FDA's new metabolic diseases committee gives hesitant nod to ultra-rare disease drug

By Amanda Conti

In its inaugural meeting, FDA’s Genetic Metabolic Diseases Advisory Committee heard Zevra’s second try for arimoclomol to treat ultra-rare lysosomal storage disorder Niemann-Pick Type C. The committee voted 11-5 in favor of the drug’s effectiveness, leaving FDA to test the limits of its regulatory flexibility toward post hoc analysis and lackluster confirmatory evidence.

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