Intercept Pharmaceuticals Inc (NASDAQ: ICPT) stock plummeted 39.73% on 29th June, 2020 and continued its bearish momentum falling over 2.3% on 30th June, 2020 (as of 9:48 am GMT-4 ; Source: Google finance). The company received a U.S. Food and Drug Administration Complete Response Letter regarding its New Drug Application for a treatment for fibrosis caused by nonalcoholic steatohepatitis, or NASH. The letter says that the regulator based on the data the regulator has reviewed to date, the forecasted benefit of the treatment, obeticholic acid, or OCA, is uncertain and does not sufficiently outweigh the potential risks to underpin the accelerated approval for the treatment of patients with liver fibrosis due to NASH. The FDA recommended 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.
However, the company believes that the totality of data submitted to date both meet the requirements of the Agency’s own guidance and absolutely support the positive benefit-risk profile of OCA. ICPT is disappointed to see that the regulator has reached the determination based on an incomplete review, and di not provide the medical experts and patients the opportunity to be heard at the expected Adcom on the merits of OCA, which is a designated Breakthrough Therapy. The FDA has therefore increased the complexity of the histologic endpoints, which has created a very high bar that only OCA has so far met in a pivotal Phase 3 study. The regulator’s apparently still evolving expectations have made it to be exceedingly challenging to bring innovative therapies to NASH patients with high unmet medical need. The company intends to meet the FDA soon to review the CRL and discuss options for an efficient path forward to approval.
Meanwhile, the NDA submission for OCA is the first for NASH, which was based on data from 35 clinical trials and more than 1,700 NASH patients treated with the drug. OCA is the only investigational NASH drug with Breakthrough Therapy designation that had uniquely demonstrated reproducible ability to reverse or otherwise stabilize liver fibrosis in patients with advanced fibrosis due to NASH. As per the FDA draft guidance for NASH fibrosis, of the histologic features of NASH, fibrosis is considered the strongest predictor of adverse clinical outcomes, that also includes liver-related death. There is currently no approved therapy for this deadly disease, which has become a leading cause of liver failure and has resulted in poor clinical outcomes.