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Primary Biliary Cholangitis Treatment Ocaliva Faces Revocation of EMA Authorization

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The European Medicines Agency (EMA) has recommended revoking the conditional marketing authorization for primary biliary cholangitis (PBC) treatment Ocaliva (obeticholic acid) after concluding that the benefits of the medicine no longer outweigh its risks. Ocaliva, used to treat adults with primary biliary cholangitis, an autoimmune condition that causes gradual destruction of the bile ducts in the liver, has not demonstrated sufficient clinical benefits in recent studies to justify its continued use.

Ocaliva was initially granted conditional marketing authorization in 2016 based on evidence that it reduced blood levels of alkaline phosphatase (ALP) and bilirubin, markers of liver damage, in patients with primary biliary cholangitis. This reduction was considered indicative of potential improvements in liver condition. However, the authorization required further studies to confirm the clinical benefits and safety of Ocaliva.

The Study Finds Ocaliva Ineffective for Primary Biliary Cholangitis

A critical study, 747-302, aimed to confirm the benefits of Ocaliva for patients who do not respond well to or cannot take ursodeoxycholic acid (UDCA). The study compared Ocaliva with a placebo and found no significant difference in terms of disease progression or mortality rates among patients.

The study’s failure to demonstrate Ocaliva’s effectiveness, coupled with data from other studies and real-world evidence, led the EMA’s human medicines committee (CHMP) to conclude that the benefits of Ocaliva do not outweigh its risks. The committee reviewed various sources of data, including study 747-302, real-world data, supportive studies submitted by the company marketing Ocaliva, and feedback from healthcare professionals and patient associations.

Expert opinions on liver disease and the experiences of people living with primary biliary cholangitis were also considered. Despite these comprehensive reviews, the data did not confirm the clinical benefits of Ocaliva. The medicine did not prove to be more effective than placebo in preventing disease progression or reducing mortality rates in the overall population or patients with early-stage primary biliary cholangitis.

Primary Biliary Cholangitis

EMA Recommends Revoking Ocaliva’s Authorization, Final Decision Pending from European Commission

The decision to recommend revoking Ocaliva’s marketing authorization will now be forwarded to the European Commission, which will issue a final legally binding decision applicable to all EU Member States. This decision reflects the EMA’s commitment to ensuring that only medicines with a positive benefit-risk balance are available to patients.

For patients currently taking Ocaliva, it is essential to consult with healthcare professionals to discuss alternative treatment options. The company may continue to supply Ocaliva through compassionate use or named-patient programs for those already receiving the medicine. Healthcare professionals are advised not to start any new patients on Ocaliva outside of clinical trials.

The EMA’s recommendation to revoke the marketing authorization for Ocaliva highlights the importance of continuous monitoring and reassessment of medicines to ensure patient safety. By prioritizing evidence-based decisions, the EMA aims to protect public health and maintain trust in the regulatory system.

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Resource: European medicines agency, June 28, 2024

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