Credit: US Food and Drug Administration

The US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the New Drug Application (NDA) of obeticholic acid (OCA) for patients with pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH).¹

The treatment, developed by Intercept Pharmaceuticals, could not be approved in its present form, according to the CRL and any resubmission of the treatment for patients with NASH would require at least the successful completion of the long-term outcomes phase of the REGENERATE study.

However, because of the CRL, Intercept announced they would discontinue all NASH-related investments.

“While this is clearly not the outcome that we have worked toward, I’m proud of the impact that Intercept has made to move the science of NASH forward and bring the field closer to a treatment option,” said Jerry Durso, President and Chief Executive Officer of Intercept, in a statement. “We believe that taking decisive action to reshape Intercept will improve our long-term ability to grow our business, innovate for patients, and create value for shareholders. We remain committed to the liver community and will continue to advance our leadership in rare and serious liver diseases where Intercept has deep expertise and a recognized dedication to therapeutic innovation.”

In past studies, the drug has shown safety and efficacy in the phase 3 REGENERATE study of patients with pre-cirrhotic liver fibrosis due to NASH. The results show OCA 25 mg consistently demonstrated double the response rate of placebo in reduction in liver fibrosis stage without worsening of any of the three histologic components of NASH—fat, inflammation, and fibrosis.²

In addition, there are a number of classes of medications currently being explored as viable treatments for NAFLD and NASH, largely because the actual pathway that leads from fat in the liver to inflammation to fibrosis is extremely complicated.

References:

Intercept Pharmaceuticals, Inc. (2023, June 22). Intercept receives complete response letter from FDA for Obeticholic acid as a treatment for pre-cirrhotic fibrosis due to Nash. GlobeNewswire News Room. https://www.globenewswire.com/news-release/2023/06/22/2693358/0/en/Intercept-Receives-Complete-Response-Letter-from-FDA-for-Obeticholic-Acid-as-a-Treatment-for-Pre-Cirrhotic-Fibrosis-due-to-NASH.html

Often neglected, hope is on the way for fatty liver disease patients. HCP Live. (n.d.). https://www.hcplive.com/view/often-neglected-hope-fatty-liver-disease-patients