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Elafibranor is indicated for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA1
Elafibranor is a first-in-class treatment for primary biliary cholangitis (PBC) – associated with severe fatigue and persistent, debilitating itch1,2,3
PBC is a life-long condition that affects approximately 1,900 people in Scotland – nine in 10 of which are women4
If inadequately treated, PBC can worsen over time and cause liver failure, leading to liver transplant and in rare cases, premature death5,6,7
The SMC has also accepted Cabozantinib Ipsen as monotherapy for the treatment of hepatocellular carcinoma (HCC) in adults who have previously been treated with sorafenib, demonstrating Ipsen’s commitment to supporting patients with rare liver disease in Scotland8
LONDON, U.K., 7 April, 2025 – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Scottish Medicines Consortium (SMC) has accepted elafibranor 80mg tablets for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.9 This makes elafibranor the first new medicine for PBC to be accepted in nearly a decade, and the first in its class.10,11
“The SMC acceptance offers a significant option to patients and healthcare teams who may have been unable to slow the progression of PBC with existing treatments,” commented Professor John Dillon, Consultant Hepatologist and Professor of Hepatology and Gastroenterology at the University of Dundee. “It is an important new therapeutic agent and a welcome addition in managing a condition that is often debilitating for patients and life threatening for some.”
PBC is a rare, autoimmune, cholestatic liver disease. People with PBC in its earlier stages commonly experience severe fatigue and a persistent, debilitating itch, known as pruritus.2,3 The disease can worsen over time if inadequately treated, causing liver failure, leading to liver transplant and in rare cases, premature death.6,7
A lifelong disease, PBC impacts approximately 1,900 people in Scotland,4 affecting approximately nine women for every one man around the ages of 40-60 years.5,12 Despite the critical impact of the disease in its advanced staged, early symptoms and its typical age of diagnosis mean it can be mistaken for ageing or the menopause, impacting effective management.
A newly launched report from the PBC Foundation and Ipsen observed that patients in Scotland can experience a marked difference in diagnosis and support. The report calls for a national consensus pathway for the care of people with PBC in Scotland.
“PBC can have a massive impact on the lives of people living with the condition, but in some cases the symptoms aren’t being taken seriously” commented Mo Christie, Head of Patient Services at the Dunfermline-based PBC Foundation. “New treatment options not only help those living with PBC but may also raise awareness of the difficulties and stigma faced, such as liver conditions being associated with alcohol misuse. Our report into the PBC care pathway in Scotland calls for all patients to receive NHS-endorsed information at the point of diagnosis, to help tackle this misunderstanding and stigma.”
The disease 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.7 While the causes of PBC are unknown, it is not caused by alcohol consumption but is associated with a combination of genetic risks and other environmental triggers.13
Elafibranor is a first-in-class, oral, once-daily peroxisome proliferator-activated receptor (PPAR) α/δ agonist.1,14 Activation of PPARα and PPARδ reduces bile toxicity, reduces inflammation and improves cholestasis.1
The SMC’s decision closely follows the approval of elafibranor by the National Institute for Health and Care Excellence (NICE) in October 2024,15 and is based on data from the Phase III ELATIVE trial published in the New England Journal of Medicine.14 In the ELATIVE trial 51% (55/108) of patients treated with elafibranor plus UDCA achieved the composite primary endpoint of cholestasis response* at week 52 compared to 4% (2/53) of patients in the placebo plus UDCA group.14
Secondary endpoints were normalisation of alkaline phosphatase (ALP) levels at week 52 and a change in pruritus intensity from baseline through week 52 and through week 24.14 The ELATIVE trial showed normalisation in ALP levels in only elafibranor-treated patients (15% for elafibranor plus UDCA (n=108) versus 0% for placebo plus UDCA (n=53)). A greater decrease in pruritus intensity, based on PBC Worst Itch Numeric Rating Scale (PBC WI-NRS), was observed in elafibranor-treated patients compared to placebo but this was not statistically significant.14
Additional patient-reported outcome measures were used to assess itch-related quality of life, including sleep and functioning, with greater improvements observed in patients treated with elafibranor compared to placebo.16 A post-hoc study of the ELATIVE trial found that 58% (14/24) of patients receiving elafibranor reported less time itching (improved duration) compared with 27% (3/11) on placebo at week 52,viii and 80% (20/25) of patients receiving elafibranor reported reduced or no sleep disturbance compared with 30% (3/10) on placebo.16
Elafibranor was generally well tolerated in the ELATIVE trial.14 Patients in the treatment group and the UDCA plus placebo group experienced similar percentages of adverse events, treatment-related adverse events, severe or serious adverse events or adverse events leading to discontinuation.14 Adverse events occurring in >10% of patients and more frequently on elafibranor versus placebo included abdominal pain (11%), diarrhoea (11%), nausea (11%), and vomiting (11%).14
The acceptance of elafibranor by the SMC follows the recent announcement (10 March 2025) that the SMC has recommended Ipsen’s cabozantinib as monotherapy for the treatment of HCC, a primary liver cancer which was responsible for 654 deaths in Scotland in 2023 and is the fastest rising cause of cancer death in the U.K.17,18 Cabozantinib is a protein kinase inhibitor that blocks mesenchymal epithelial transition factor (MET), vascular endothelial growth factor (VEGF) receptors and other tyrosine kinases including the GAS6 receptor (AXL).8
“The SMC’s recommendation of both elafibranor and cabozantinib demonstrates Ipsen’s commitment to bringing high value medicines to the U.K. to address unmet clinical and patient needs in rare liver diseases. These approvals are an important step in improving outcomes for patients and bringing parity of access across the U.K., following NICE approval of elafibranor in October last year”, said Dr David Montgomery, U.K. & Ireland Medical Director at Ipsen.
