FDA Grants Breakthrough Therapy Designation to Obeticholic Acid for NASH

The FDA has granted breakthrough therapy designation to Intercept Pharmaceutical’s obeticholic acid, a potential therapy to treat nonalcoholic steatohepatitis with liver fibrosis, according to a news release from the drugmaker.

Obeticholic acid is a bile acid analog and first-in-class agonist of the farnesoid X receptor, according to the release. It is currently being developed for nonalcoholic steatohepatitis (NASH), primary biliary cirrhosis and primary sclerosing cholangitis.  

“We are very pleased to have received breakthrough therapy designation for NASH with liver fibrosis, as it will enable us to work closely with the FDA to finalize the design of our phase 3 program,” Mark Pruzanski, MD, president and chief executive officer of Intercept, said in the release. “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 [obeticholic acid] has the potential to be an important treatment option for patients with no currently approved medicines.”

The designation comes after results from two phase 2 clinical trials, including the FLINT trial, showed patients with NASH treated with obeticholic acid had significant improvements in the primary histological endpoint of nonalcoholic fatty liver disease Activity Score, as well as steatosis, lobular inflammation, hepatocellular ballooning and fibrosis.

The FDA granted Intercept fast track designation for obeticholic acid in May 2014.

Disclosure: Pruzanski is an employee of Intercept Pharmaceuticals.