September 10, 2024 | 12.51
READING TIME: 2 minutes
“If we do not have” obeticholic acid “we will go back 7 years, we will have a reset of scientific research and we will leave patients” with primary biliary cholangitis “without a drug that has become a lifesaver for them. Those already in therapy would be harmed, as well as those who could not enter treatment and benefit not only from the reduction of symptoms, but also from the progression of the disease and a gain in years of life”. Annarosa Floreani, senior scholar, University of Padua and scientific consultant Irccs Ospedale Sacro Cuore Don Calabria di Negrar (Verona), said this morning during the event – organized by Omar, Osservatorio Maladie Rare, in collaboration with Amaf Aps Ets, Associazione Maladie Autoimmuni del fegato, and Associazione EpaC Ets and the unconditional contribution of Advanz Pharma – on the subject of the revocation by the European Commission, following a decision by the EMA Agency, of the conditional marketing authorization (AIC) for obeticholic acid. The measure has been suspended, but the risk of no longer making the drug available to European patients remains.
“Primary biliary cholangitis – explains Floreani – is a chronic, rare, autoimmune disease of the liver that damages the small bile ducts that can lead to chronic inflammation, cirrhosis and liver transplant or death due to liver failure. It mainly affects women between 45 and 55 years of age, with a ratio of 9 to 1 compared to men. It is a complex pathology because it can be associated with other autoimmune diseases, including rheumatological ones such as hypothyroidism or arthritis. Its symptoms, mainly persistent itching and asthenia, impact the quality of life”.
In terms of therapies, “in 1987, the first line of treatment, ursodeoxycholic acid, was introduced on the market, which improved survival – explains the expert – but 30-40% of patients do not respond to the therapy and require a second line, which is obeticholic acid. This has been on the market since 2017 in Italy, where approximately 1,400 people have benefited from it. This is demonstrated by data from real-world studies, activated all over the world and involving over 700 patients in Italy”. Obeticholic acid “was introduced in 2017 in a conditional manner because it was a question of documenting the benefit on major events, such as cirrhosis – Floreani specifies – The Cobalt study recruited patients who had an advanced disease, but after obeticholic acid was released on the market, those in the placebo group left the trial to take the drug because they were benefiting from the therapy. Statistical biases were therefore created: it is therefore not possible to draw conclusions based on these highly flawed results”. But it is precisely starting from these data that “there was the revocation of the AIC, fortunately interrupted within 2 days. But the analysis that we expect to be considered” by the EMA “is that of the data from the real world studies which have a very high number and results are splendid and optimal”, he concludes.
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