NICE approves first biliary cholangitis drug in 20 years

Intercept Pharmaceuticals has announced that Ocaliva (obeticholic acid) has been approved by the National Institute for Health and Care Excellence (NICE) for the treatment of the chronic liver disease primary biliary cholangitis (PBC), enabling NHS England and Wales to incorporate it into routine operation.

A selective agonist of the farnesoid X receptor (FXR) in the liver and intestine, Ocaliva was conditionally approved in the European Union two months ago for the same indication in combination with ursodeoxycholic acid (UDCA) in adult patients who have exhibited an inadequate response to UDCA, or as monotherapy in those unable to tolerate it. It becomes the first new treatment for the disease approved by NICE in almost 20 years.

PBC is a rare autoimmune cholestatic disease which is the leading cause of liver transplantation in adult women in the UK, affecting approximately around 20,000 people in the country and one in 1,000 women over the age of 40.

"This very rapid decision by NICE, one of the fastest approvals to date for an orphan medication, is an important affirmation of the scientific innovation, clinical value and cost-effectiveness of Ocaliva by one of the most respected health technology assessment bodies," said Intercept’s President, International Lisa Bright. "We welcome NICE’s decision to provide broad access to Ocaliva and we owe a tremendous debt to people living with PBC and the clinical groups who helped us to achieve this milestone for the PBC community."

David Jones, Professor of Liver Immunology at Newcastle University and Consultant Hepatologist at Newcastle upon Tyne Hospitals Trust, also commented on the implications of this approval:

"I am excited to see that the substantial group of PBC patients who are not achieving treatment goals with UDCA alone or who cannot tolerate UDCA will soon be able to access the first new therapeutic option in nearly 20 years. This truly is good news for our PBC patients. The development of new treatments for PBC is a powerful example of the medical innovation that can occur when government, industry, academia, community clinicians and, most importantly, patients come together to address an unmet need."