Genfit’s Elafibranor earns Orphan Drug Designations for Primary Biliary Cirrhosis
Genfit’s drug Elafibranor has gained the FDA and EMA’s orphan drug designation for treating primary biliary cirrhosis (PBC).
In the drugmaker’s Phase 2 placebo-controlled trial of PBC patients that inadequately responded to ursodeoxycholic acid (UDCA), researchers saw a “[clear] statistical significance on achieving the primary endpoint of reducing ALP versus placebo,” the company said.
PBC is a rare liver disease that leads to scarred liver tissue or cirrhosis through the gradual destruction of a patient’s bile ducts. The disease affects mostly women.