FDA Accepts Intercept’s NDA for OCA for the Treatment of Liver Fibrosis Due to NASH and Grants Priority Review

Posted on November 25, 2019 by Medtech[y] Staff

Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted Intercept’s New Drug Application (NDA) for obeticholic acid (OCA) seeking accelerated approval for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH) and granted priority review. The FDA grants priority review to drugs that have the potential to treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness.

“If approved, OCA would be the first available therapy for patients with fibrosis due to NASH, a condition that is expected to become the leading cause of liver transplant in the U.S. as soon as 2020,” said Mark Pruzanski, M.D., President and Chief Executive Officer of Intercept. “It is exciting to achieve this critical regulatory milestone that brings us one step closer to our goal of delivering the first approved therapeutic to those living with this devastating disease. From OCA’s prior designation as a Breakthrough Therapy to the grant of priority review today, our work with FDA continues to set an important precedent for the field, and we look forward to working with the agency over the coming months as they review the first NDA in NASH.”

The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 26, 2020 for the NDA. In the NDA filing acceptance notification letter, the FDA also indicated that it currently plans to hold an advisory committee meeting to discuss the application. A date for the advisory committee meeting has not been finalized and the timeline for the review of the NDA by the FDA remains subject to change.

OCA is the only investigational therapy to have received Breakthrough Therapy designation from the FDA for NASH with fibrosis. The NDA filing for OCA is supported by positive interim analysis results from the pivotal Phase 3 REGENERATE study in patients with liver fibrosis due to NASH. In the study, OCA 25 mg demonstrated robust improvement in liver fibrosis (by ≥1 stage) with no worsening of NASH at 18 months.

About Liver Fibrosis due to NASH

Nonalcoholic steatohepatitis (NASH) is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death. Advanced fibrosis is associated with a substantially higher risk of liver-related morbidity and mortality in patients with NASH and, as early as 2020, the disease is projected to become the leading cause of liver transplants in the United States. There are currently no medications approved for the treatment of NASH.

About the REGENERATE Study

REGENERATE is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study assessing the safety and efficacy of obeticholic acid (OCA) on clinical outcomes in patients with liver fibrosis due to NASH. A pre-specified 18-month analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. REGENERATE has completed target enrollment for the clinical outcomes cohort, with more than 2,400 adult NASH patients randomized across 339 qualified centers worldwide, and will continue through clinical outcomes for verification and description of clinical benefit. The end-of-study analysis will evaluate the effect of OCA on all-cause mortality and liver-related clinical outcomes, as well as its long-term safety.

About Accelerated Approval

The FDA grants accelerated approval under subpart H for drugs that address serious or life-threatening illnesses and appear to provide meaningful therapeutic benefit to patients over existing treatments on the basis of an adequate and well-controlled clinical trial demonstrating that the drug has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit. Drugs granted accelerated approval must meet the same standards for safety and effectiveness as those granted traditional approval and are required to be further evaluated on a post-marketing basis. Approval of a drug may be withdrawn by the FDA if clinical benefit cannot be verified or sufficiently demonstrated.

About Intercept

Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Founded in 2002 in New York, Intercept has operations in the United States, Europe and Canada. For more information, please visit www.interceptpharma.com or connect with the company on Twitter and LinkedIn.