The European Medicines Agency’s committee for human medicines (CHMP) has made a critical decision regarding the medicine Ocaliva (obeticholic acid), recommending that its marketing authorization be revoked across the European Union. This decision comes after a thorough review concluded that the benefits of Ocaliva no longer outweigh its risks for the treatment of primary biliary cholangitis (PBC), an autoimmune liver condition.
Ocaliva was initially granted conditional marketing authorization in 2016 based on its ability to reduce blood levels of markers like alkaline phosphatase (ALP) and bilirubin, indicating potential improvement in liver health for PBC patients. However, further studies were required to confirm its clinical benefits.
The pivotal study, known as 747-302, aimed to verify Ocaliva’s effectiveness and safety in PBC patients who either did not respond adequately to or could not tolerate another medication, ursodeoxycholic acid (UDCA). Disappointingly, this trial failed to demonstrate Ocaliva’s superiority over a placebo in preventing disease progression or death in both the overall patient population and those in early stages of PBC.
In reaching its decision, the CHMP considered extensive data, including real-world evidence and input from healthcare professionals, patients, and liver disease experts. Despite additional supportive studies submitted by the manufacturer, these did not counterbalance the negative outcomes of study 747-302.
As a result, the CHMP concluded that the risks associated with Ocaliva outweigh its unproven benefits in treating PBC. The recommendation for revocation of its marketing authorization will now be forwarded to the European Commission, which will issue a final decision applicable to all EU member states.
For patients currently using Ocaliva, healthcare professionals are advised to consider alternative treatment options. The EMA will provide further guidance through direct communication channels to ensure patient safety during this transition.
Ocaliva, an orphan medicine designated for rare diseases, remains available for compassionate use or through named-patient programs for existing patients until the final decision takes effect.
This recommendation underscores the rigorous evaluation process undertaken by regulatory authorities to ensure the safety and efficacy of medicines available to patients across Europe.