Gilead's Seladelpar Receives Conditional European Approval for Primary Biliary Cholangitis Treatment

Gilead Sciences, Inc. has announced that the European Commission has given conditional marketing approval to seladelpar, a treatment designed for individuals with primary biliary cholangitis (PBC). This decision allows the use of seladelpar in conjunction with ursodeoxycholic acid (UDCA) for adults who do not respond adequately to UDCA alone or as a standalone treatment for those unable to tolerate UDCA. Seladelpar, recognized as an orphan drug, is set to become a significant therapy option for individuals suffering from this rare liver condition within the European Economic Area.

Primary biliary cholangitis is a chronic autoimmune disorder affecting the bile ducts, which impacts approximately 22 out of every 100,000 people in Europe. Predominantly diagnosed in women, this disease leads to liver damage that can escalate to liver failure if not properly managed. Common symptoms include pruritus (a persistent itch) and fatigue, which can severely affect the quality of life. Current treatment strategies aim to slow disease progression and alleviate symptoms of cholestasis, such as cholestatic itch. Liver biochemical test improvements, particularly the normalization of alkaline phosphatase (ALP) levels, are key measures of treatment efficacy in PBC.

The conditional approval of seladelpar marks a significant development for patients within Europe who have long-awaited new therapeutic solutions. Previously, no approved medications effectively addressed both the surrogate biomarkers of the underlying disease and pruritus, a frequent and often severe symptom of PBC. Seladelpar has demonstrated the capability to treat the condition and reduce pruritus.

The decision by the European Commission is based on positive feedback from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency. It draws from results of the pivotal Phase 3 RESPONSE trial, which was placebo-controlled. In this trial, 62% of participants treated with seladelpar achieved a composite biochemical response at the 12-month mark, compared to 20% in the placebo group. Additionally, 25% of those on seladelpar saw ALP normalization, a change not observed in the placebo group. Another significant outcome was the reduction in pruritus among patients, with those on seladelpar experiencing a 3.2-point improvement on a pruritus scale compared to a 1.7-point reduction for those given a placebo. Importantly, no serious adverse events related to the treatment were reported, though headaches, nausea, abdominal pain, and distension were observed in a small percentage of participants.

Gilead Sciences is actively collaborating with health authorities throughout Europe to ensure that eligible patients can access seladelpar promptly. The approval is conditional, dependent on further confirmation of clinical benefits through ongoing trials. Outside the European Economic Area, seladelpar has been granted accelerated approval by the U.S. FDA and also received approval from the UK's regulatory body. The drug is under regulatory review in both Canada and Australia.

Seladelpar is an oral therapy acting as a PPAR-delta agonist, influencing critical metabolic and liver disease pathways. It has shown potential in providing symptom relief and improving biochemical markers in PBC. Seladelpar has been recognized with various designations, including the FDA's Breakthrough Therapy and Orphan Drug Designations, and has been prioritized under the EU's PRIME initiative for medicines addressing unmet medical needs.

Gilead Sciences remains committed to addressing liver diseases, focusing on innovative treatments that not only manage symptoms but offer hope for improved patient outcomes in diseases like PBC.