Obeticholic Acid in Europe, USA and Canada

Obeticholic Acid in Europe, USA and Canada


PBCers and Canadian PBC Society Press Release for PBC Patients: Important Information for PBC Patients Regarding OCALIVA (OCA) European Medicines Agency (EMA) Ruling. 

On June 28, 2024 the Committee for Medicinal Products for Human Use (CHMP),  issued an opinion to the European Commission to revoke the marketing authorization for OCALIVA in Europe.

The PBC Community responds

The PBCers Organization, together with the Canadian PBC Society and our patient advocate colleagues around the world, as well as PBC healthcare specialists are concerned that CHMP did not consider the totality of evidence regarding PBC patients’ needs and real-world experiences with OCALIVA. Regulators, researchers, clinicians and patients alike understand that inadequate response to treatment over time leads to life-altering outcomes, which is why PBC treatments have been receiving fast-track approval.

There needs to be a broader acknowledgment that patient voices and experiences also represent forms of empirical evidence, which is why the PBC patient community and the Global PBC Study Group have been working diligently for years to propose real world data alternatives to traditional clinical studies.

The FDA (Food and Drug Administration) and Health Canada are currently undertaking independent reviews of longer term outcomes in patients taking OCALIVA and we urge them to fully consider the totality of data, including real-world patient evidence in their deliberations.  Furthermore, it is our hope that these factors will be fully considered by the European Commission in reaching their final decision.

What does this mean for patients in the USA and Canada?

We believe it’s important to make sure that PBC patients are aware of these developments, and to reassure you that OCALIVA can continue to be prescribed to new and existing PBC patients.

Remember, your health is unique. Always talk to your doctor about what’s best for you and your PBC. They know your specific situation and can give you the best advice.

We will continue to keep you informed of any new developments as these reviews progress. If you have questions, don’t hesitate to ask your doctor, and stay tuned to www.PBCers.org and www.pbc-society.ca for more information on this review

Why is this happening?

  • OCALIVA was initially approved in Europe with the condition that additional long-term studies would be completed.
  • After 5 years, the COBALT study was stopped early in 2021 due to recruitment challenges and difficulty retaining a placebo group when patients had access to OCALIVA .
  • The early termination of this study means that some important long-term data is unavailable and contributed to the EMA recommendation.

What does the EMA recommendation mean?

  • This is a recommendation, not a final decision. The European Commission will make the final decision.
  • The final decision will be legal in Europe.
  • In the coming months the FDA and Health Canada will review the additional data and make a decision regarding final approval of OCALIVA in the USA and Canada.

Can I still take OCALIVA?

  • Yes, if you’re in the USA or Canada, you can continue taking OCALIVA as your doctor prescribed.
  • Don’t stop taking it without talking to your doctor first.

Is OCALIVA safe?

  • OCALIVA has been used for more than 8 years since it was approved across North America and Europe.
  • With many years of real-world patient experience, OCALIVA’s safety profile is well characterized.
  • Doctors and researchers have been studying its safety all along.
  • Like all medications, patients may experience side effects which your doctor will discuss with you.

Does OCALIVA work?

  • Studies show that OCALIVA helps patients with PBC by lowering the levels of toxic bile acids in the liver, resulting in lower ALP and bilirubin levels.  This may slow or prevent progression of PBC.
  • Researchers have looked at how patients do in the real world, not just in clinical trials and continue to prescribe OCALIVA to their patients based on the real-world benefits they have seen.

What should I do?

  • Keep taking your medicine as prescribed.
  • Talk to your doctor if you have any concerns.
  • Share your experience with OCALIVA with your doctor.
  • Stay informed about any news from the FDA, Health Canada and PBC patient groups.

How can I help?

  • Join a PBC patient group if you haven’t already. See www.PBCers.org and www.pbc-society.ca
  • Share your experience with OCALIVA. Your story matters and can help researchers.
  • Ask your doctor about any PBC research studies you might join.

What’s next?

  • The FDA and Health Canada will continue reviewing OCALIVA with decisions expected in the coming months.
  • Researchers will keep studying how OCALIVA works for patients over time.
  • We’ll share any important updates about OCALIVA when we have them.
  • Patient organizations will continue to advocate for safe and effective treatment options for all PBC patients.


Carol Roberts, President PBCers 

Gail Wright, President Canadian PBC Society


Additional Resources

PBC Foundation: Obeticholic Acid in Europe

EMA recommendation