Intercept Pharmaceuticals (NASDAQ:ICPT) slumps 37% premarket on increased volume in reaction to its announcement that it received a Complete Response Letter (CRL) from the FDA regarding its marketing application seeking approval to use obeticholic acid (OCA) to treat liver fibrosis due to nonalcoholic steatohepatitis (NASH).
The CRL cited the uncertainty of a predicted treatment benefit based on a surrogate histopathologic endpoint, precluding accelerated approval. The agency recommends that the company conduct additional post-interim analyses of data from the ongoing REGENERATE study in support of potential accelerated approval, adding that the long-term outcomes phase should continue.
Chief Mark Pruzanski, M.D., says, “At no point during the review did the FDA communicate that OCA was not approvable on an accelerated basis, and we strongly believe that the totality of data submitted to date both meet the requirements of the Agency’s own guidance and clearly support the positive benefit-risk profile of OCA. We are disappointed to see the determination the Agency has reached based on an apparently incomplete review, and without having provided medical experts and patients the opportunity to be heard at the anticipated Adcom on the merits of OCA, which is a designated Breakthrough Therapy. The FDA has progressively increased the complexity of the histologic endpoints, creating a very high bar that only OCA has so far met in a pivotal Phase 3 study. On behalf of the hepatology community, we are very concerned that the Agency’s apparently still evolving expectations will make it exceedingly challenging to bring innovative therapies to NASH patients with high unmet medical need. We plan to meet as soon as possible with the FDA to review the CRL and discuss options for an efficient path forward to approval.”
Management will host a conference call this morning at 8:30 am ET to discuss the situation.
