Ipsen, Genfit’s elafibranor wins FDA approval for primary biliary cholangitis

FDA

Ipsen, Genfit’s elafibranor wins FDA approval for primary biliary cholangitis

Published: Jun 11, 2024

By Tristan Manalac

BioSpace

The FDA on Monday granted accelerated approval to Ipsen and Genfit’s elafibranor, now to carry the brand name Iqirvo, for the treatment of the rare liver disease primary biliary cholangitis.

Christelle Huguet, head of R&D at Ipsen, said in a statement that Iqirvo is a “much-needed treatment option” for patients with primary biliary cholangitis (PBC) and is the first new drug for this condition in nearly a decade.

“For a significant number of people living with PBC, available treatments do not control the condition and may exacerbate symptoms of PBC,” Huguet said. When left unchecked, patients with PBC can develop liver failure and may ultimately need liver transplantation.

PBC is a rare, autoimmune and cholestatic liver disease characterized by the build-up of bile and toxins as well as chronic inflammation, both of which lead to irreversible liver scarring. Iqirvo, a first-in-class oral, once-daily oral drug in-licensed from Genfit in 2021, works by blocking the peroxisome proliferator-activated receptor, which could lower the production of bile acids and ease inflammation and fibrosis.

Iqirvo’s exact mechanism of action for PBC is not completely known, according to its label. Iqirvo is indicated as a combination treatment with ursodeoxycholic acid (UDCA) in adult patients who have shown an inadequate response to UDCA. The drug can also be given as a monotherapy in patients who are intolerant to UDCA. Iqirvo does not come with a boxed warning but its label recommends against its use in patients with decompensated cirrhosis.

Because Iqirvo was approved under the FDA’s accelerated pathway, using a surrogate biomarker endpoint, its benefits in terms of survival or prevention of liver decompensation “have not been demonstrated,” according to the companies. To keep Iqirvo’s approval, Ipsen and Genfit may need to run a confirmatory trial to verify its clinical benefit.

Monday’s approval was supported by data from the Phase III ELATIVE trial. In November 2023, Ipsen and Genfit published data from the study in The New England Journal of Medicine, touting a 51% biochemical response rate as assessed through alkaline phosphatase levels, a biochemical marker often used as a surrogate endpoint in PBC studies. By comparison, only 4% of placebo counterparts hit the primary outcome.

Elafibranor also met its key secondary endpoints, normalizing alkaline phosphatase levels in 15% of treated patients, compared to none in the placebo arm.

The most common side effects associated with elafibranor included abdominal pain, diarrhea, weight gain, nausea and vomiting. Some patients developed myalgia, myopathy, rhabdomylosis, fractures and drug-induced liver injuries.

Source: BioSpace