Gilead Sciences’ seladelpar has been granted conditional marketing authorisation by the European Commission (EC) to treat patients with the rare liver disease primary biliary cholangitis (PBC).
The drug has been approved for use in combination with ursodeoxycholic acid (UDCA) to treat adults who have an inadequate response to UDCA alone, and as a monotherapy for those who are unable to tolerate UDCA.
Approximately 163,000 people in Europe are affected by PBC, an autoimmune disease of the bile ducts that disproportionally affects women.
The disease usually develops very slowly over many years and is characterised by pruritus, or chronic itch, and fatigue. It can also cause liver damage and possible liver failure if left untreated.
Seladelpar, an oral peroxisome proliferator-activated receptor-delta agonist, is designed to reduce the production of bile acid in the liver, preventing liver damage and reducing circulating bile acid levels.
The EC’s decision follows a recent recommendation from the European Medicines Agency’s human medicines committee and was supported by positive results from the phase 3 RESPONSE study, in which 62% of patients receiving daily seladelpar achieved the primary endpoint of composite biochemical response at one year, compared to 20% of those on placebo.
Seladelpar led to normalisation of alkaline phosphatase values, a cholestatic marker that is a predictor of risk for liver transplant and death, in 25% of patients at one year, while this change was not seen in any patients receiving placebo.
Patients treated with Gilead’s drug also demonstrated a statistically significant reduction in pruritus compared to placebo.
Timothy Watkins, vice president, clinical development of inflammation therapeutics at Gilead, said: “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.
“That changes… with the conditional approval of seladelpar, which has been shown to both help treat the disease and reduce pruritus.”
Watkins added that the company is “[looking] forward to working with health authorities across Europe to bring this promising new treatment to all those who could benefit”.
In line with the EC’s conditional approvals pathway, continued authorisation of seladelpar in these PBC indications will be contingent on the verification and description of clinical benefit.