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Review of Ocaliva started EMA's human medicines committee (CHMP) has started a review of the medicine Ocaliva (obeticholic acid), used to treat adults with primary biliary cholangitis (PBC). PBC is an autoimmune condition that causes gradual destruction of the small bile ducts in the liver, which can lead to liver failure and increase the risk of liver cancer. The review was prompted by the final results from two studies in patients with PBC, which were requested by EMA in 2016 as part of the conditions to the initial marketing authorisation of Ocaliva. Study 747-302 was designed to confirm the benefits and safety of Ocaliva, while study 747-401 assessed the safety of Ocaliva in patients with advanced liver disease. In particular, study 747-302 failed to show that Ocaliva was more effective than placebo (a dummy treatment) in terms of the number of patients whose disease worsened or who died. In addition, side effects, including serious ones, occurred more frequently in patients treated with Ocaliva. EMA will now review these findings alongside all other available data and assess their impact on the overall benefit-risk balance of Ocaliva. The Agency will then make a recommendation on whether the medicine's marketing authorisation in the EU should be amended.
More about the medicine 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.
More about the procedure The review of Ocaliva has been initiated at the request of the European Commission, under Article 20 of Regulation (EC) No 726/2004. The review is being carried out by the Committee for Medicinal Products for Human Use (CHMP), responsible for questions concerning medicines for human use, which will adopt the Agency's opinion. The CHMP opinion will then be forwarded to the European Commission, which will issue a final legally binding decision applicable in all EU Member States.
Posted In: ICPT
