The Scottish Medicines Consortium (SMC) has today recommended elafibranor as a second-line treatment for primary biliary cholangitis (PBC).
This is the first new PBC drug accepted by the SMC in nearly a decade, offering an additional treatment option for people living with PBC in Scotland. The decision closely follows the approval of elafibranor by the National Institute for Health and Care Excellence (NICE) in October 2024, where the British Liver Trust collated insight from patients and submitted evidence to NICE as part of the process.
Primary biliary cholangitis (PBC) is an autoimmune liver condition that targets the liver’s bile ducts. In this condition, the immune system mistakenly attacks these ducts, causing damage. As a result, bile can leak from the affected ducts and harm the liver. Although PBC typically advances slowly, the damage can accumulate over time, potentially leading to cirrhosis and, in some cases, liver failure.
PBC impacts approximately 1,900 people in Scotland, affecting approximately nine women for every one man around the ages of 40-60 years.
The primary treatment for PBC is ursodeoxycholic acid, often referred to as UDCA or urso. It’s effective for many people, but not for everyone—some find it doesn’t adequately control the condition, while others experience side effects that prevent them from taking it. Until recently, the only other approved medication for PBC was obeticholic acid (OCA), which, like urso, isn’t suitable for all patients. As a result, some people have been left without a treatment option that works well for them.
“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.”
The detailed advice document from the SMC can be seen here.