CHMP positive for seladelpar for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid.- Gilead Sciences

Gilead Sciences, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending seladelpar for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA. The final European Commission decision is anticipated in the first quarter of 2025. This follows the accelerated approval by the  FDA in August 2024.

PBC is a rare, chronic, autoimmune disease of the bile ducts that affects approximately 15 per 100,000 people in Europe, primarily women, and can cause liver damage and possible liver failure if untreated. The most common symptoms of PBC are pruritus (chronic itch) and fatigue, which can be debilitating for some people. The disease currently has no cure and treatment goals for people living with PBC include suppressing liver damage and reducing the symptoms related to cholestasis. The effect of treatment on slowing disease progression is primarily measured by an improvement in liver biochemical tests, including the normalization of alkaline phosphatase (ALP) levels, an important marker of disease progression in PBC.

“This positive opinion from the Committee confirms promising clinical benefit and value of seladelpar, which has been underscored by its differentiated body of data,” said Palak Trivedi, MD, BSc (Hons), MBBS, MRCP (UK), ESEGH, PhD, Associate Professor and Consultant Hepatologist at the Queen Elizabeth Hospital in Birmingham. “After many years of treating people with PBC, I have seen the critical unmet need for additional effective and symptom-directed treatment options. This recommendation of a potential new therapy that can help treat both the disease and improve symptoms that impact quality of life is a significant milestone for the PBC community.”

The positive opinion was supported primarily by data from the pivotal placebo-controlled Phase III RESPONSE study. In the study, 62% of participants taking seladelpar achieved the primary endpoint of composite biochemical response at month 12 compared with 20% of participants taking placebo. Treatment with seladelpar led to normalization of ALP values in 25% of trial participants at month 12. This change was not seen in any trial participants receiving placebo. ALP is a cholestatic marker that is a predictor of risk for liver transplant and death. Change from baseline pruritus score at month 6 was a key secondary endpoint; treatment with seladelpar led to a statistically significant reduction in pruritus compared with placebo. Participants entering the study with moderate to severe itch experienced a 3.2-point improvement on a pruritus scale of 0-10 after six months of treatment with seladelpar, compared to a decrease of 1.7 points with placebo.