Ipsen has announced that its Iqirvo (elafibranor) tablets have been accepted by the Scottish Medicines Consortium (SMC) to treat adults with the rare cholestatic liver disease primary biliary cholangitis (PBC).
The drug can now be used on the NHS in Scotland alongside ursodeoxycholic acid (UDCA) in patients with an inadequate response to UDCA, or as a monotherapy in those who are unable to tolerate UDCA.
The decision makes Iqirvo the first medicine for PBC to be accepted by the SMC in nearly a decade and comes after the National Institute for Health and Care Excellence recommended the drug for PBC patients on the NHS in England and Wales in October.
Affecting approximately 1,900 people in Scotland, PBC is a life-long condition that leads to irreversible scarring of the liver and the destruction of bile ducts.
The disease disproportionately impacts women and is associated with symptoms such as persistent, debilitating itch, known as pruritus, and severe fatigue. The condition can also worsen over time and cause liver failure if not adequately treated.
The SMC’s decision was based on results from the late-stage ELATIVE trial, in which 51% of patients receiving Iqirvo in combination with UDCA achieved the composite primary endpoint of cholestasis response at week 52 compared to 4% of those in the placebo plus UDCA group.
Additionally, 15% of Iqirvo-treated patients demonstrated alkaline phosphatase normalisation compared to 0% on placebo at week 52, and a greater decrease in pruritus intensity was observed in Iqirvo-treated patients compared to placebo, but this did not reach statistical significance.
A post-hoc study of the trial also showed that 58% of patients in the Iqirvo group reported less time itching compared to 27% of those on placebo at week 52, and 80% of Iqirvo-treated patients reported reduced or no sleep disturbance versus 30% on placebo
John Dillon, consultant hepatologist and professor of hepatology and gastroenterology at the University of Dundee, said: “The SMC acceptance offers a significant option to patients and healthcare teams who may have been unable to slow the progression of PBC with existing treatments.
“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.”
