NICE approves elafibranor for treatment of rare liver disease

Interim funding for elafibranor has been provided through the Innovative Medicines Fund (IMF), ensuring immediate access for eligible patients

The National Institute for Health and Care Excellence (NICE) has recommended elafibranor 80mg tablets (marketed as IQIRVO) for treating primary biliary cholangitis (PBC), a rare liver disease.

Developed by Ipsen, IQIRVO is the first medicine for PBC approved for use on the NHS in nearly a decade.

Elafibranor is indicated for use in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy for those who cannot tolerate UDCA.

PBC is a lifelong condition that can worsen over time if inadequately treated, potentially leading to liver failure, the need for a liver transplant, or, in rare instances, premature death.

Approximately 25,000 people in the UK are affected by PBC—90 per cent of whom are women. Early-stage PBC patients often suffer from severe fatigue and a persistent, debilitating itch known as pruritus.

Dr. David Montgomery, medical director for Ipsen in the UK and Ireland, 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.”

Interim funding for elafibranor has been made available through the Innovative Medicines Fund (IMF), allowing eligible patients immediate access.  This support will continue until NICE publishes its final Technology Appraisal, following which the treatment will be routinely funded by the NHS.

Professor David Jones, Professor of Liver Immunology at Newcastle University, emphasised the significance of this approval, calling it “an important step forward in the management of this potentially life-threatening rare liver condition.”

“The availability of a new therapeutic option on the NHS for PBC is welcome news for healthcare teams supporting patients with this debilitating and often misunderstood condition,” he added.

How elafibranor works

PBC is a rare, autoimmune, cholestatic liver disease that causes a build-up of bile and toxins (cholestasis) and chronic inflammation, leading to irreversible fibrosis (scarring) of the liver and destruction of the bile ducts.

While its exact causes remain unclear, PBC is associated with genetic and environmental factors.

Elafibranor is a first-in-class, oral, once-daily peroxisome proliferator-activated receptor (PPAR) α/δ agonist.

By activating PPARα and PPARδ, elafibranor reduces bile toxicity, decreases inflammation, and improves cholestasis.

The NICE recommendation is based on the Phase III ELATIVE trial results, which demonstrated that 51 per cent of patients treated with elafibranor plus UDCA achieved the composite primary endpoint of a cholestasis response at week 52, compared to just 4 per cent in the placebo plus UDCA group.

A post-hoc analysis of the ELATIVE trial further revealed that 58 per cent of patients taking elafibranor reported a reduction in itching compared to 27per cent in the placebo group at week 52, and 80 per cent of elafibranor patients reported reduced or no sleep disturbances, versus 30per cent in the placebo group.

Mo Christie, head of patient services at The PBC Foundation, highlighted the importance of this new treatment, noting that PBC primarily affects women around menopausal age and is often diagnosed late or not taken seriously.

She sees elafibranor as significant step forward in raising awareness, promoting earlier diagnosis, and improving disease management for those living with PBC.