Gilead Treatment for PBC Granted EC Conditional Marketing Authorization
Seladelpar, which is marketed in the US as Livdelzi, was recommended for an orphan drug designation by CHMP in December 2024.
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Gilead Sciences, headquartered in Foster City, Calif., announced on Feb. 20, 2025 that its seladelpar, an oral, peroxisome proliferator-activated receptor (PPAR)-delta agonist, has been granted conditional marketing authorization by the European Commission (EC) for treatment of the rare liver disease primary biliary cholangitis (PBC), either in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA alone, or as a monotherapy in patients unable to tolerate UDCA (1).
In December 2024, seladelpar was among five drugs recommended to be an orphan designated product on a larger list of 17 drugs green-lighted for market authorization by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) (1,2). It received approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) in January 2025 (1). The EC’s series of decisions concerning seladelpar was based on results of a placebo-controlled Phase III trial (RESPONSE), in which 62% of those who took seladelpar achieved the primary endpoint of composite biochemical response after 12 months, versus 20% of those taking placebo (1).
The effectiveness of treatments for PBC is commonly marked by an improvement in liver biochemical tests, particularly in the normalization of alkaline phosphatase (ALP) levels; ALP values normalized in 25% of trial participants taking seladelpar at month 12, while there was no normalization in those given placebo (1). Improvement of pruritus, or chronic itch, one of the most common symptoms of PBC, was also more pronounced after six months of treatment with seladelpar than with placebo, according to a Gilead press release (1).
“People living with PBC in Europe have been waiting for treatment advancements for many years. Up until now, there has been no approved treatment for PBC addressing both the surrogate biomarkers for underlying disease and pruritus, a common and at times debilitating symptom of PBC,” Timothy Watkins, MD, vice-president, Clinical Development of Inflammation Therapeutics for Gilead Sciences, said in the press release. “We look forward to working with health authorities across Europe to bring this promising new treatment to all those who could benefit.”
PBC is defined as a rare, chronic, and autoimmune disease of the bile ducts, for which there is no cure. In Europe, it affects approximately 22 out of every 100,000 people, according to Gilead (1). Women are more commonly affected than men, and the disease if left untreated can cause liver disease and ultimately liver failure.
As mentioned, the pruritus and fatigue which are often symptoms of PBC—related to cholestasis, or impaired bile flow—can be debilitating. In lieu of a cure, current treatment options include slowing disease progression and mitigating symptoms.
“Today’s decision reinforces the clinical benefit and value of seladelpar and offers people living with PBC in Europe an important new treatment option,” María-Carlota Londoño, MD, PhD, a hepatologist at Hospital Clinic Barcelona, said in the press release. “There are people in Europe who do not have an adequate response to first-line therapy, and seladelpar helps address the unmet need for effective and symptom-directed treatment.”
Continued authorization of seladelpar in the European Union, Gilead said, will be contingent on the verification and description of its clinical benefits in confirmatory trials (1).
Marketed as Livdelzi in the United States, seladelpar was granted accelerated approval by FDA in August 2024 (1,3). It is currently under regulatory review in Canada and Australia (1).
References
1. Gilead Sciences. Gilead’s Seladelpar Granted Conditional European Marketing Authorization for the Treatment of Primary Biliary Cholangitis. Press Release. Feb. 20, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 9–12 December 2024. Press Release. Dec. 13, 2024.
3. Gilead Sciences. Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by US FDA. Press Release. Aug. 14, 2024.
