Headquartered in New York City, Intercept Pharmaceuticals (NASDAQ:ICPT) announced the receipt of a Complete Response Letter (CLR) from the U.S. Food and Drug Administration (FDA) regarding its marketing application which sought the approval to use obeticholic acid (OCA) to treat fibrosis due to nonalcoholic steatohepatitis (NASH).

NASH is a liver disease caused by excessive fat accumulation in the liver that can lead to cirrhosis, liver failure, cancer, and death.

The company’s stock plunged around 40% in midday trading on June 29th in reaction to the unfavorable response.

Intercept Pharmaceuticals SEC Filings

The FDA cited the uncertainty of a predicted treatment benefit based on a surrogate histopathologic endpoint. Based on its review of the OCA data, the FDA decided that the potential benefits of the treatment were offset by the potential patient risks. In declining to approve the New Drug Application (NDA), the therapy fell short of becoming the first drug on the market for obesity-linked liver disease.

Intercept is a mid-cap biopharmaceutical company that develops novel therapeutics for the treatment of progressive, non-viral liver diseases including NASH and biliary cholangitis.

Hope Remains for Further Advancement

Yet all hope is not lost for the company. The FDA also recommended that Intercept submit additional post-interim analysis efficacy and safety data from its ongoing REGENERATE study to support the potential accelerated approval of OCA. It also urged the company to continue the phase of the study related to long-term outcomes.

President and CEO Mark Pruzanski commented, “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.”

Pruzanski also expressed disappointment that the FDA’s decision was made without input from Intercept’s medical experts and patients at the forthcoming Adcom event calling it “an apparently incomplete review”.

OCA has been designated a Breakthrough Therapy by the FDA and is the only drug candidate that has had some success in a pivotal Phase 3 study. The FDA has reportedly increased the complexity of the study’s endpoints making it harder for Intercept to hit a challenging, moving target.

The company’s NDA was the first submission to the FDA targeting the use of OCA as a treatment for NASH. It was based on data from 35 separate clinical trials encompassing over 1,700 NASH patients that were treated with the investigational drug. Results from these trials have shown OCA’s ability to reverse or stabilize liver fibrosis in patients with advanced fibrosis due to NASH.

Looking ahead

Management noted its plans to meet with the FDA to review the CRL and explore approval options moving forward. Despite the setback, Intercept may still be able to advance its OCA treatment depending on the outcome of further FDA discussions and potential studies. With no approved therapy on the market for fibrosis due to NASH, the company maintains the potential to commercialize a treatment.

Intercept Pharmaceuticals SEC Filings


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