Ipsen has shared positive three-year results for Iqirvo (elafibranor) in patients with the rare cholestatic liver disease primary biliary cholangitis (PBC).
Interim results from the open-label extension (OLE), which has been evaluating the drug in 138 patients who completed the double-blind period of the phase 3 ELATIVE study, were presented at this year’s American Association for the Study of Liver Disease congress.
In patients receiving three years of continuous Iqirvo treatment across the double-blind period and OLE, 85% had a biochemical response and 39% achieved ALP normalisation at week 156.
Improvements in pruritus were also shown to be sustained for patients with moderate or severe pruritus at baseline who were continuously receiving Iqirvo for up to 156 weeks, and surrogate markers of liver fibrosis suggest stabilisation when measured from baseline to week 130.
Approximately 100,000 people in the US, the majority of whom are women, are affected by PBC, which causes irreversible scarring of the liver and destruction of the bile ducts.
The most common symptoms of the disease are pruritus, or itching, and fatigue, and the condition can worsen over time if not effectively treated, potentially leading to liver transplant and in some cases, premature death.
Taken orally once daily, Iqirvo is a peroxisome proliferator-activated receptor agonist that decreases bile toxicity and improves cholestasis by modulating bile acid synthesis, detoxification and transporters.
A primary investigator on the ELATIVE study, Kris Kowdley, director at the Liver Institute Northwest, said: “Over three years, Iqirvo data suggests sustained efficacy and supports the safety profile of the medicine. Importantly, when patients tell me they are less impacted by itch and fatigue – that matters to me as a physician.”
The results comes just one month after Iqirvo was recommended by the National Institute for Health and Care Excellence for use in combination with ursodeoxycholic acid (UDCA) in adult PBC patients with an inadequate response to UDCA, or as a monotherapy in those who are unable to tolerate UDCA.
The drug was also authorised by the US Food and Drug Administration and European Commission for PBC earlier this year.