Intercept's Ocaliva is being backed by the National Institute for Health and Care Excellence for routine NHS commissioning as a treatment for people with the chronic liver disease primary biliary cholangitis (PBC).
The autoimmune disease is rare - afflicting around one in 1,000 women over the age of 40 - but still the leading cause of liver transplantation in adult women in the UK.
Ocaliva (obeticholic acid) 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.
The drug, is a highly selective agonist of the farnesoid X receptor (FXR), a nuclear receptor expressed in the liver and intestine that regulates bile acid, inflammatory, fibrotic and metabolic pathways.
In a pivotal Phase III clinical trial of the drug, nearly half of patients (46 percent) in the titration group treated with Ocaliva in combination with UDCA achieved the primary endpoint of achieving a significant reduction in serum alkaline phosphatas (ALP) compared to 10 percent in the control group, while 77 percent of patients taking the combination achieved a reduction of more than 15 percent in ALP at 12 months, compared to 29 percent taking UDCA alone.
The treatment was only approved by EU regulators mid-December by EU regulators, giving patients the potential to access the first new therapy for the condition in nearly 20 years.
According to the firm, its availability addresses an important unmet need as up to 40 percent of patients may have an inadequate response to UDCA, the only other available treatment for this condition.
"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 Lisa Bright, Intercept's president, International.
"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 has also welcomed the decision.
"This is a really important day for PBC patients and a great example of patients, scientists, doctors, industry and regulators working together to make difference," noted Professor David Jones, director of the UK-PBC group which undertook much of the work that led to the approval of Ocaliva.
"What is so important to say is that Ocaliva is manufactured in the UK and exported around the world so the UK is taking a lead in work that truly benefits people around the world."
"The development and rapid approval of Ocaliva is a major success story for the UK life science sector," added Mark Samuels, managing director of the National Institute for Health Research Office for Clinical Research Infrastructure (NOCRI).
"We are seeing the breakthrough results that working closely with industry and the PBC community has delivered for PBC patients. The government's Industrial Strategy is a clear commitment to invest in research and development, and Ocaliva demonstrates the potential the UK has to deliver new treatments that reach patients quickly where there is high unmet need."