Ipsen reports positive three-year results for Iqirvo in primary biliary cholangitis

Ipsen has unveiled encouraging three-year outcomes for Iqirvo (elafibranor) in treating primary biliary cholangitis (PBC), a rare liver disease. These findings stem from an open-label extension (OLE) involving 138 patients who had completed the phase 3 ELATIVE study's double-blind segment. The data were shared at the recent American Association for the Study of Liver Disease congress.

Patients undergoing continuous Iqirvo treatment for three years across both the double-blind and OLE phases exhibited promising results. Specifically, 85% showed a biochemical response, and 39% achieved normal levels of alkaline phosphatase (ALP) at week 156. Additionally, patients with moderate or severe pruritus at the start of the treatment experienced sustained improvements in itching after up to 156 weeks of continuous Iqirvo therapy. Surrogate markers of liver fibrosis also suggested stabilization from the baseline to week 130.

PBC is a rare condition that affects around 100,000 people in the United States, with a majority being women. This disease leads to irreversible liver scarring and bile duct destruction. Common symptoms include pruritus (itching) and fatigue, which can progressively worsen without effective treatment, potentially resulting in liver transplants or premature death.

Iqirvo, taken orally once a day, is a peroxisome proliferator-activated receptor agonist. It works by decreasing bile toxicity and enhancing cholestasis through the modulation of bile acid synthesis, detoxification, and transporters.

Kris Kowdley, a primary investigator in the ELATIVE study and director at the Liver Institute Northwest, highlighted the significance of these findings. He noted that the three-year data for Iqirvo indicate sustained efficacy, reinforcing the safety profile of the medication. Kowdley emphasized the importance of patient feedback, particularly when they report reduced itching and fatigue, which significantly impacts their quality of life.

The positive results come shortly after the National Institute for Health and Care Excellence recommended Iqirvo for use with ursodeoxycholic acid (UDCA) in adult PBC patients who do not respond adequately to UDCA, or as a monotherapy for those unable to tolerate UDCA. Earlier this year, Iqirvo received authorization from both the US Food and Drug Administration and the European Commission for the treatment of PBC.