Seladelpar receives conditional EC approval for rare liver disease PBC

Gilead Sciences' drug seladelpar has received conditional marketing authorization from the European Commission for the treatment of primary biliary cholangitis (PBC), a rare liver disease. This approval marks a significant advancement in treating this condition, particularly for patients who have not responded adequately to ursodeoxycholic acid (UDCA) or cannot tolerate it.

PBC is an autoimmune disorder that affects the bile ducts, primarily impacting women. It progresses slowly, often over many years, and is associated with symptoms such as persistent itching, known as pruritus, and fatigue. If left untreated, it can result in liver damage, potentially leading to liver failure.

Seladelpar functions as an oral peroxisome proliferator-activated receptor-delta agonist, aiming to lower bile acid production in the liver. This mechanism helps prevent liver damage and decreases the levels of circulating bile acid. The European Commission's decision to conditionally approve seladelpar was preceded by a favorable recommendation from the European Medicines Agency's human medicines committee. This endorsement was bolstered by data from the phase 3 RESPONSE study, which revealed that 62% of the patients taking seladelpar daily achieved the primary endpoint of composite biochemical response after a year, compared to just 20% in the placebo group.

The study further demonstrated that seladelpar normalized alkaline phosphatase levels, a crucial cholestatic marker indicative of liver transplant risk and mortality, in 25% of patients after one year. In contrast, no such normalization was observed in patients on the placebo. Additionally, patients on seladelpar experienced a statistically significant reduction in pruritus when compared to those receiving the placebo.

Timothy Watkins, vice president of clinical development of inflammation therapeutics at Gilead, emphasized the importance of this approval, noting the long-standing need for improved therapies for PBC in Europe. He highlighted that until now, there was no approved treatment addressing both the biochemical markers of the disease and the pruritus that often accompanies it. With the conditional approval of seladelpar, there is now a treatment option that addresses both these aspects, potentially improving the quality of life for those affected by PBC.

Watkins also expressed the company's commitment to collaborating with health authorities across Europe to make this innovative treatment accessible to patients who could benefit from it. The continuation of seladelpar's authorization for treating PBC hinges on further verification and description of its clinical benefits, in accordance with the European Commission’s conditional approvals process.

Ultimately, the approval of seladelpar represents a promising new development for individuals suffering from primary biliary cholangitis, offering hope for better management of this challenging condition and its symptoms.