The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to elafibranor for the treatment of primary biliary cholangitis (PBC) in adults with inadequate response to ursodeoxycholic acid (UDCA).
Data from a phase 2 trial of elafibranor, a first-in-class double peroxisome proliferator-activated receptor alpha and delta (PPAR alpha/delta) agonist, showed that the treatment had a positive effect in non-cirrhotic patients with PBC and inadequate UDCA response (N=45).
At 12 weeks, elafibranor was associated with a significant decrease in alkaline phosphatase levels compared with placebo (primary endpoint). In addition, patients treated with elafibranor showed improvements in other PBC markers, including gamma-glutamyl transferase, lipid markers (total cholesterol, LDL and triglycerides), and anti-inflammatory markers (IgM, CRP, haptoglobin and fibrinogen).
“Approximately 50% of patients have an inadequate response to existing therapies, either because they do not respond to treatment or because they experience intolerable side effects like aggravated pruritus or hepatic toxicity,” said Pascal Birman, Deputy Chief Medical Officer of GENFIT. “Elafibranor has shown promising anticholestatic effects in a phase 2 clinical trial, while showing a trend in reducing pruritus.”
Elafibranor is also being evaluated in a phase 3 study in patients with nonalcoholic steatohepatitis (NASH). The primary outcome measures of this study include the proportion of elafibranor-treated patients who achieve resolution of NASH without worsening fibrosis, as well as a composite long-term outcome composed of all-cause mortality, cirrhosis, and liver-related clinical outcomes.
For more information visit genfit.com.