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 the safety and efficacy data presented in this application, does the risk-benefit profile of Zavesca support its approval for treatment of NPC?” The FDA usually follows the advice of its panels but is not required to.¬† The drugs sponsor, Actelion said the agency is expected to decide in March whether to approve Zavesca for treating NPC.

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