EDINBURGH – Patients in Scotland suffering from a rare and debilitating autoimmune liver disease will soon have access to the first new treatment in nearly ten years, as Ipsen’s Iqirvo (elafibranor) has been accepted for use by NHS Scotland, the pharmaceutical company confirmed Monday.
The Scottish Medicines Consortium (SMC) approved Iqirvo for use in primary biliary cholangitis (PBC), a rare, chronic liver condition that can lead to liver failure if left inadequately treated. The green light comes after strong results in clinical trials and marks a key milestone for Ipsen’s expanding rare disease portfolio.
Targeting a gap in care
Iqirvo, a peroxisome proliferator-activated receptor (PPAR) agonist, is indicated for use alongside ursodeoxycholic acid (UDCA) in adults whose disease is not sufficiently controlled by UDCA alone—or as monotherapy for patients who cannot tolerate it. The treatment works by reducing bile toxicity and inflammation and improving cholestasis, a process impaired in PBC.
The SMC’s decision reflects an unmet medical need for patients suffering from PBC, which affects nine times more women than men, typically diagnosed between the ages of 40 and 60. Its early symptoms—often including fatigue and itchiness—can be mistaken for signs of menopause or general ageing, delaying diagnosis and treatment.
Without adequate therapy, the disease can progress to cirrhosis, liver transplant, or premature death.
Results from ELATIVE trial tipped the scale
The SMC’s acceptance follows promising data from Ipsen’s ELATIVE trial, which evaluated the efficacy and safety of Iqirvo versus placebo, both used in combination with UDCA. The results were compelling:
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51% of patients treated with Iqirvo plus UDCA achieved the primary endpoint of cholestasis response,
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compared with just 4% in the placebo group.
This marked statistically significant progress in managing a disease with limited options for those who fail first-line UDCA therapy.
Expert endorsement
Dr. John Dillon, consultant hepatologist and professor at the University of Dundee, welcomed the approval as a long-awaited development for the medical community:
“It offers a significant option to patients and healthcare teams who may have been unable to slow PBC progression 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.”
Ipsen doubles down on liver portfolio
The Iqirvo approval builds on Ipsen’s growing liver-focused drug pipeline. It follows the October 2024 SMC approval of cabozantinib monotherapy for hepatocellular carcinoma (HCC)—a common primary liver cancer.
Ipsen’s strategy emphasizes expansion in oncology, rare diseases, and neuroscience, where regulators are increasingly responsive to high-need patient populations and clinically differentiated therapies.
The broader adoption of Iqirvo in the UK and internationally will now depend on individual health authorities and pricing decisions in the coming months.
