The U.S. Food and Drug Administration gave accelerated approval for seladelpar to treat primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who had an inadequate response to UDCA alone, Gilead Sciences announced recently. The company will market seladelpar under the name Livdelzi.
“People living with PBC have been waiting for treatment advancements for many years,” said Daniel O’Day, Gilead’s chairman and CEO, in a statement. “Today’s approval of Livdelzi, with its distinct profile, provides them with an important new option.”
Results of the RESPONSE trial
FDA approval came after recent results from a phase 3 clinical RESPONSE trial, which tested the effectiveness of seladelpar in reducing laboratory parameters of bile retention (cholestasis) alone or in combination with UDCA in patients with PBC.
Read more about PBC treatment and care
The patients in the study were divided into two groups: one receiving seladelpar and one receiving a placebo. The study lasted 12 months.
Results showed that more than 60% of patients receiving the drug achieved the study’s primary endpoint, compared with 20% of patients receiving a placebo. Furthermore, 25% of patients on the drug achieved a significant reduction of alkaline phosphatase (AP), a key predictor of fatal liver insufficiency.
The study also evaluated seladelpar’s capacity to reduce itching, a common PBC symptom. Results were positive, with a significant percentage of patients reporting substantial and sustained relief.
Further study
The safety and efficacy of seladelpar in patients with advanced PBC complicated with liver cirrhosis was not demonstrated, and the FDA approval did not extend to patients with cirrhosis. The AFFIRM study, currently in progress, will shed light on whether the drug is safe for patients with compensated liver cirrhosis.
Adverse effects and interactions
Although considered tolerable and safe for the majority of patients, research showed that seladelpar could cause adverse effects, including increased risk of fractures, headache, abdominal pain and nausea.
Seladelpar should not be combined with the antibiotic rifampin, as it can reduce the effectiveness of seladelpar. The concomitant use of bile acid sequestrants four hours prior to seladelpar could increase the risk of adverse effects.
