FDA Sets Review Date for Elafibranor for Rare Liver Disease

The FDA has accepted Ipsen’s new drug application (NDA) for elafibranor and granted the application priority review. If approved, it would be indicated as a second-line treatment for patients with the rare liver disease, primary biliary cholangitis (PBC). The Prescription Drug User Fee Act action date is June 10, 2024.

PBC is a progressive autoimmune disease that results from the destruction of the bile ducts in the liver. This leads to inflammation, scarring and cirrhosis. The disease affects mostly women

This is a condition where many patients are living with worsening disease and debilitating symptoms despite being on treatment,” Christelle Huguet, Ph.D., EVP and read of research and development at Ipsen, said in a press release.

Elafibranor, which is being developed by Ipsen and Genfit, is an oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist. It targets multiple cell types and biological processes involved in the pathophysiology of PBC, including impairment of bile flow in the liver, bile toxicity, inflammation and fibrosis and bile acid output.

The application was based on data from the phase 3 ELATIVE clinical trial, and results were recently publishedin the New England Journal of Medicine. The trial evaluated the efficacy and safety of elafibranor 80 mg once daily versus placebo for the treatment of patients with PBC with an inadequate response or intolerance to ursodeoxycholic acid, which is used to treat PBC. The trial enrolled 161 patients.

Data confirmed the potential for elafibranor to be an effective new treatment option compared with placebo with more patients achieving a biochemical response. Reductions in levels of alkaline phosphatase (ALP) were rapid, seen as early as week 4 in the elafibranor group, and were sustained through Week 52, with a decrease in ALP of 41% on elafibranor compared with placebo. ALP and bilirubin are important predictors of PBC disease progression.

ELATIVE also assessed as a secondary endpoint the effect of elafibranor on severe itch, a symptom of PBC. Using the PBC Worst Itch NRS score, the trial showed a reduction in itch in patients treated elafibranor, but it was not statistically significant. Data reported from two separate patient-reported outcome measures demonstrated reductions in moderate to severe pruritus, which favored elafibranor versus placebo.

Elafibranor was well-tolerated in the trial and has a well-documented safety profile. Adverse events included abdominal pain, diarrhea, nausea, and vomiting.

This story first appeared on Managed Healthcare Executive.