NEW YORK, Dec. 13, 2019 (GLOBE NEWSWIRE) -- 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 it has submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for obeticholic acid (OCA) for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH). The MAA submission is supported by the positive interim analysis results from the pivotal Phase 3 REGENERATE study in patients with liver fibrosis due to NASH.
Intercept also announced that the U.S. Food and Drug Administration (FDA) has notified the company of the tentative date for the previously announced advisory committee meeting (AdCom) related to Intercept’s New Drug Application (NDA) for OCA in liver fibrosis due to NASH. The FDA has tentatively scheduled the AdCom for April 22, 2020. Intercept anticipates that the FDA accordingly will extend the recently announced March 26, 2020 Prescription Drug User Fee Act (PDUFA) target action date for Intercept’s NDA. Intercept previously announced the FDA’s acceptance of the NDA and granting of priority review.
“NASH is quickly becoming one of the most significant public health challenges in Europe and costs associated with advanced fibrosis and cirrhosis are estimated to represent approximately 95% of the total NASH related costs to health care systems,” said Mark Pruzanski, M.D., President and Chief Executive Officer of Intercept. “We believe that OCA has the potential to become an essential treatment for people living with advanced fibrosis due to NASH and we look forward to working with the EMA during this review period. Separately, we are pleased that FDA has provided us with the tentative AdCom date and we look forward to working collaboratively with the agency during its review of the NDA as we continue to prepare for our anticipated NASH launch, if approved, within the first half of 2020.”
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. NASH is anticipated to become the leading indication for liver transplantation in Europe within the next decade. 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 interim analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. The intent-to-treat population for the interim analysis included 931 patients with stage 2 and 3 fibrosis (placebo, n=311; OCA 10 mg, n=312; OCA 25 mg, n=308). REGENERATE has completed target enrollment for the clinical outcomes cohort, with 2,480 adult NASH patients randomized at 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.
The safety population of the interim analysis included 1,968 randomized patients who received at least one dose of investigational product (OCA or placebo). Adverse events were generally mild to moderate in severity and the most common were consistent with the known profile of OCA. The frequency of serious adverse events was similar across treatment arms (11% in placebo, 11% in OCA 10 mg and 14% in OCA 25 mg). The most common adverse event reported was dose-related pruritus (placebo, 19%; OCA 10 mg, 28%; OCA 25 mg, 51%). The large majority of pruritus events were mild to moderate, with severe pruritus occurring in a small number of patients.
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.
