Ursodeoxycholic acid

The FDA issued a Complete Response Letter to Intercept Pharmaceuticals’ obeticholic acid (Ocaliva) for primary biliary cholangitis (PBC), unable to approve its supplemental New Drug Application in its current form. The decision aligns with a negative opinion from a Gastrointestinal Drugs Advisory Committee meeting in September 2024, which found insufficient evidence to verify obeticholic acid’s benefits on clinical outcomes in PBC and its favorable benefit-risk profile. Despite the CRL, obeticholic acid remains available for PBC treatment under accelerated approval status in the US.

FDA extends review of Intercept Pharmaceuticals' sNDA for full approval of obeticholic acid (Ocaliva) for primary biliary cholangitis (PBC), with no new action date provided. Despite a negative advisory committee opinion on its clinical outcomes and benefit-risk profile, Ocaliva remains available under accelerated approval.

Dr. Steven-Huy Han prescribes Ocaliva for primary biliary cholangitis, unsure of its long-term benefits. The FDA's accelerated approval process, often based on surrogate markers, has led to debate over drug effectiveness. Ocaliva's approval was supported by patient testimony, despite limited long-term data. The FDA requires additional trials post-approval, but some companies struggle to meet these requirements. The accelerated approval process, driven by impatience for new treatments, has led to the approval of drugs with uncertain long-term benefits.

The PBC drug market sees two new FDA-approved therapies, Iqirvo and Livdelzi, alongside an FDA advisory committee's unfavorable vote on Ocaliva's long-term safety and efficacy.

Iqirvo (elafibranor) has been conditionally approved in the EU for treating adults with primary biliary cholangitis (PBC), either with UDCA for non-responders or alone for those intolerant to UDCA. The approval is based on ELATIVE trial data showing Iqirvo reduced liver damage markers more than a placebo. Iqirvo activates PPAR alpha and delta receptors to control PBC-driving processes, offering a new treatment option for PBC patients.

European Commission grants conditional marketing authorization for Iqirvo® (elafibranor), a first-in-class treatment for primary biliary cholangitis (PBC), following positive CHMP opinion based on ELATIVE phase III trial data. Iqirvo demonstrated significant efficacy over placebo and was well-tolerated, reinforcing Ipsen’s commitment to advancing medical innovations for rare cholestatic liver diseases.

FDA grants Gilead Sciences' Livdelzi (seladelpar) accelerated approval for treating PBC in adults intolerant or unresponsive to UDCA. Livdelzi, a PPAR delta agonist, showed significant improvement in liver disease markers and reduced pruritus in the RESPONSE study, with potential for continued approval pending further trials.

FDA's GIDAC discussed Intercept's sNDA for OCALIVA for PBC treatment; Intercept disappointed by the committee's vote, emphasizing OCALIVA's clinical benefits. FDA to make final decision by October 15, 2024.

FDA advisors voted 10-1 against full approval of obeticholic acid (Ocaliva) for primary biliary cholangitis (PBC), citing insufficient data and safety concerns. The main trial, COBALT, showed trends of excess liver transplants and death. The drug had previously received accelerated approval in 2016, but now risks withdrawal from the market.