In another blow for NASH, Intercept gets FDA rejection for obeticholic acid

Shares of US biotech firm Intercept Pharmaceuticals (Nasdaq: ICPT) closed down virtually 40% at $46.70 yesterday, after the company revealed the it had received a complete response letter from the Food and Drug Administration regarding its New Drug Application (NDA) for obeticholic acid (OCA) for the treatment of fibrosis due to non-alcoholic steatohepatitis (NASH).

The CRL indicated that, based on the data the FDA has reviewed to date, the agency has determined that the predicted benefit of OCA based on a surrogate histopathologic endpoint remains uncertain and does not sufficiently outweigh the potential risks to support accelerated approval for the treatment of patients with liver fibrosis due to NASH. The FDA recommends that Intercept submit additional post-interim analysis efficacy and safety data from the ongoing REGENERATE study in support of potential accelerated approval and that the long-term outcomes phase of the study should continue.

“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,” said Dr Mark Pruzanski, president and chief executive of Intercept.