FDA Approves Ipsen's Iqirvo for Primary Biliary Cholangitis in Adults

Ipsen’s Iqirvo (elafibranor) has recently received accelerated approval from the US Food and Drug Administration (FDA) for the treatment of primary biliary cholangitis (PBC) in adult patients. This approval allows the use of Iqirvo, a daily oral peroxisome proliferator-activated receptor agonist, in combination with ursodeoxycholic acid (UDCA) for adults who do not adequately respond to UDCA, or as a standalone therapy for those who cannot tolerate UDCA.

Primary biliary cholangitis is a rare, chronic autoimmune liver disease that affects around 100,000 people in the United States. This condition leads to the accumulation of bile and toxins in the liver, causing chronic inflammation, irreversible liver scarring, and bile duct destruction. Without proper treatment, PBC can progress to liver failure, necessitating a liver transplant.

The FDA’s approval of Iqirvo is based on the results of the phase 3 ELATIVE trial, which were published in the New England Journal of Medicine. The study revealed that patients treated with Iqirvo plus UDCA were 13 times more likely to achieve the composite primary endpoint of biochemical response compared to those treated with placebo plus UDCA. Additionally, secondary endpoints showed that 15% of patients treated with Iqirvo reached normal levels of alkaline phosphatase (ALP), a biochemical marker used in PBC trials, compared to 0% in the placebo group.

Christelle Huguet, Ipsen’s executive vice president and head of research and development, emphasized the significance of Iqirvo, calling it “the first new medicine for PBC in nearly a decade.” She highlighted that Iqirvo showed statistically significant improvements in biochemical response in comparison to UDCA alone, making it a crucial new treatment option for patients.

Dr. Kris Kowdley, director of the Liver Institute Northwest in Washington and the primary investigator of the ELATIVE study, stated that the approval of Iqirvo provides healthcare providers in the US with a new option to address an unmet need. He noted the potential of Iqirvo to significantly reduce ALP levels in PBC patients.

Iqirvo was initially discovered and developed by Genfit, with Ipsen acquiring the exclusive worldwide rights to the drug, except in China, Hong Kong, Taiwan, and Macau, in 2021. Applications for Iqirvo are also under consideration by the European Medicines Agency and the Medicines and Healthcare products Regulatory Agency, with final regulatory decisions anticipated in the second half of 2024.

In addition to its work on Iqirvo, Ipsen has recently broadened its oncology research collaboration with Marengo Therapeutics. This partnership, first announced in 2022, now includes Marengo’s T-cell engager platform, TriSTAR, in a deal valued up to $1.2 billion. This expansion aims to develop treatments for challenging 'cold' tumours, further demonstrating Ipsen’s commitment to advancing therapeutic options across various medical fields.