Intercept's Ocaliva has been issued a final green light for use by the NHS to treat the rare chronic liver disease primary biliary cholangitis (PBC), which affects around one in 1,000 women over the age of 40 but is still the leading cause of liver transplantation in adult women in the UK.
Ocaliva (obeticholic acid) can now be routinely prescribed via the NHS to treat patients with the condition 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.
Regulatory clearance was issued late last year on the back of data showing that nearly half of patients (46 percent) treated with Ocaliva in combination with UDCA (the only other treatment available for the condition) achieved a significant reduction in serum alkaline phosphatase (ALP) compared to 10 percent in the control group.
Also, 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.