Intercept receives FDA CRL for obeticholic acid recommending additional data
Intercept Pharmaceuticals (ICPT) announced that the U.S. Food and Drug Administration has issued a Complete Response Letter regarding the New Drug Application for obeticholic acid for the treatment of fibrosis due to nonalcoholic steatohepatitis.
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.
NASH is the severe form of Non-Alcoholic Fatty Liver Disease (NAFLD). It can lead to liver cancer, cirrhosis, and liver transplants.
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.
Intercept had previously disclosed that, based on the FDA’s decision to postpone a tentatively scheduled advisory committee meeting, it was expected that the Agency’s review of its NDA would extend beyond the PDUFA goal date and that the FDA would move forward with rescheduling the Adcom.
The NDA submission for OCA is the first for NASH and 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 and has uniquely demonstrated reproducible ability to reverse or otherwise stabilize liver fibrosis in patients with advanced fibrosis due to NASH.
According to the FDA draft guidance for NASH fibrosis, of the histologic features of NASH, fibrosis is considered the strongest predictor of adverse clinical outcomes, including liver-related death.
There is currently no approved therapy for this devastating disease, which has become a leading cause of liver failure and resulting poor clinical outcomes.
ICPT closed at $77.49, last traded at $48.00, down 38%.
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