Advanz Pharma said it is “considering all options” after the European Commission (EC) revoked Ocaliva’s (obeticholic acid) conditional marketing authorisation.
The EC’s decision is based on a recommendation by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) made in June this year to remove market approval of the drug.
Only existing patients on the treatment regime may continue to use under compassionate access or via named patient programmes, as per a 3 September press release.
Ocaliva, which was conditionally approved in Europe in 2016, has been the only approved second-line treatment option for patients with primary biliary cholangitis (PBC), an autoimmune condition that causes gradual destruction of the bile ducts in the liver, which can lead to liver failure and increased cancer risk.
UK-based Advanz acquired the drug’s rights in Europe after acquiring non-American subsidiaries of Intercept Pharmaceuticals, which developed the drug, for an upfront payment of $405m in July 2022.
The CHMP recommendation followed a non-pharmacovigilance Article 20 procedure to reassess the benefit-risk profile of the Ocaliva’s use in PBC, with the committee stating in June that the benefits of Ocaliva no longer outweigh its risks. Article 20 procedures are used for already approved medicinal products where marketing authorisations need to be amended, suspended, or withdrawn.
The EC hasn’t been the only regulatory body to take a dim view of the drug’s benefit-to-risk profile. The US Food and Drug Administration (FDA) rejected obeticholic acid's use in nonalcoholic steatohepatitis (NASH) in June last year, following a negative Advisory Committee decision.
Ocaliva was originally shown to reduce the blood levels of alkaline phosphatase (ALP) and bilirubin in patients with PBC – indicating an improvement in liver condition. However, after the CHMP evaluated data from a confirmatory trial alongside real-world data, it stated the drug was no more effective than a placebo.
Ocaliva’s main side effects are intolerable itching and tiredness, according to the EMA.
Advanz said it disagreed with the EC’s decision and is considering all options to ensure continued access to the treatment. The company added that the CHMP “did not adequately consider the totality of available data”, especially real-world evidence, which backs efficacy and safety claims. Advanz admitted the confirmatory trial had several limitations such as patients on a placebo later choosing to switch to commercially available therapies.
Advanz Pharma CEO Steffen Wagner said: “The removal of Ocaliva could have a profoundly negative impact on the lives of the thousands of patients with PBC across Europe who have benefitted from this important treatment over many years.
“We will continue to work with the relevant authorities across the European Union to help ensure Ocaliva remains available for patients who require it.”
Note: The seventh paragraph of this story has been updated to reflect that the FDA rejected an application for obeticholic acid's use in nonalcoholic steatohepatitis (NASH) in June. An earlier version of the story omitted the indication in this instance.
