European Commission withdraws Ocaliva authorization before FDA advisory

Advanz Pharma recently encountered a significant challenge as the European Commission rescinded the conditional marketing authorization for its liver disease treatment, Ocaliva. This decision impacts Ocaliva's availability in Europe, where it was used as a second-line treatment for patients suffering from primary biliary cholangitis (PBC). The original conditional authorization for Ocaliva was granted in 2016 but came under review by the European Medicines Agency’s Committee for Medicinal Products for Human Use. The committee reassessed the drug's benefit-risk profile, leading to the recent revocation. Advanz Pharma emphasized that the decision was not based on safety concerns.

CEO Steffen Wagner expressed strong disagreement with the European Commission's decision. He highlighted that Ocaliva is the only FXR agonist approved for PBC patients in Europe and the sole second-line treatment option until its authorization was withdrawn. Wagner affirmed that the company plans to continue collaborating with European regulators to try to maintain the drug's availability for PBC patients.

In the United States, Ocaliva is also under scrutiny. Alfasigma, which holds the U.S. rights to the drug, faces an FDA advisory committee meeting on September 13. This comes after the FDA began assessing the drug for a potential risk of liver disorder in PBC patients, just a month before issuing a Complete Response Letter (CRL) for Ocaliva in nonalcoholic steatohepatitis (NASH). Alfasigma acquired Ocaliva as part of an $800 million purchase of Intercept Pharmaceuticals last year. Although the FDA granted accelerated approval for Ocaliva in 2016, the drug has faced several challenges, including a black box warning added in 2018. This warning was due to incorrect daily dosing, which increased the risk of severe liver damage.

Advanz Pharma, which holds the rights to Ocaliva outside the U.S., criticized the committee's recommendation to revoke the drug's authorization. The company argued that the committee did not sufficiently consider all the efficacy and safety data. They pointed out that the COBALT trial, which influenced the decision, had several limitations. One key issue was that patients in the placebo group opted to switch to commercially available therapies but were still analyzed as placebo-treated patients under the Intention to Treat (ITT) methodology.

Ocaliva now faces heightened competition in the market. Gilead recently received accelerated approval for Livdelzi (seladelpar) in August, a new PBC drug acquired through its $4.3 billion purchase of CymaBay Therapeutics. Livdelzi is priced at $12,606 for a 30-day supply. Another competitor, Ipsen, offers Iqirvo for PBC treatment at a price of $11,500. In contrast, Ocaliva is priced at approximately $10,000. 

Advanz Pharma's ongoing efforts to keep Ocaliva available underscore the complexities and challenges in navigating regulatory landscapes while attempting to provide effective treatment options for patients with rare liver diseases like PBC. The outcome of the FDA's review and the company's negotiations with European regulators will be crucial in determining the future availability of Ocaliva for patients who rely on this treatment.

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