Genfit's double agonist was awarded orphan drug status by the FDA and EMA

PBC is a rare liver disease, mainly due to autoimmune damage to the bile tube (which functions to transport bile out of the liver), leading to bile siltation, inhibiting the liver's ability to remove toxins from the body, and leading to scarring of liver tissue, or cirrhosis of the liver.recently, Genfit announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have awarded its pilot drug PPAR alpha/δ dual astigid elafibranor for the treatment of primary bile tubeitis (PBC) orphan drug qualification.April 2019, the FDA also awarded Elafibranor Breakthrough Drug Qualification (BTD) for the treatment of adult PBC patients who did not respond well to UDCA.about elafibranor elafibranor is a pioneering (first-in-class) peroxidase proliferator activation subject α and δ (PPAR alpha/δ) double astristor, in the Phase II study to treat UDCA underreactive PBC adult patients have obtained positive safety and efficacy results., Elafibranor is also evaluating the potential for the treatment of non-alcoholic fatty hepatitis (NASH) in Phase III clinical studies.related studythis BTD award, based on data from a randomized, double-blind, placebo-controlled Phase II clinical study conducted in adult PBC patients who underreacted to UDCA.results showed that elafibranor significantly reduced alkaline phosphatase (ALP) levels, showed significant therapeutic effects in primary endpoints compared to placebos, and also reached compound endpoints for drug registration.study also showed improvements in other PBC markers in 2 groups of patients, including γ-glutamine transferase (GGT), lipid markers (total cholesterol, LDL and triglycerides) and anti-inflammatory markers (IgM, CRP, binding globin, and fibrinogen), in addition to significant reductions in ALP.safety , 2 doses of elafibranor therapy were well-to-do. Genfit plans to launch Phase III clinical studies in 2019 to further evaluate the efficacy and safety of elafibranor's treatment of PBC.
(Drug Information Network)