Ipsen’s Iqirvo approved for use in NHS Scotland to treat rare liver disease
Ella Day | April 8, 2025 | News story | Research and Development | R&D, Rare Diseases, Scottish Medicines Consortium, ipsen, primary biliary cholangitis (PBC)
The Scottish Medicines Consortium (SMC) has accepted the use of Ipsen’s Iqirvo (elafibranor) in NHS Scotland for the treatment of primary biliary cholangitis (PBC), a rare liver disease. This marks the first new medicine for PBC to be accepted by the medical body in nearly a decade.
Iqirvo is a peroxisome proliferator activated receptor (PPAR) agonist. It is a treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA. It reduces bile toxicity and inflammation, and improves cholestasis.
PBC is a rare, autoimmune, cholestasis liver disease. It is a lifelong disease, affecting approximately nine women for every man (aged 40 to 60 years). Its early symptoms and typical age of diagnosis mean that PBC can be mistaken for ageing or menopause, impacting effective treatment. If inadequately treated, PBC can worsen over time and cause liver failure, leading to liver transplant and even premature death.
The approval follows successful results from Ipsen’s ELATIVE trial, which evaluated the efficacy and safety of Iqirvo against a placebo, both used with UDCA. It demonstrated that Iqirvo had a statistically significant treatment benefit, with 51% of patients treated with Iqirvo plus UDCA achieving the primary endpoint of cholestasis response compared to only 4% of patients in the placebo group.
The trial reflects Ipsen’s focus on treatments in oncology, rare disease and neuroscience. The approval of Iqirvo follows the acceptance of its cabozantinib monotherapy for the treatment of hepatocellular carcinoma (HCC) – a primary liver cancer by the SMC in October 2024.
John Dillon, consultant hepatologist and professor of hepatology and gastroenterology at the University of Dundee, UK, commented that the acceptance “offers a significant option to patients and healthcare teams who may have been unable to slow PBC progression with existing treatments”.
He added: “It is an important new therapeutic agent and a welcome addition in managing a condition that is often debilitating for patients and life threatening for some.”
Ella Day
8/4/25
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