPC Parents Help Get First Drug Recommendation!
A Food and Drug Administration (FDA) advisory panel recommended Zavesca to treat NPC. Phil Marella of DART, Cindy Parseghian of the Parseghian Foundation and Barb Vorpahl of the NNPDF spoke during the public portion of the hearing. The panel of outside medical experts voted 10-to-3 in favor of a question that asked “in light of …