The National Institute for Health and Care Excellence (NICE) in the UK has approved Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) for routine use by the National Health Service (NHS) in England, Wales and Northern Ireland.
It has been conditionally approved in the European Union to treat primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults.
Ocaliva is expected to be available to patients with PBC within 90 days of NICE's final appraisal publication.
Intercept Pharmaceuticals international president Lisa Bright said: "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.
"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."
Ocaliva is a new treatment option for patients with PBC.
In December last year, it received conditional marketing authorisation in Europe based on efficacy and safety data derived from three clinical trials.
The most commonly reported adverse reactions were pruritus (63%) and fatigue (22%).
PCB is a rare, autoimmune, cholestatic liver disease that puts patients at risk of life-threatening complications. It is primarily a disease in women, afflicting approximately one in 1,000 females older than 40.