EC Approves Ipsen's Iqirvo for Adult Primary Biliary Cholangitis Treatment

Ipsen’s drug Iqirvo (elafibranor) has received conditional approval from the European Commission (EC) for the treatment of primary biliary cholangitis (PBC). This decision allows the use of the oral peroxisome proliferator-activated receptor (PPAR) agonist in combination with ursodeoxycholic acid (UDCA) for adults who do not respond adequately to UDCA, or as a standalone therapy for those unable to tolerate UDCA.

PBC is a rare cholestatic liver disease that mainly affects women, occurring approximately nine times more frequently in women than in men. The disease leads to irreversible liver fibrosis, or scarring, and the destruction of bile ducts. Common symptoms include pruritus (itching) and fatigue, and the condition can progressively worsen if not effectively managed.

The EC's conditional approval follows a positive recommendation from the European Medicines Agency’s human medicines committee. This makes Iqirvo the first new treatment for PBC in nearly ten years. The approval is contingent on further verification of clinical benefits.

Supporting this approval were the promising results from the late-stage ELATIVE trial. In this study, 51% of patients treated with Iqirvo achieved a biochemical response, compared to just 4% in the placebo group.

Sandra Silvestri, Ipsen’s chief medical officer, highlighted the importance of this development, noting that PBC is a rare liver disease predominantly affecting women, and that there hasn't been significant innovation in treatment options for almost a decade. She expressed enthusiasm about offering an effective treatment choice for patients at risk of disease progression and those suffering from debilitating symptoms.

This conditional approval in the EU follows the accelerated approval granted by the US Food and Drug Administration just three months earlier for similar indications. Ipsen has also submitted applications for Iqirvo to other regulatory agencies, including the UK Medicines and Healthcare products Regulatory Agency.

Iqirvo was developed by Genfit, and Ipsen acquired the exclusive worldwide rights to the drug (excluding China, Hong Kong, Taiwan, and Macau) in 2021. At the time of this agreement, Ipsen’s chief executive officer, David Loew, expressed excitement about Iqirvo's potential benefits for the PBC community. He emphasized the innovative nature of the treatment and looked forward to global regulatory submissions to make this new treatment option available to patients worldwide.