Ocaliva (obeticholic acid) is used to treat adults with primary biliary cholangitis, an autoimmune condition in which there is gradual destruction of the small bile ducts in the liver. As a result of the damage to the biliary ducts, bile builds up in the liver causing damage to the liver tissue. This may lead to scarring and liver failure, and may increase the risk of liver cancer. Ocaliva is used together with another medicine, ursodeoxycholic acid (UDCA), in patients who do not respond sufficiently to UDCA alone, and on its own in patients who cannot take UDCA.

Primary biliary cholangitis is rare, and Ocaliva was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 July 2010.

Ocaliva was granted a conditional marketing authorisation in December 2016. Conditional authorisation is granted on the basis of less comprehensive data than are normally required. It is granted for medicines that fulfil an unmet medical need to treat serious diseases and when the benefits of having them available earlier outweigh any risks associated with using the medicines while waiting for further evidence.

At the time of approval, the main study showed that Ocaliva reduced the blood levels of the substances bilirubin and ALP (markers of liver damage) in patients with primary biliary cholangitis, including those who could not be treated with UDCA. Reductions in bilirubin and ALP were considered to be indicators for future improvements in the condition of the liver. However, the benefits of Ocaliva needed to be confirmed in further studies.

The medicine was therefore granted a marketing authorisation on condition that the company provided further data on its benefits and safety from two additional studies (study 747-302 and study 747-401).

More information about the medicine can be found on the EMA website.