FDA advisory committee recommends approval of miglustat in Niemann-Pick disease

The US FDA's endocrinologic and metabolic drugs advisory committee has virtually urged the agency to ignore its own legislative mandate and give Actelion Pharmaceuticals ethe green light to market miglustat for the genetic lysosomal storage disorder Niemann-Pick disease, type 3 (NP-C). On January 12th, the panel voted 10 to three that the risk-benefit profile in the safety and efficacy data presented by Actelion justified approval. But that majority included four committee members who had voted "no" to the question of whether Actelion had presented "substantial efficacy" of efficacy - the legal standard the FDA is supposed to use. On that issue, six members found there was substantial evidence of efficacy, while seven members said there was not.