Shares of Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) are up more than 12.60% to $194 in after hours trading, as traders react to the company’s announcement that its investigational product obeticholic acid (OCA) has received “breakthrough therapy designation” from the FDA for the treatment of patients with nonalcoholic steatohepatitis (NASH) with liver fibrosis. This indication constitutes a population of patients with a serious and life-threatening condition reflected by a higher risk of progression to cirrhosis and liver failure. OCA is currently being developed for the treatment of several chronic liver diseases, including primary biliary cirrhosis (PBC), NASH and primary sclerosing cholangitis (PSC).
“We are very pleased to have received breakthrough therapy designation for NASH with liver fibrosis, as it will enable us to work closely with FDA to finalize the design of our Phase 3 program,” said Mark Pruzanski, M.D., President and Chief Executive Officer of Intercept. “This designation underscores a recognition of the urgent need to bring novel treatments to NASH patients who have developed liver fibrosis, which is expected to make this serious disease the leading cause for liver transplant in just the next few years. As a first-in-class FXR agonist, we believe OCA has the potential to be an important treatment option for patients with no currently approved medicines.”
Breakthrough therapy designation for OCA was based on clinical efficacy and safety data from two placebo-controlled, Phase 2 clinical trials of OCA. A Phase 2 trial in 64 patients with nonalcoholic fatty liver disease (NAFLD) assessed the impact of OCA treatment on insulin sensitivity (published in Gastroenterology in June 2013). The FLINT Phase 2 trial in 283 patients with NASH assessed the impact of OCA treatment on liver histology and fibrosis (published in The Lancet in November 2014).
The breakthrough therapy designation was created by the FDA to speed the availability of new therapies for serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation confers several benefits, including intensive FDA guidance and discussion and eligibility for submission of a rolling NDA.