The FDA recommends additional safety and efficacy data from the ongoing REGENERATE trial.
The US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Intercept Pharmaceuticals for obeticholic acid (OCA) to treat liver fibrosis caused by nonalcoholic steatohepatitis (NASH).
The company previously submitted a New Drug Application (NDA) based on data from 35 clinical trials involving more than 1700 NASH patients.
OCA currently represents the only NASH drug with a Breakthrough Therapy designation and has shown the reproducible ability to reverse or stabilize liver fibrosis in NASH patients with advanced fibrosis, considered the strongest predictor of adverse clinical outcomes such as liver-related death.
The FDA determined after reviewing all available data that the predicted benefit of the treatment was based on a surrogate histopathologic endpoint that remains uncertain and does not sufficiently outweigh the potential risks to support the accelerated approval of obeticholic acid.
The agency recommends Intercept submit additional post-interim analysis efficacy and safety data from the ongoing REGENERATE study to support a potential accelerated approval. The FDA also said the long-term outcomes phase of the study should continue as planned.
However, the company has come out in disagreement with the FDA decision.
“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,” Mark Pruzanski, MD, President and CEO of Intercept, said in a statement.
“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.”
Pruzanski went on to see that the FDA has set too high a bar for their application.
Intercept previously disclosed based on the FDA’s decision to postpone a tentatively scheduled advisory committee meeting, that they expected the review of the NDA would extend beyond the PDUFA goal data and the FDA would reschedule the Adcom.