Elafibranor, now Iqirvo, wins FDA accelerated approval for PBC

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Ipsen’s elafibranor as a second-line treatment for primary biliary cholangitis (PBC).

The oral therapy, which will be sold under the brand name Iqirvo, is indicated for use in combination with the first-line therapy ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy (on its own) in those who can’t tolerate UDCA.

The decision makes Iqirvo the first new therapy for PBC to win FDA approval since the clearance of Ocaliva (obeticholic acid) as a second-line therapy in 2016. Iqirvo is much-needed treatment option and the first new medicine for PBC in nearly a decade.

FDA’s accelerated approval pathway allows promising therapies to be marketed based on early clinical evidence that they’re probably effective. As a condition of accelerated approval, drug developers have to conduct further testing to prove the therapy provides clinical benefits as expected.

Iqirvo is available in 80 mg tablets, and the approved dosage is 80 mg once daily taken with or without food. To support patients accessing the newly authorized therapy, which will be immediately available in the U.S.

Iqirvo is also up for approval in the European Union and the U.K., with decisions expected in the second half of 2024.

PBC is an autoimmune disorder characterized by chronic inflammation in the bile ducts, a series of tubes that carry the digestive fluid bile out of the liver and to the intestines. This can lead to damage and scarring in the liver.

Originally developed by Genfit, Iqirvo works to modulate the activity of proteins called PPARs, which are involved in regulating gene activity related to processes that contribute to liver damage in PBC, including inflammation and scarring. The treatment is the first PPAR-targeting medication to be approved for PBC.

June 13, 2024