Regulatory Affairs

UK's NICE backs use of Intercept's Ocaliva for primary biliary cholangitis

Published 03 March 2017

Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) has been approved by the UK's National Institute for Health and Care Excellence (NICE) for routine use by the National Health Service (NHS) in England, Wales and Northern Ireland.

Ocaliva has been conditionally approved in the European Union for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.

The NHS is expected to make Ocaliva available to patients with PBC within 90 days of NICE's final appraisal publication and Intercept will work with local reimbursement authorities to help ensure eligible patients obtain access.

Although it is a rare disease, PBC is a leading cause of liver transplantation in adult women in the UK. Ocaliva is a new treatment option for patients with PBC who do not fully respond to, or are intolerant to, current treatment and remain at risk of their disease progressing toward cirrhosis, liver transplantation or death.

Liver Immunology at Newcastle University professor and Newcastle upon Tyne Hospitals Trust consultant hepatologist David Jones said: "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."

Ocaliva is a potent and selective agonist of the farnesoid X receptor (FXR), which is expressed at high levels in the liver and intestine and thought to be a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways.

In December 2016, Ocaliva received conditional marketing authorization in Europe based on efficacy and safety data derived from three randomized double-blind, placebo-controlled clinical trials evaluating the effect of Ocaliva on alkaline phosphatase (ALP) and bilirubin in patients with PBC.

The marketing authorization was also supported by two clinical databases that include more than 10,000 patients from the Global PBC Study Group and UK-PBC Group, both independently confirming that achieving lower ALP and/or bilirubin levels is significantly correlated with increased transplant-free survival.

The most commonly reported adverse reactions were pruritus (63%) and fatigue (22%). Adverse reactions leading to discontinuation were 1% in the Ocaliva titration arm and 11% in the Ocaliva 10 mg arm. The most common adverse reaction leading to discontinuation was pruritus. The majority of pruritus occurred within the first month of treatment and tended to resolve over time with continued dosing.

Intercept 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."

The PBC Foundation CEO Thain MBE said: "It is exciting news for PBC patients that this new treatment option will now be routinely available in England, Wales and Northern Ireland," said Collette Thain MBE, CEO of The PBC Foundation.

"When I was diagnosed with PBC, UDCA was the only approved treatment option and PBC wasn't a major priority for many researchers. Thankfully, so much has changed for people living with PBC since then.

“After decades of advocacy from the PBC community, we have a new treatment option, a growing awareness of the disease among the general public, greater expertise amongst clinicians and an acceleration of PBC research in the UK and around the globe."

Source: Company Press Release