FDA Grants Accelerated Approval for Ipsen’s Iqirvo for Primary Biliary Cholangitis

Ipsen has obtained accelerated approval from the US Food and Drug Administration (FDA) for its drug Iqirvo (elafibranor) in 80mg tablet form. Iqirvo is targeted at treating primary biliary cholangitis (PBC), a chronic liver disease. The treatment is recommended for use in combination with ursodeoxycholic acid (UDCA) for adult patients who do not respond adequately to UDCA, or as a standalone treatment for those who are intolerant to UDCA.

Ipsen will manage the marketing and commercialisation of Iqirvo under its trademark name and will make it available immediately for prescription in the US to eligible patients. The FDA’s accelerated approval for this drug is based on its ability to reduce alkaline phosphatase (ALP) levels. However, the approval does not confirm improvements in survival rates or prevention of liver decompensation events.

The Phase III ELATIVE clinical trial demonstrated that patients treated with Iqirvo in combination with UDCA were 13 times more likely to reach the composite primary endpoint of biochemical response compared to those treated with a placebo plus UDCA. Secondary endpoints were met, showing ALP level normalization exclusively in patients treated with Iqirvo. Continued approval for this indication will rely on the results and clinical benefits observed in ongoing confirmatory trials.

Iqirvo is not recommended for patients with decompensated cirrhosis, which includes conditions such as ascites, variceal bleeding, or hepatic encephalopathy. The drug was initially discovered and developed by GENFIT, with Ipsen acquiring the global licensing rights for elafibranor in 2021, excluding China, Hong Kong, Macau, and Taiwan. Ipsen has committed to milestone payments amounting to €89 million ($95.7 million) to GENFIT, which includes €48.7 million upon the first commercial sale of Iqirvo in the US and an additional €13.3 million milestone payment due in February 2024.

Christelle Huguet, Head of Research and Development and Executive Vice-President at Ipsen, commented on the approval, emphasizing the significant need for new treatment options for PBC. According to Huguet, many patients suffering from PBC do not find existing treatments effective in controlling their condition, which can lead to severe consequences such as liver failure and, in some cases, the need for a liver transplant. Iqirvo offers statistically significant improvements in biochemical response compared to UDCA alone and is the first new treatment for PBC in nearly ten years.

This approval for Iqirvo follows Ipsen's recent expansion of its ongoing oncology research collaboration with Marengo Therapeutics. The partnership aims to advance T cell engagers using Marengo’s TriSTAR antibody fusion platform.