Ipsen’s Iqirvo (elafibranor) has been granted accelerated approval from the US Food and Drug Administration (FDA) to treat adult patients with primary biliary cholangitis (PBC).
The oral, once-daily peroxisome proliferator-activated receptor agonist has been approved in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as a monotherapy in patients unable to tolerate UDCA.
Affecting approximately 100,000 people in the US, PBC is a rare, autoimmune, cholestatic liver disease that occurs when a buildup of bile and toxins, along with chronic inflammation, causes irreversible scarring of the liver and destruction of the bile ducts.
If left untreated, the lifelong condition can progress, leading to liver failure and potentially the need for a liver transplant.
The FDA’s decision was based on results from the phase 3 ELATIVE trial, published in the New England Journal of Medicine, which demonstrated that 13 times more patients achieved the composite primary endpoint of biochemical response when treated with Iqirvo plus UDCA compared to placebo plus UDCA.
Secondary endpoints demonstrated normalisation in alkaline phosphatase (ALP), a biochemical marker used as a surrogate endpoint in PBC trials, levels in 15% of Iqirvo-treated patients versus 0% for placebo plus UDCA.
Marked as “the first new medicine for PBC in nearly a decade”, Christelle Huguet, executive vice president, head of research and development, Ipsen, added: “Iqirvo demonstrated statistically significant improvements in biochemical response compared to UDCA alone [and] is therefore a much-needed treatment option.”
Dr Kris Kowdley, director, Liver Institute Northwest, Washington and primary investigator on the study, said: “The approval of Iqirvo will allow healthcare providers in the US to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC.”
Iqirvo was discovered and developed by Genfit and Ipsen licensed the exclusive worldwide rights (except in China, Hong Kong, Taiwan and Macau) to the drug in 2021.
Applications for Iqirvo have also been submitted to the European Medicines Agency and the Medicines and Healthcare products Regulatory Agency, with final regulatory decisions expected in the second half of 2024.
Most recently, Ipsen and Marengo Therapeutics expanded their ongoing oncology research partnership, which was first announced in 2022, to include Marengo’s T-cell engager platform, TriSTAR, in a deal worth up to $1.2bn to treat difficult-to-treat ‘cold’ tumours.