A panel of expert advisers to the Food and Drug Administration voted Friday against approving Intercept Pharmaceuticals’ investigational treatment for NASH, a prevalent liver disease with no available medicines.
The group voted 12-2, with two abstentions, that the benefits of Intercept’s medicine did not outweigh its risks, citing serious concerns about fatal drug-related liver damage and uncertainty over whether the drug’s modest effects will ultimately improve the lives of NASH patients. The panel voted 15-1 in favor of rejecting Intercept’s application for an accelerated approval and waiting until the company has gathered data on the drug’s effects on long-term patient health.
“For this drug, we have clear evidence of safety risk — including very serious safety concerns with [liver injury] — but we have only evidence for potential efficacy,” said James Floyd, an epidemiologist at the University of Washington. “It’s impossible in my mind to ensure a good risk-benefit profile based on this surrogate endpoint data.”
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