Ipsen’s Iqirvo (elafibranor) has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) to treat adults with primary biliary cholangitis (PBC).
The oral peroxisome proliferator-activated receptor agonist has been authorised for use in combination with ursodeoxycholic acid (UDCA) in patients with an inadequate response to UDCA, and as a monotherapy in those who are unable to tolerate UDCA.
Affecting approximately 25,000 people in the UK, the majority of whom are women, PBC is a rare cholestatic liver disease that causes irreversible fibrosis (scarring) of the liver and destruction of the bile ducts.
The most common symptoms of PBC are pruritus, or itching, and fatigue, and the condition can worsen over time if not effectively treated.
The MHRA’s decision makes Iqirvo the first new treatment for PBC to be granted marketing authorisation in almost a decade and was supported by positive results from the late-stage ELATIVE trial, in which 51% of patients receiving Iqirvo achieved a cholestasis response at week 52, compared to 4% of those in the placebo group.
Additionally, 15% of Iqirvo-treated patients demonstrated ALP normalisation versus 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.
The drug has now been submitted for review by the National Institute for Health and Care Excellence (NICE), with a decision from the health technology assessment agency expected later this year.
David Montgomery, UK and Ireland medical director at Ipsen, said: “[Iqirvo] is the first medicine for PBC in nearly a decade, offering a promising new treatment option for people living with this rare condition. This is especially significant for those who may need an additional option beyond current first-line treatments.
“The MHRA’s decision represents real progress and we’re now committed to working with NICE to obtain reimbursement and ensure people living with PBC who are eligible can access [Iqirvo].”
The authorisation comes less than three weeks after Iqirvo was approved by the European Commission to treat adults with PBC. The drug was also approved by the US Food and Drug Administration in June for the same patient population.