Gilead Sciences’ seladelpar has been recommended by the European Medicines Agency’s human medicines committee to treat the rare liver disease primary biliary cholangitis (PBC).
The Committee for Medicinal Products for Human Use (CHMP) has recommended that the drug be approved for use alongside ursodeoxycholic acid (UDCA) to treat adults who have an inadequate response to UDCA alone, and also as monotherapy for those who are unable to tolerate UDCA.
Affecting approximately 15 per 100,000 people in Europe, PBC is a rare autoimmune disease of the bile ducts that mainly affects women and currently has no cure.
Pruritus, or chronic itch, and fatigue are among the most common symptoms of PBC, and the disease can cause liver damage and possible liver failure if left untreated.
Gilead’s seladelpar is a peroxisome proliferator-activated receptor-delta agonist designed to reduce the production of bile acid in the liver, preventing liver damage and reducing circulating bile acid levels.
The CHMP’s recommendation of the drug was based on positive results from the late-stage RESPONSE study, in which 62% of patients receiving oral seladelpar achieved the primary endpoint of composite biochemical response at one year, versus 20% of those on placebo.
Treatment with 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, and seladelpar-treated patients also demonstrated a statistically significant reduction in pruritus compared with placebo.
The European Commission will now consider the CHMP’s recommendation as it makes its decision on seladelpar in this indication.
Timothy Watkins, vice president, clinical development of inflammation therapeutics, Gilead, said: “We are encouraged by the CHMP’s positive opinion as we are one step closer to providing seladelpar to people living with PBC in Europe. There are still people living with PBC who do not have an adequate response to current medicines or who are still experiencing symptoms, such as debilitating itch.”
The recommendation comes four months after the US Food and Drug Administration granted accelerated approval to seladelpar to treat PBC under the brand name Livdelzi.