The European Medicines Agency's (EMS's) recent recommendation to revoke market authorization for Ocaliva, a treatment for primary biliary cholangitis (PBC), a progressive autoimmune liver disease leading to cancer, has sparked controversy and significant backlash from patients and advocacy groups.
The CHMP advised against the continued use of obeticholic acid, marketed as Ocaliva, following a review of clinical trial results and expert feedback. The committee argued that the drug’s clinical benefits were not sufficiently confirmed.
PBC damages bile ducts, potentially leading to liver failure or an increased risk of liver cancer. Ocaliva, which received conditional marketing authorization in 2016, was approved with the stipulation that a subsequent clinical trial would confirm its effectiveness.
In response to the CHMP’s recommendation, the PBC Foundation organized a protest on July 25 outside the European Commission’s headquarters in Brussels to pressure the Commission to reject the CHMP’s recommendation, emphasizing the importance of real-world evidence in assessing the drug’s efficacy.
Critics of the CHMP’s decision have raised concerns about the randomized controlled trial, noting that many participants opted for the active drug rather than a placebo.
Ocaliva remains a vital treatment option for patients who do not respond adequately to ursodeoxycholic acid (UDCA), another approved therapy for PBC.
The European Association for the Study of the Liver (EASL) has supported the PBC Foundation’s stance, citing real-world evidence that demonstrates significant improvements in biochemical markers and reduced clinical events in early-stage PBC patients.
A source from the European Commission confirmed that it had received the EMA’s assessment and would make a decision within the 67-day legislative deadline. While the Commission has refrained from further comment, it expressed confidence in the EMA’s scientific evaluations.
If the Commission upholds the EMA’s recommendation, Ocaliva will no longer be authorized for use in the EU. However, the EMA has allowed for the continued supply of the medicine through compassionate use or limited named-patient programs for those currently benefiting from it.