NICE Recommends Ipsen’s Iqirvo for Primary Biliary Cholangitis

Ipsen's medication Iqirvo (elafibranor) has gained a recommendation from the National Institute for Health and Care Excellence (NICE) for treating adults with primary biliary cholangitis (PBC), a rare liver disorder. This endorsement marks a significant milestone as it makes Iqirvo the first new treatment for PBC to be approved for use by the NHS in nearly a decade. The approval was granted two weeks after the Medicines and Healthcare products Regulatory Agency provided marketing authorization for the drug.

Iqirvo can be administered alongside ursodeoxycholic acid (UDCA) for patients who do not respond adequately to UDCA alone. It can also be used as a standalone treatment for individuals who cannot tolerate UDCA. The medication is intended to address PBC, a condition characterized by the accumulation of bile and toxins (cholestasis) which leads to chronic inflammation and eventually irreversible liver scarring and bile duct destruction. In the UK, the disease affects around 25,000 people, predominantly women, and is accompanied by severe symptoms such as persistent itching (pruritus) and extreme fatigue. If not managed effectively, PBC can progress and result in liver failure.

Iqirvo is taken orally once daily and functions as a peroxisome proliferator-activated receptor agonist. It works by reducing bile toxicity and improving cholestasis through the modulation of bile acid synthesis, detoxification, and transport.

The recommendation from NICE was based on encouraging findings from the phase 3 ELATIVE trial. Results showed that 51% of patients treated with Iqirvo in combination with UDCA achieved a cholestasis response at week 52, compared to just 4% in the placebo group also receiving UDCA. Additionally, 15% of patients on Iqirvo showed normalization of alkaline phosphatase (ALP) levels, compared to none in the placebo group. While a reduction in pruritus intensity was observed in patients treated with Iqirvo, it did not reach statistical significance.

Iqirvo will be made available immediately through the Innovative Medicines Fund, which aims to provide quicker access to new treatments while further data is gathered.

David Montgomery, UK and Ireland medical director at Ipsen, commented on the approval, emphasizing its importance during a critical time. He highlighted that current treatments do not effectively manage both the progression of PBC and its life-impacting symptoms, like itching and sleep disturbances. He expressed hope that this approval will improve outcomes for those living with PBC.