Following a nearly unanimous adcomm against Alfasigma and Intercept Pharmaceuticals’ rare liver disease drug Ocaliva, the FDA has delayed its decision on whether to convert the drug from an accelerated to a full approval.
The original PDUFA date for the drug’s sNDA was Oct. 16. For now, the treatment remains available via the accelerated approval for patients with primary biliary cholangitis in the US, Intercept president Vivek Devaraj wrote in a statement, adding that the company “will continue to engage with the FDA regarding our pending application.”
The treatment has faced multiple setbacks since its accelerated approval in 2016, including the addition of a boxed warning in 2018 after it was being incorrectly dosed as a daily treatment, as opposed to a weekly treatment. Alfasigma bought the drug as part of its $800 million buyout of Intercept Pharmaceuticals last year.
The FDA adcomm in September recommended against full approval for the FXR agonist by a 13-1 vote, concluding that confirmatory trial data didn’t verify that the treatment provided a clinical benefit. The adcomm discussed data from two studies: a traditional confirmatory study designed to be randomized and placebo-controlled, and a real-world evidence trial.
In a separate vote, panelists decided 10-1 that the benefits of Ocaliva did not outweigh its risks, with three abstentions.
The European Commission also revoked Ocaliva’s conditional marketing authorization in September, which was available in Europe as a second-line treatment in the liver disease. The decision came after a June recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use, which reassessed the benefit-risk profile of the drug.