MHRA approves Ipsen’s Iqirvo for adult primary biliary cholangitis

Ipsen’s Iqirvo (elafibranor) has received approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of adults with primary biliary cholangitis (PBC). This oral peroxisome proliferator-activated receptor agonist is indicated for use in combination with ursodeoxycholic acid (UDCA) in patients who do not respond adequately to UDCA, and as a standalone therapy for those who cannot tolerate UDCA.

PBC is a rare cholestatic liver disease that affects about 25,000 people in the UK, most of whom are women. The disease leads to irreversible liver fibrosis (scarring) and bile duct destruction. Common symptoms include pruritus (itching) and fatigue, and without effective treatment, the condition can progressively worsen over time.

The MHRA’s recent authorisation marks Iqirvo as the first new treatment for PBC in almost ten years. This decision was supported by positive outcomes from the ELATIVE late-stage clinical trial. In this study, 51% of patients treated with Iqirvo showed a cholestasis response at week 52, compared to just 4% in the placebo group. Additionally, 15% of the patients on Iqirvo achieved ALP normalisation at week 52, whereas none in the placebo group did. While a greater decrease in pruritus intensity was seen in patients treated with Iqirvo compared to the placebo group, this did not reach statistical significance.

The drug has also been submitted for evaluation to the National Institute for Health and Care Excellence (NICE), with a decision expected later in the year.

David Montgomery, the UK and Ireland medical director at Ipsen, commented on the approval, highlighting that Iqirvo is the first PBC medicine in almost a decade. He underscored its significance as a new treatment option, particularly for patients needing alternatives beyond existing first-line therapies. He also noted the MHRA’s decision as a significant step forward and expressed Ipsen’s commitment to working with NICE to secure reimbursement, ensuring eligible PBC patients can access Iqirvo.

Less than three weeks prior to this approval, Iqirvo was also approved by the European Commission for the treatment of adults with PBC. Furthermore, the US Food and Drug Administration (FDA) granted its approval for the same patient group in June.