FDA, EMA Grant GENFIT’s Elafibranor Orphan Drug Status

GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to the discovery and development of innovative therapeutic and diagnostic solutions in metabolic and liver related diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted Orphan Drug Designation to elafibranor, a PPAR alpha/delta agonist, for the treatment of PBC.

PBC is a cholestatic liver disease, wherein bile ducts become damaged leading to scarring of liver tissue or cirrhosis. The causes are still unknown. This disease mainly affects women, and many patients cannot benefit from existing therapies, representing a significant unmet medical need.

Elafibranor, in a Phase 2 placebo-controlled trial in PBC patients with inadequate response to UDCA, clearly showed statistical significance on achieving the primary endpoint of reducing ALP versus placebo. Beneficial effects in patients also included improvements in cholestatic markers (GGT, 5’), lipid markers (total cholesterol, LDL, and triglycerides) and anti-inflammatory markers (IgM, CRP, haptoglobin and fibrinogen). These improvements are consistent with the results from the Phase 2b clinical trial evaluating elafibranor in NASH, and essential when treating a cholestatic disease such as PBC, or when treating NASH which is considered as the liver manifestation of the metabolic syndrome.

https://www.marketscreener.com/GENFIT-16311755/news/GENFIT-FDA-and-EMA-Grant-GENFIT-s-Elafibranor-Orphan-Drug-Designation-for-Primary-Biliary-Cholangit-28973734/