Intercept files new drug application with FDA for obeticholic acid

Intercept Pharmaceuticals, Inc. has submitted a new drug application to the FDA for obeticholic acid to be used in combination with ursodeoxycholic acid to treat primary biliary cirrhosis, according to a press release.

Obeticholic acid (OCA) will be intended for the treatment of primary biliary cirrhosis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who did not respond to previous UDCA therapy or as monotherapy in adults unable to tolerate UDCA, according to the release.

UDCA is the current standard of care for all patients with PBC, as it is the only approved regimen. However, the release stated that patients are experiencing elevated alkaline phosphatase levels.

These elevated levels correspond with “increased risk of liver failure, need for liver transplant and death,” the release said. “Thus, there continues to be a critical need for new treatments for patients with PBC.”

In addition to the application with the FDA, Intercept also announced that the European Medicines Agency accepted its marketing authorization application for OCA, according to the release.

“Over the past several years, the medical community has gained a deeper appreciation of the extent of the unmet medical need in PBC,” Mark Pruzanski, MD, president and CEO of Intercept, said in the release. “In each of our PBC clinical trials, OCA has demonstrated the ability to rapidly and sustainably lower ALP and improve bilirubin levels, both when added to UDCA and as monotherapy. If approved, we believe OCA will become an important new treatment option that will help patients with PBC.”

Both the new drug application and marketing authorization come after data from clinical trials showed OCA to be safe, effective and well-tolerated across 1,507 patients.

“The key efficacy and safety data are derived from three randomized double-blind, placebo-controlled clinical trials in patients with PBC evaluating the effect of OCA on ALP and bilirubin,” the release said. “All three trials met their primary endpoints with high statistical significance and improvements were seen in secondary endpoints including markers of liver injury, immunity, inflammation and apoptosis.”

Disclosures: Pruzanski reports being employed by Intercept.