CHMP starts review of Intercept’s Ocaliva

The European Medicines Agency’s human medicines committee has started a review of Intercept’s Ocaliva, used to treat adults with primary biliary cholangitis. PBC is an autoimmune condition that causes gradual destruction of the small bile ducts in the liver, which can lead to liver failure and increase the risk of liver cancer. The review was prompted by the final results from two studies in patients with PBC, which were requested by EMA in 2016 as part of the conditions to the initial marketing authorisation of Ocaliva. Study 747-302 was designed to confirm the benefits and safety of Ocaliva, while study 747-401 assessed the safety of Ocaliva in patients with advanced liver disease. In particular, study 747-302 failed to show that Ocaliva was more effective than placebo in terms of the number of patients whose disease worsened or who died. In addition, side effects, including serious ones, occurred more frequently in patients treated with Ocaliva. EMA will now review these findings alongside all other available data and assess their impact on the overall benefit-risk balance of Ocaliva. The Agency will then make a recommendation on whether the medicine’s marketing authorisation in the EU should be amended.