The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to pipeline product obeticholic acid (OCA), developed by Intercept Pharmaceuticals, Inc. for the treatment of nonalcoholic steatohepatitis (NASH) with liver fibrosis.
Intercept Pharmaceuticals has been studying obeticholic acid and hopes to advance it as a treatment for chronic liver diseases, such as primary biliary cirrhosis (PBC), primary sclerosing cholangitis (PSC) and nonalcoholic steatohepatitis.
“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 the president and CEO of Intercept, Mark Pruzanski, M.D.
“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,” Pruzanski added.
The designation of breakthrough therapy is granted by the FDA to accelerate the transition of novel therapeutic options for the treatment of severe or life-threatening diseases. The administration grants this status to investigational drugs with promising evidence of an ability to provide better outcomes than existing therapies, and for conditions with no therapy currently available.
In the case of OCA, the status was granted on the basis of two Phase 2 clinical trials that revealed the clinical efficacy and safety of the drug compared to placebo. The first study included 64 patients with nonalcoholic fatty liver disease (NAFLD), in which the researchers evaluated the impact of the therapy with OCA on insulin sensitivity. This was published in June 2013 in the journal, Gastroenterology. The other one, called the FLINT phase 2 trial, which included 283 NASH patients, was published last November in The Lancet.
The breakthrough therapy designation grants OCA several benefits, such as guidance from the FDA and eligibility for submission of a rolling NDA. OCA, which is a bile acid analog and a first-in-class agonist of the farnesoid X receptor (FXR), had already been granted the FDA’s fast track designation to treat primary biliary cirrhosis, and orphan drug designation in both the United States and Europe to treat primary biliary cirrhosis and primary sclerosing cholangitis.