CHMP Recommends Gilead's Seladelpar for Primary Biliary Cholangitis

Gilead Sciences has made significant progress in the treatment of primary biliary cholangitis (PBC) with their drug, seladelpar, which has received a positive recommendation from the European Medicines Agency's human medicines committee. The Committee for Medicinal Products for Human Use (CHMP) has advised that seladelpar be approved for use in conjunction with ursodeoxycholic acid (UDCA) for adults who do not respond adequately to UDCA alone. Moreover, it is recommended as a standalone treatment for individuals who cannot tolerate UDCA.

PBC is a rare autoimmune disorder affecting the bile ducts, predominantly impacting women, and it currently lacks a definitive cure. In Europe, it affects around 15 per 100,000 people. The disease is characterized by symptoms such as chronic itch, known as pruritus, and fatigue. Without treatment, PBC can progress, leading to liver damage and potentially liver failure.

Seladelpar works as a peroxisome proliferator-activated receptor-delta agonist, aiming to lower bile acid production in the liver. This mechanism helps prevent liver damage and decreases circulating bile acid levels. The CHMP's recommendation was influenced by the promising outcomes from the late-stage RESPONSE study. In this study, 62% of patients on oral seladelpar reached the primary endpoint of a composite biochemical response after a year, compared to just 20% of those given a placebo.

Additionally, seladelpar treatment resulted in the normalization of alkaline phosphatase levels—a cholestatic marker indicating liver transplant and mortality risk—in 25% of patients after one year. This improvement was not observed in any patients receiving a placebo. Moreover, patients treated with seladelpar experienced a statistically significant reduction in pruritus compared to those on placebo.

The European Commission is now tasked with considering the CHMP's recommendation as it deliberates on the approval of seladelpar for this specific use. Timothy Watkins, vice president of clinical development for inflammation therapeutics at Gilead, expressed optimism about this development. He noted that the positive opinion from CHMP brings them closer to offering seladelpar to individuals with PBC in Europe, particularly those who do not respond well to existing treatments or continue to suffer from symptoms like severe itchiness.

This recommendation from the CHMP follows the U.S. Food and Drug Administration's recent accelerated approval of seladelpar for treating PBC, where it is marketed under the brand name Livdelzi. As Gilead edges nearer to European approval, the hope is that seladelpar will provide much-needed relief to PBC patients, improving their quality of life and offering a new therapeutic option for managing this challenging condition.