Ipsen’s Iqirvo (elafibranor) has been conditionally approved by the European Commission (EC) to treat primary biliary cholangitis (PBC).
The oral peroxisome proliferator-activated receptor (PPAR) agonist has been authorised for use in combination ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as a monotherapy in patients who are unable to tolerate UDCA.
Affecting approximately nine women for every one man, PBC is a rare cholestatic liver disease that causes irreversible fibrosis (scarring) of the liver and destruction of the bile ducts.
The most common symptoms of PBC are pruritus, or itching, and fatigue, and the condition can worsen over time if not effectively treated.
The EC approval, which is contingent on the further verification of clinical benefit, follows a recent recommendation from the European Medicines Agency’s human medicines committee and makes Iqirvo the first new treatment for PBC in almost a decade.
The decision was supported by positive results from the late-stage ELATIVE trial, in which 51% of patients receiving Iqirvo achieved a biochemical response, compared to 4% of those in the placebo group.
Ipsen’s chief medical officer, Sandra Silvestri, said: “[PBC] is a rare liver disease, predominantly found in women, which hasn’t seen new innovation for nearly a decade… For those patients with PBC that may be at risk of disease progression and who continue to suffer from debilitating symptoms of the disease, we are delighted to be able to offer an effective treatment choice.”
The authorisation comes just three months after Iqirvo was granted accelerated approval by the US Food and Drug Administration for the same indications. Applications for the drug have also been submitted to other regulators, including the UK Medicines and Healthcare products Regulatory Agency.
Iqirvo was developed by Genfit and Ipsen licensed the exclusive worldwide rights (except in China, Hong Kong, Taiwan and Macau) to the drug in 2021.
David Loew, Ipsen’s chief executive officer, said at the time of the agreement: “We are excited by [Iqirvo’s] data package, demonstrating the potential benefit of this first-in-class, innovative treatment option to help the PBC community. We look forward to… regulatory submissions around the world to bring this potential new treatment option to patients.”