Elafibranor Receives FDA Priority Review for Rare Cholestatic Liver Disease PBC

▎WuXi

Edited by Kant Content Team

Recently, Ipsen and GENFIT jointly announced that the U.S. FDA has accepted the New Drug Application (NDA) for their investigational small molecule therapy elafibranor. The press release noted,Elafibranor is expected to become the first new second-line treatment for rare cholestatic liver disease primary biliary cholangitis (PBC) in nearly a decade.The application has been granted Priority Review status, with a PDUFA target date of June 10, 2024.

The European Medicines Agency (EMA) has also accepted the Marketing Authorization Application (MAA) for elafibranor, and on October 26, 2023, began reviewing the application submitted to the EMA's Committee for Medicinal Products for Human Use (CHMP). In addition, the UK Medicines and Healthcare products Regulatory Agency (MHRA) has accepted the third concurrent regulatory submission for elafibranor.

Elafibranor's Pivotal Phase 3TestELATIVE

The positive results for

Elafibranor's global marketing application as a potential therapy for PBC was supported by data previously presented at the American Association for the Study of Liver Diseases (AASLD) annual meeting and published in The New England Journal of Medicine. The trial evaluated the efficacy and safety of elafibranor in treating patients with PBC.

The analysis shows,Patients showed statistically significant improvements in biomarkers of disease progression at each key endpoint, with the primary composite endpoint achieving significant therapeutic benefit.The difference in biochemical response rates between the 80 mg elafibranor group (51%) and the placebo group (4%) was 47% (P<0.001). In terms of safety, elafibranor was well-tolerated, with a safety profile consistent with previous trials.

Image Source: 123RF

Elafibranor is a once-daily, oral, potential "first-in-class" dual peroxisome proliferator-activated receptor (PPAR) α/δ agonist currently under investigation for the treatment ofRare AutoimmunePBC patients.Simultaneously targeting the activation of PPAR α/δ can potentially treat inflammation, cholestasis, and fibrosis in PBC. In 2019, the U.S. FDA granted elafibranor Breakthrough Therapy designation for the treatment of adult PBC patients with an inadequate response to ursodeoxycholic acid (UDCA).

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