Outrage Over EMA's Recommendation to Revoke Ocaliva Authorization

The European Medicines Agency (EMA) human medicines committee has sparked significant backlash from patients and advocacy groups by recommending the revocation of market authorization for obeticholic acid, marketed as Ocaliva. This medication is used to treat primary biliary cholangitis (PBC), a rare and progressive autoimmune liver disease. The EMA's decision, announced on June 28, has been criticized for allegedly disregarding real-world data and relying on a flawed clinical trial that led to high dropout rates among participants seeking access to the drug. The PBC Foundation has taken a stand against the EMA's recommendation, urging the European Commission to reconsider the decision. Robert Mitchell-Thain, the foundation's chief executive, emphasized the need for a review of real-world evidence that they believe demonstrates the drug's clinical benefits. A protest was organized near the European Commission's headquarters on July 25, highlighting the community's demand for a thorough evaluation of the drug's efficacy. Ocaliva was initially granted conditional marketing authorization in 2016, contingent upon the results of a randomized clinical trial. However, the EMA's Committee for Medicinal Products for Human Use (CHMP) concluded that the clinical benefits of the drug were not sufficiently confirmed, despite support from the European Association for the Study of the Liver (EASL), which cited significant improvements in patients' health metrics. As the European Commission prepares to make a decision on the EMA's recommendation within the mandated 67-day timeframe, it has expressed confidence in the EMA's scientific assessments. Meanwhile, the EMA has allowed continued access to Ocaliva through compassionate use programs for patients already on the medication.