FDA Approves Ipsen, Genfit’s Elafibranor for Primary Biliary Cholangitis

The FDA has granted accelerated approval to Ipsen and Genfit’s new drug, elafibranor, which will be marketed under the brand name Iqirvo, for treating the rare liver disease primary biliary cholangitis (PBC). Christelle Huguet, head of R&D at Ipsen, emphasized that Iqirvo represents a vital new treatment option for patients suffering from PBC, marking the first new drug for this condition in nearly ten years. Huguet highlighted that current treatments often fail to adequately control the condition and can sometimes worsen symptoms, potentially leading to liver failure and the need for liver transplantation.

Primary biliary cholangitis is a rare autoimmune liver disease characterized by the accumulation of bile and toxins, accompanied by chronic inflammation that results in irreversible liver damage. Iqirvo, an oral, once-daily medication, was originally in-licensed from Genfit in 2021. It works by inhibiting the peroxisome proliferator-activated receptor, which may reduce the production of bile acids while alleviating inflammation and fibrosis.

Although the exact mechanism of Iqirvo in treating PBC is not fully understood, the drug is intended to be used alongside ursodeoxycholic acid (UDCA) in adult patients who have not responded adequately to UDCA alone. Additionally, Iqirvo can be used as a standalone treatment for patients who are intolerant to UDCA. The drug’s label advises against its use in patients with decompensated cirrhosis, although it does not carry a boxed warning.

Given that Iqirvo received approval through the FDA’s accelerated pathway based on a surrogate biomarker endpoint, its long-term benefits concerning survival or prevention of liver decompensation have not yet been proven. As a condition of its approval, Ipsen and Genfit may need to conduct a confirmatory trial to validate its clinical benefits.

The approval was supported by results from the Phase III ELATIVE trial. In November 2023, Ipsen and Genfit published data in The New England Journal of Medicine showcasing a 51% biochemical response rate, as measured by alkaline phosphatase levels, a common surrogate endpoint in PBC studies. In contrast, only 4% of the placebo group achieved the primary outcome. Elafibranor also met key secondary endpoints, with 15% of treated patients achieving normalized alkaline phosphatase levels compared to none in the placebo group.

The common side effects of elafibranor include abdominal pain, diarrhea, weight gain, nausea, and vomiting. Some patients reported more severe side effects such as myalgia, myopathy, rhabdomyolysis, fractures, and drug-induced liver injuries.

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