Advanz Pharma on Tuesday said its liver disease treatment Ocaliva suffered a setback as the European Commission revoked its conditional marketing authorization.
Ocaliva, which was available in Europe as a second-line treatment for patients with primary biliary cholangitis (PBC), had its 2016 conditional authorization yanked following 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. Advanz said in its announcement that the decision wasn’t rooted in safety.
“We strongly disagree with the European Commission’s decision to withdraw the conditional marketing authorisation for Ocaliva, the only FXR agonist for patients with PBC, and until now the only approved and available second-line treatment option in Europe,” Advanz CEO Steffen Wagner wrote in a statement, adding that the company will continue to work with European regulators to keep the drug available.
Ocaliva is also facing scrutiny in the US. Alfasigma, which owns the US rights to the drug, is facing an FDA adcomm on Sept. 13. Just a month before receiving a CRL for Ocaliva in NASH, the FDA began evaluating the drug for a potential risk of liver disorder in PBC patients.
Alfasigma bought the drug as part of its $800 million buyout of Intercept Pharmaceuticals last year. The FDA handed down an accelerated approval for the drug in 2016, but it has since faced headwinds — Ocaliva saw a black box warning added in 2018 after it was being incorrectly dosed as a daily treatment, as opposed to a weekly treatment. The incorrect dosing possibly increased the risk for more severe liver damage, the agency said.
In its announcement, Advanz, which has the ex-US rights to Ocaliva, said that the committee’s recommendation to pull the authorization didn’t “adequately” take into account all of the efficacy and safety data for the drug. The company said that the committee relied on one analysis based on the COBALT trial, “which had multiple limitations, including the fact that patients in the placebo arm unsurprisingly chose to switch to commercially available therapy, but were required to be analyzed as placebo treated patients under the Intention to Treat (ITT) methodology.”
Ocaliva is now facing competition: Gilead secured accelerated approval for Livdelzi (seladelpar) in August, a PBC drug that was a part of the $4.3 billion CymaBay Therapeutics buyout. Livdelzi costs $12,606 for a 30-day supply, Ipsen’s primary biliary cholangitis treatment Iqirvo costs $11,500, and Ocaliva costs about $10,000.