Ipsen has received US Food and Drug Administration (FDA) accelerated approval for Iqirvo (elafibranor) 80mg tablets for primary biliary cholangitis (PBC).
The treatment is indicated for usage in combination with ursodeoxycholic acid (UDCA) for adult patients with an inadequate response to UDCA, or as monotherapy in patients who are intolerant to UDCA.
Ipsen will handle the marketing and commercialisation of elafibranor under the trademark name Iqirvo and will make it available immediately for prescription in the US for eligible patients.
The accelerated approval is based on the reduction of alkaline phosphatase levels, although improvements in survival or liver decompensation event prevention are not established.
In the Phase III ELATIVE trial, 13 times more patients attained the composite primary endpoint of biochemical response following Iqirvo plus UDCA treatment than did so with placebo plus UDCA.
Secondary endpoints showed ALP level normalisation in only Iqirvo-treated patients.
Continued approval for this indication will depend on results and clinical benefits observed in confirmatory trials.
Iqirvo is not recommended for individuals with decompensated cirrhosis, characterised by conditions such as ascites, variceal bleeding or hepatic encephalopathy.
GENFIT discovered and developed elafibranor with Ipsen licensing the sole global rights for the asset in 2021, excluding China, Hong Kong, Macau and Taiwan.
GENFIT is entitled to receive milestone payments of €89m ($95.7m) from Ipsen.
This comprises €48.7m on the first commercial sale of Iqirvo in the US, in addition to a €13.3m in milestone payment obtained in February 2024.
Ipsen Research and Development head and executive vice-president Christelle Huguet stated: “For a significant number of people living with PBC, available treatments do not control the condition and may exacerbate symptoms of PBC. Left unmanaged, PBC can progress, leading to liver failure and in some cases, the need for a liver transplant.
“Iqirvo demonstrated statistically significant improvements in biochemical response compared to UDCA alone. Iqirvo is therefore a much-needed treatment option and the first new medicine for PBC in nearly a decade.”
The latest development comes after Ipsen expanded its ongoing oncology research partnership with Marengo Therapeutics to advance T cell engagers using TriSTAR, Marengo’s antibody fusion platform.
