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Orphan drugs

European conditional approval given for rare liver disease drug

The European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended a new drug to treat a rare and potentially lethal liver disease for conditional marketing approval across the EU

Obeticholic acid (Ocaliva; Intercept Pharma) has been recommended as a combination therapy with ursodeoxycholic acid (UDCA) for patients with primary biliary cirrhosis. It is a disease which causes the gradual destruction of the liver’s small bile ducts which can eventually lead to liver failure and increase the risk of liver cancer. 

The recommendation states that the drug should only be used in cases where patients have not responded to treatment with UDCA, or as a monotherapy when patients are unable to tolerate UDCA.

The CHMP gave obeticholic acid a positive opinion at its October 2016 meeting. The positive opinion was based on the results of a phase lll clinical trial which involved 216 patients, which studied alkaline phosphatase levels, an indicator of disease progression.

The proposed marketing authorisation for the drug — which already has orphan drug status in recognition of its potential to address a rare disease — is on condition that the manufacturer supplies the EMA with data from two ongoing clinical trials.

The recommendation will now go to the European Commission for approval.

Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2016.20201843

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Supplementary images

  • Magnification of primary biliary cirrhosis

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