Ipsen's Iqirvo Receives U.S. FDA Accelerated Approval for Primary Biliary Cholangitis Treatment

Iqirvo® (elafibranor) 80 mg tablets have been approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. This approval is significant as it introduces the first new treatment for PBC in nearly a decade, addressing a critical unmet need for patients with this rare liver disease.

Primary biliary cholangitis is a rare, autoimmune, cholestatic liver disease that affects approximately 100,000 people in the U.S., predominantly women. If left untreated, PBC can lead to liver failure and, in some cases, necessitate a liver transplant. The disease also significantly impacts patients' quality of life, with symptoms including severe fatigue and debilitating itch (pruritus).

The accelerated approval of Iqirvo is based on data from the Phase III ELATIVE trial, which demonstrated that 51% of patients treated with Iqirvo plus UDCA achieved a biochemical response compared to 4% of those treated with placebo plus UDCA. The trial also showed normalization in ALP levels in 15% of Iqirvo-treated patients versus 0% in the placebo group. ALP is a biochemical marker used as a surrogate endpoint in PBC trials.

Iqirvo is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. The most common adverse reactions reported in ≥10% of study participants were weight gain, abdominal pain, diarrhea, nausea, and vomiting. The drug has also been associated with serious risks, including myalgia, myopathy, rhabdomyolysis, fractures, adverse effects on fetal and newborn development, drug-induced liver injury, hypersensitivity reactions, and biliary obstruction.

The FDA's approval of Iqirvo under the accelerated approval program is contingent upon the verification of clinical benefit in confirmatory trials. Iqirvo has been submitted to the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) for authorization, with decisions anticipated in the second half of 2024.

This development represents a significant advancement in the treatment of PBC, offering hope to patients for whom existing treatments have been inadequate. Ipsen's commitment to addressing rare cholestatic liver diseases is further solidified by this approval, enhancing its portfolio of treatments available to patients in the U.S.