Ipsen’s Iqirvo (elafibranor) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat adults with the rare liver disease primary biliary cholangitis (PBC).
The drug can now be used in combination with ursodeoxycholic acid (UDCA) in patients with an inadequate response to UDCA, or as a monotherapy in those who are unable to tolerate UDCA.
The recommendation makes Iqirvo the first medicine for PBC to be approved for use on the NHS in nearly a decade and comes just two weeks after the Medicines and Healthcare products Regulatory Agency granted marketing authorisation to the drug in this setting.
PBC is characterised by the build-up of bile and toxins (cholestasis) and chronic inflammation, which leads to irreversible scarring of the liver and destruction of the bile ducts.
The disease affects approximately 25,000 people in the UK, the majority of whom are 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 effectively treated.
Taken orally once daily, Iqirvo is a peroxisome proliferator-activated receptor agonist that decreases bile toxicity and improves cholestasis by modulating bile acid synthesis, detoxification and transporters.
NICE’s decision on the drug was supported by positive results from the phase 3 ELATIVE trial, in which 51% of patients receiving Iqirvo in combination with UDCA achieved a cholestasis response at week 52, compared to 4% of those in the placebo plus UDCA 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.
Iqirvo will be made immediately available via the Innovative Medicines Fund, which facilitates faster access to new treatments while further data is collected.
David Montgomery, UK and Ireland medical director at Ipsen, said: “NICE’s decision comes at a pivotal time as currently available treatments do not effectively manage both PBC disease progression and life-impacting symptoms like itch and sleep disturbances. This approval is an important step for us in improving outcomes for people living with PBC…”
