OCA Plus Bezafibrate Gets FDA Orphan Drug Approval for PBC

The U.S. Food and Drug Administration (FDA) on May 16 granted its orphan drug designation for the fixed-dose combination of obeticholic acid (OCA), a farnesoid X receptor (FXR) agonist, and bezafibrate, a peroxisome proliferator-activated receptor (PPAR) agonist, for the treatment of primary biliary cholangitis (PBC).

The FXR regulates bile acid synthesis and transport, while PPARs can inhibit fibrogenesis of liver cells. By targeting both pathways, the combination of these drugs is projected to help control the factors that contribute to PBC—a progressive autoimmune disease that causes liver inflammation and fibrosis.

The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S.

However, the Gastrointestinal Drugs Advisory Committee on May 19 voted 12-2 that the 25-mg dose of OCA do not outweigh the risks of drug-induced liver injury despite improvements in liver scarring that were revealed in the REGENERATE trial. The committee also recommended against accelerated approval for OCA, advising the FDA to wait to see additional data from an ongoing phase 3 trial.

—Rebecca Mashaw

References:

Intercept Pharmaceuticals receives FDA orphan drug designation for the fixed-dose combination of OCA and bezafibrate for the treatment of primary biliary cholangitis (PBC). News release. Intercept Pharmaceuticals; May 16, 2023. Accessed June 5, 2023. https://ir.interceptpharma.com/news-releases/news-release-details/intercept-pharmaceuticals-receives-fda-orphan-drug-designation

FDA advisory committee votes against approval for NASH drug. Medscape. May 19, 2023. Accessed June 5, 2023. https://www.medscape.com/viewarticle/992236?reg=1&icd=login_success_email_match_norm