GENFIT announced the FDA has granted Breakthrough Therapy Designation to its lead product candidate elafibranor for the treatment of Primary Biliary Cholangitis (PBC) in adults with inadequate response to ursodeoxycholic acid (UDCA).
Breakthrough Therapy Designation is granted by the FDA to expedite the development and review of drugs designed to treat serious conditions for which preliminary data and evidence indicate that the product candidate may demonstrate substantial improvements over existing therapies on one or more clinically significant endpoints.
Earlier this month, 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). The 12-week, double-blind, randomized, placebo-controlled trial enrolled 45 non-cirrhotic patients with PBC and with inadequate response to UDCA. The patients were randomized to receive 12 weeks of add-on oral elafibranor at a dose of 80 mg/day or 120 mg/day or to placebo. The primary endpoint of the study was the percentage change from baseline in ALP at Week 12 relative to placebo. Both doses of elafibranor significantly decreased mean ALP compared with placebo (p<0.001), with reductions of 48% with the 80 mg/day dose, 41% with the 120 mg/day dose, and an increase of 3% with placebo. At Week 12, 67% and 79% of patients who received elafibranor 80 mg/day and 120 mg/day, respectively, had an ALP <1.67 x ULN, a reduction in ALP >15%, and a total bilirubin within normal limits compared with 6.7% of patients who received placebo. Significant improvements in lipid and inflammatory markers and a trend in decreased pruritis were also observed. Twelve weeks of elafibranor treatment was well tolerated.
A Phase 3 clinical trial expected to be initiated in 2019.
About Primary Biliary Cholangitis (PBC)
PBC is a serious chronic, progressive hepatic disease that leads to inflammation and scarring of the small bile ducts in the liver which, if left untreated, can lead to cirrhosis, liver failure and ultimately liver transplantation.
Elafibranor is a dual agonist of the PPARα and PPARδ. Targeting PPAR receptors has shown multiple beneficial activities, including the reduction of bile acid synthesis, improved detoxification of bile in the bile duct and anti-inflammatory activity. Elafibranor has also been shown to reduce serum alkaline phosphatase (ALP).