MHRA Approves Gilead's Livdelzi for Adult Primary Biliary Cholangitis

Gilead Sciences has achieved a significant milestone with the approval of its innovative drug, Livdelzi (seladelpar), by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. This approval marks a pivotal advancement in the treatment of primary biliary cholangitis (PBC), a rare and chronic autoimmune disorder primarily affecting women and characterized by inflammation of the bile ducts. The MHRA has endorsed Livdelzi for use in conjunction with ursodeoxycholic acid (UDCA) for patients not responding adequately to UDCA alone, and also as a standalone therapy for those unable to tolerate UDCA.

Primary biliary cholangitis impacts around 25,000 individuals in the UK and currently lacks a definitive cure. The condition is often accompanied by debilitating symptoms such as pruritus, commonly known as chronic itch, and fatigue. If not addressed, PBC can progressively damage the liver, potentially leading to liver failure. Livdelzi represents a new class of treatment, known as peroxisome proliferator-activated receptor-delta agonists, which work by reducing bile acid production in the liver. This mechanism aims to mitigate liver damage and lower bile acid levels circulating in the body.

The development and approval of Livdelzi follow Gilead's acquisition of CymaBay Therapeutics in March for $4.3 billion, giving it access to this promising drug. The drug has already gained approval from the US Food and Drug Administration and received a recommendation from the European Medicines Agency’s human medicines committee for the treatment of PBC.

The positive outcomes that led to these regulatory approvals are based on data from the phase 3 RESPONSE study. In this study, Livdelzi demonstrated its efficacy, with 62% of patients achieving the primary endpoint of a composite biochemical response after one year, compared to just 20% of those on a placebo. The treatment also resulted in the normalization of alkaline phosphatase levels—a significant marker indicating the risk of liver transplant and mortality—in 25% of patients, a result not observed in the placebo group. Furthermore, patients treated with Livdelzi reported a statistically significant reduction in pruritus compared to those receiving a placebo.

Julian Beach, the interim executive director of healthcare quality and access at the MHRA, emphasized the rigorous regulatory standards met by Livdelzi during the approval process. He assured that the safety of the drug would continue to be closely monitored as it becomes available to patients.

The approval of Livdelzi is a timely addition to Gilead’s portfolio, coinciding with the company’s recent strategic alliance with LEO Pharma. Together, they are working on advancing LEO's small molecule oral STAT6 program for inflammatory diseases, a partnership valued at $1.7 billion. This collaboration underscores Gilead's commitment to expanding its influence and capabilities in treating inflammatory and immune-mediated diseases.

Overall, the introduction of Livdelzi offers renewed hope for patients suffering from primary biliary cholangitis, addressing a significant unmet medical need in this patient population and potentially improving the quality of life for thousands affected by this challenging condition.