Intercept Pharmaceuticals Inc. (ICPT) announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use or CHMP adopted a positive opinion recommending marketing authorization of the Company's Marketing Authorization Application (MAA) for obeticholic acid or OCA, an FXR agonist, for the treatment of primary biliary cholangitis or PBC conditional to the company providing further data post-approval to confirm benefit.
Ursodeoxycholic acid or UDCA is currently the only approved medication for the treatment of PBC in Europe and is the standard of care for all PBC patients. However, a substantial percentage of patients treated with UDCA continue to experience persistent elevations above the upper limit of normal in the serum marker alkaline phosphatase (ALP), which has been shown to correspond with increased risk of liver failure, need for liver transplant and death. Patients with PBC also face a risk of experiencing adverse outcomes when bilirubin levels are elevated. Total bilirubin levels, even within the normal range, have been shown to predict clinical outcomes in PBC.
The MAA submission included data from more than 1,500 subjects exposed to at least a single dose of OCA. The positive opinion of the CHMP was based on efficacy and safety data derived from three randomized double-blind, placebo-controlled clinical trials in patients with PBC evaluating the effect of OCA on ALP and bilirubin. The MAA submission was 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.
The CHMP opinion will form the basis for a European Commission (EC) decision as to whether to formally grant the conditional marketing authorization for OCA with unified labelling in the 28 countries that are Member States of the European Union, as well as European Economic Area members Iceland, Liechtenstein and Norway. As the conditions for approval, Intercept is required to provide post-approval updates on safety and efficacy analyses for OCA from the ongoing COBALT outcomes trial and a short-term trial in patients with hepatic impairment.
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