EMA recommends Ocaliva, with conditions, for PBC

Ocaliva received recommendation for a conditional marking authorization in Europe for the treatment of primary biliary cholangitis, according to a release from the European Medicines Agency.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended Ocaliva (obeticholic acid, Intercept Pharmaceuticals) to be used in combination with ursodeoxycholic acid (UDCA) in those patients with PBC who did not respond to UDCA. Additionally, in those intolerant of UDCA, obeticholic acid can be used as a single agent. Up to 50% of the patients with PBC fall into one of these two categories of nonresponders or intolerance to UDCA, according to the release.

These recommendations reflect the parameters set forth in the FDA approval from May 2016.

“Although it is a rare disease, PBC remains one of the most common indications for liver transplant among women in Europe,” David Jones, MD, PhD, professor of liver immunology at Newcastle University and consultant hepatologist at Newcastle upon Tyne Hospitals Trust, said in a release from the manufacturer. “There is substantial unmet need in this disease and a real urgency around the need for new therapies to help the many PBC patients who are either intolerant of the single existing approved therapy ursodeoxycholic acid or don't respond to it sufficiently to protect their livers and prevent the development of cirrhosis and the need for transplant.”

A phase 3 study of 216 participants produced a greater reduction in alkaline phosphatase in patients treated with obeticholic acid as compared to placebo (47% vs. 10%), according to the EMA. Outlined in the conditional authorization, the manufacturer must provide results from two additional studies and, until those results are finalized, a risk-benefit analysis will occur annually to determine maintenance of the conditional authorization.

“The opinion adopted by the CHMP at its October 2016 meeting is an intermediary step on Ocaliva’s path to patient access. The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorization. Once a marketing authorization has been granted, a decision on price and reimbursement will then take place at the level of each Member State considering the potential role/use of the medicine in the context of the national health system of that country,” according to the release. – Katrina Altersitz

Reference: European Medicines Agency. 2016. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/10/news_detail_002618.jsp&mid=WC0b01ac058004d5c1. Accessed: October 14, 2016.

Disclosure: Jones has a financial relationship with Intercept Pharmaceuticals.