The US Food and Drug Administration's Genetic Metabolic Diseases Advisory Committee will face a host of efficacy issues with Zevra Denmark A/S’ arimoclomol at its inaugural meeting, including uncertainty about the treatment effect in the lone pivotal trial, challenges interpreting the primary endpoint, and adequacy of the confirmatory evidence.
Key Takeaways
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The FDA questioned the validity of the clinical outcome measure, as well as whether a treatment benefit can be ascertained, in the lone randomized, placebo-controlled study of arimoclomol in Niemann-Pick disease type C.
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The agency also will ask its advisors whether various types of proffered data collectively can serve as confirmatory evidence of efficacy in support of single study
Zevra is seeking approval of arimoclomol for treatment of Niemann-Pick disease type C (NPC), a rare lysosomal storage disorder without approved therapies