Is Elafibranor-Iqirvo available?

Elafibranor-Iqirvo produced by Ipsen Biopharmaceuticals, Inc was approved by the US Food and Drug Administration (FDA) on June 10, 2024, with the trade name Iqirvo, becoming the first approved new drug for primary cholangitis (PBC) in the past decade. Iqirvo was licensed from GENFIT in 2021, and continued approval may be contingent on positive clinical benefit in further clinical trials.

Iqirvo is a first-in-class, once-daily oral peroxisome proliferator-activated receptor (PPAR) agonist. The marketing approval is based on the reduction of alkaline phosphatase (ALP) without increasing hepatotoxicity. Improved survival or prevention of hepatic decompensation events have not been demonstrated. For a considerable number of PBC patients, existing treatments cannot control the disease, but may worsen the symptoms of PBC. If left unmanaged, PBC can worsen, leading to liver failure and, in some cases, the need for a liver transplant.

Related trials showed that 13 times more patients achieved the composite primary biochemical response endpoint when Iqirvo plus UDCA was compared with placebo plus ursodeoxycholic acid (UDCA) (51% vs. 4%, respectively, treatment difference 47%). ALP is a biochemical marker used as a surrogate endpoint in PBC trials. A secondary endpoint showed normalization of ALP levels in only Iqirvo-treated patients. The majority of patients (95%) received study treatment (Ikirvo or placebo) in combination with UDCA.

Iqirvo demonstrated statistically significant improvements in biochemical responses compared to UDCA alone As a result, Iqirvo is a much-needed treatment option and the first new drug for PBC in nearly a decade. But Iqirvo is not recommended for people who have or develop decompensated cirrhosis (such as ascites, variceal bleeding, hepatic encephalopathy).Iqirvo is not recommended.