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The European Medicines Agency recommends the withdrawal of the marketing authorisation of Ocaliva for the treatment of primary biliary cholangitis - Statement from the European Reference Network for Rare and Complex Liver Diseases, ERN RARE-LIVER.
The European Medicines Agency (EMA) today recommended the recall of Ocaliva, a medicine provisionally authorised since 2016 for the treatment of adults with primary biliary cholangitis (PBC). The provisional authorisation was granted on the condition that the benefits of the medicine were confirmed in a long-term study. The data from this study has now been carefully analysed by the European Medicines Agency, and the assessment together with international experts and patient representatives, including representatives of the ERN RARE-LIVER, has led to a re-evaluation of the risks and benefits of the drug.
In particular, no improvement in life expectancy and avoidance of the need for liver transplantation could be demonstrated with Ocaliva compared to placebo. In addition to the lack of evidence of a survival benefit, the long-term study showed that itching, a major unpleasant symptom experienced by some people with PBC, worsened or reappeared with Ocaliva, similar to other studies of this drug.
Although Ocaliva will remain available until a final decision on withdrawal is made by the European Commission, a complete withdrawal from the pharmaceutical market can be expected very soon as a result of this recommendation. Prof Ansgar W. Lohse, Coordinator of the ERN RARE-LIVER and Clinical Director of the I. Medical Clinic at the University Medical Centre Hamburg-Eppendorf, comments: ´This recommendation by the EMA demonstrates the impressive diligence of the drug authorities, who also critically and constructively weigh up the benefits and safety of rare diseases, in close consultation with specialists and patient representatives. As disappointing as the decision is at first glance, it is encouraging to see that drug safety is in good hands.`
Based on these new findings, patients who have previously taken Ocaliva are asked to contact their treating physician to discuss their further treatment. In addition to the standard therapy with UDCA, which is a sufficiently effective therapy for the vast majority of people with PBC, treatment with fibrates, in particular Bezafibrate, is a very effective reserve therapy for the few patients who do not respond sufficiently well to UDCA alone. However, fibrates are not authorised for this indication, so the costs should be covered by health insurance in advance. Marketing authorisation for further drugs and thus new treatment options is expected in the coming months and years.
The expert centres of the European Reference Network were founded by the Euro-pean Commission in 2017 to provide better medical care for patients with rare diseases. For this reason, the ERN RARE-LIVER centres are also available as advisory reference centres for doctors and patients in this special situation.
https://rare-liver.eu/news/detail/ocaliva-withdrawal-ern-rare-liver-statement/
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