Intercept's liver drug approval delayed after negative adcomm

Following a nearly unanimous advisory committee decision against Alfasigma and Intercept Pharmaceuticals' rare liver disease medication Ocaliva, the FDA has postponed its verdict on whether to transition the drug from accelerated to full approval.

The initial Prescription Drug User Fee Act (PDUFA) date for the drug's supplemental New Drug Application (sNDA) was October 16. For the time being, the treatment remains available under accelerated approval for patients in the United States suffering from primary biliary cholangitis. In a statement, Intercept President Vivek Devaraj noted that the company intends to continue discussions with the FDA regarding the pending application.

Since its accelerated approval in 2016, the treatment has encountered several challenges, including the addition of a boxed warning in 2018. This warning was implemented after it was found that the drug was being incorrectly administered on a daily basis rather than the recommended weekly schedule. The drug was acquired by Alfasigma as part of its $800 million acquisition of Intercept Pharmaceuticals last year.

In September, an FDA advisory committee voted 13-1 against granting full approval for the FXR agonist, determining that the confirmatory trial data did not demonstrate a clinical benefit. The committee reviewed data from two studies: a traditional confirmatory study that was randomized and placebo-controlled, and a real-world evidence trial.

Additionally, in a separate vote, panelists concluded 10-1 that the benefits of Ocaliva did not outweigh its risks, with three members abstaining from the vote.

In Europe, the European Commission also revoked Ocaliva's conditional marketing authorization in September. This authorization had allowed the drug to be used as a second-line treatment for liver disease. The decision followed a June recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use, which reassessed the drug's benefit-risk profile.