EMA’s human medicines committee (CHMP) has concluded its review of the medicine Ocaliva (obeticholic acid) and has recommended that the medicine’s marketing authorisation be revoked, because its benefits are no longer considered to outweigh its risks. Ocaliva is used to treat adults with primary biliary cholangitis (PBC), an autoimmune condition that causes gradual destruction of the bile ducts in the liver, which can lead to liver failure and increase the risk of liver cancer. Ocaliva is used together with another medicine, ursodeoxycholic acid (UDCA), in patients who do not respond sufficiently to UDCA alone, and on its own in patients who cannot take UDCA.
At the time of its conditional marketing authorisation in 2016, Ocaliva was shown to reduce the blood levels of alkaline phosphatase (ALP) and bilirubin (markers of liver damage) in patients with PBC, and this was considered indicative of an improvement in the condition of the liver. However, the clinical benefits of Ocaliva needed to be demonstrated in further studies, which were requested by EMA as part of the conditions to the marketing authorisation of the medicine. In particular, study 747-302 was a randomised clinical trial aimed at confirming the clinical benefits and safety of Ocaliva in patients for whom ursodeoxycholic acid (UDCA, another medicine for PBC) does not work well enough, or who cannot take UDCA.
The CHMP has now reviewed the findings from this study, alongside other available data including real-world data and data from supportive studies submitted by the company that markets Ocaliva, and information submitted by healthcare professional and patient associations. In addition, the CHMP took into account the feedback from a group of experts in liver disease, which provided their views on specific questions posed by the CHMP, and views from people with experience living with PBC. After reviewing the available evidence, the committee concluded that the clinical benefits of Ocaliva have not been confirmed. In particular, study 747-302 failed to show that Ocaliva was more effective than placebo (a dummy treatment) in terms of the number of patients whose disease worsened or who died, both in the overall population and in a group of patients with early stage PBC. The committee also considered that the data from supportive studies and real-world data were not sufficient to confirm the benefits of Ocaliva and could not counterbalance the negative results of study 747-302. The CHMP therefore concluded that the benefits of Ocaliva do not outweigh its risks and recommended that its marketing authorisation be revoked in the European Union (EU).
EMA will now send the CHMP opinion to the European Commission, which will issue in due course a final legally binding decision applicable in all EU Member States.
Learn more at: EMA recommends revoking conditional marketing authorisation for Ocaliva | European Medicines Agency (europa.eu)
