FDA grants breakthrough status to Genfit’s Elafibranor

The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to Genfit’s Elafibranor to treat adults with primary biliary cholangitis (PBC) that experience an inadequate response to ursodeoxycholic acid (UDCA).

Elafibranor is a double peroxisome proliferator-activated receptor alpha and delta agonist that was originally developed to treat non-alcoholic steatohepatitis (NASH).

Results from a Phase II clinical trial showed that the therapeutic could also treat PBC, a rare, chronic live disease characterised by gradual damage to bile ducts. The condition hinders the liver’s ability to remove toxins from the body, which leads to cirrhosis.

Genfit deputy chief medical officer Pascal Birman said: "PBC is a severe liver disease that can lead to cirrhosis and liver failure, and is commonly associated with debilitating symptoms such as pruritus, which affect patients’ quality of life.

"Approximately 50% of patients have an inadequate response to existing therapies."

"Approximately 50% of patients have an inadequate response to existing therapies, either because they do not respond to treatment or because they experience intolerable side effects like aggravated pruritus (itching) or hepatic toxicity. Elafibranor has shown promising anticholestatic effects in a Phase II clinical trial, while showing a trend in reducing pruritus."

The FDA’s designation is based on results from a 12-week, double-blind, randomised placebo-controlled Phase II trial that treated non-cirrhotic subjects with PBC that showed an inadequate response to UDCA.

According to the results, Genfit’s drug candidate demonstrated a significant reduction of alkaline phosphatase (ALP) levels, compared to placebo. This was the primary endpoint of the study.

The trial also found improvements in other PBC markers such as gamma-glutamyl transferase (GGT), lipid markers, low-density lipoprotein (LDL) and anti-inflammatory markers in patients treated with Elafibranor.

Genfit noted that the GGT, lipid and anti-inflammatory marker improvements are consistent with those observed in the Phase II trial for NASH.

Elafibranor was generally well-tolerated in the trial, and is set to be studied in another Phase III trial.

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