Breakthrough therapy designation to elafibranor

June 11, 2019 0 By FM

Genfit announced that its lead product candidate elafibranor was granted Breakthrough Therapy Designation by the US FDA for the treatment of primary biliary cholangitis (PBC) in adults with inadequate response to ursodeoxycholic acid (UDCA).

Elafibranor is a first-in-class double peroxisome proliferator-activated receptor alpha and delta (PPAR alpha/delta) agonist which has produced positive results in a phase 2 clinical trial evaluating its safety and efficacy in adults with PBC and inadequate response to UDCA.

Elafibranor is also currently evaluated in a phase 3 clinical trial in non-alcoholic steatohepatitis (NASH).

Genfit presented detailed results from its positive phase 2 clinical trial evaluating elafibranor in PBC during the European Association for the Study of the Liver (EASL) annual International Liver Congress (ILC).

In a 12-week double-blind randomized placebo-controlled phase 2 trial of non-cirrhotic patients with PBC and with inadequate response to UDCA, elafibranor showed a significant decrease of alkaline phosphatase (ALP) levels, resulting in significant treatment effects versus placebo on the primary endpoint.