EMA Recommends Seladelpar for Primary Biliary Cholangitis

The European Medicines Agency (EMA) has recommended granting marketing authorization for Seladelpar Gilead (seladelpar lysine dihydrate) to treat primary biliary cholangitis (PBC).

PBC is a chronic autoimmune liver disease marked by inflammation and progressive destruction of the small bile ducts within the liver. This leads to the accumulation of toxic bile acids, potentially progressing to cirrhosis and liver failure. The condition primarily affects women and is associated with symptoms such as severe fatigue and pruritus.

Seladelpar Gilead is an oral peroxisome proliferator–activated receptor (PPAR) delta agonist. Its active substance, seladelpar lysine dihydrate, helps normalize alkaline phosphatase (ALP) and bilirubin levels in adults with PBC. 

Clinical Trial Results

A phase 3 trial involving 193 patients, published in February 2024 in The New England Journal of Medicine, demonstrated that patients receiving oral seladelpar had significantly higher ALP normalization rates compared with those who received placebo. The drug also significantly reduced pruritus among patients with moderate to severe pruritus at baseline.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) determined that expected clinical benefits of seladelpar include delayed development of liver fibrosis and cirrhosis, potentially reducing the need for liver transplant and lowering mortality risks.

Common side effects of the drug are abdominal pain, headache, nausea, and abdominal distension.

Indication and Use

The full indication for seladelpar, which will be available as 10-mg hard capsules, is for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA.

The drug was supported via the EMA’s priority medicines scheme (PRIME), which provides early and enhanced scientific and regulatory support for medicines with a potential to address unmet medical needs.

Following authorization by the European Commission, the company will be expected to submit further clinical data on the drug’s efficacy and safety. 

Peter Russell has been a journalist for 40 years covering international news, health, medicine, and national politics on radio, TV, and online. He is based in the UK.