Approvals

The Scottish Medicines Consortium (SMC) has approved Ipsen’s Iqirvo (elafibranor) in NHS Scotland to treat the rare liver disease primary biliary cholangitis (PBC).

The decision, facilitated by a confidential NHS Scotland Patient Access Scheme agreement with Ipsen, marks the first new medicine for PBC, a lifelong disease, to be approved for use in Scotland in nearly a decade.

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.”

Known as a peroxisome proliferator-activated receptor (PPAR) agonist, Iqirvo is used to treat 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 that affects approximately nine women for every man (aged 40 to 60 years). Its early symptoms and typical age of diagnosis mean it can be mistaken for ageing or menopause.

If inadequately treated, PBC can worsen over time and cause liver failure, leading to liver transplant and even premature death.

The SMC’s decision comes on the back of 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.