INT 747 (obeticholic acid) filed with FDA and EMA for primary biliary cirrhosis- Intercept Pharmaceuticals

Intercept Pharmaceuticals announced two regulatory milestones for INT 747 (obeticholic acid or OCA) in primary biliary cirrhosis (PBC): submission of a New Drug Application for accelerated approval to the FDA and acceptance of the Marketing Authorization Application by the European Medicines Agency (EMA). Intercept is seeking approval of OCA for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. UDCA is currently the only approved medication for the treatment of PBC and is the standard of care for all PBC patients.

The submissions include a total of 1,507 subjects exposed to at least a single dose of OCA. Of these subjects, 432 were patients with PBC, with 290 treated for at least six months and 232 treated for at least one year. 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. 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.

OCA treatment was generally well-tolerated, with primarily mild or moderate pruritus being the most common adverse event. The regulatory submissions are also supported by two clinical databases that include more than 10,000 patients from the Global PBC Study Group and UK-PBC Group, both independently confirming that achieving lower ALP and/or bilirubin levels is significantly correlated with increased transplant-free survival.