2 The technology

2 The technology

Description of the technology

Ocaliva, Intercept Pharma. Obeticholic acid is a selective and potent agonist for the farnesoid X receptor (FXR), a nuclear receptor expressed at high levels in the liver and intestine. FXR is thought to be an important regulator of bile acid, inflammatory, fibrotic and metabolic pathways. FXR activation lowers intracellular hepatocyte concentrations of bile acids by suppressing de novo synthesis from cholesterol, and by increasing transport of bile acids out of the hepatocytes. These mechanisms limit the overall amount of bile acid circulating in the body while promoting secretion of bile by the liver and reducing hepatic exposure to bile acids.

Marketing authorisation

A conditional marketing authorisation was received for the treatment of primary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid in people whose disease responded inadequately to ursodeoxycholic acid or as monotherapy in people who cannot tolerate ursodeoxycholic acid.

Adverse reactions

For full details of adverse reactions and contraindications, see the summary of product characteristics.

Recommended dose and schedule

The starting dose is 5 mg once daily. Based on an assessment of tolerability after 6 months, the dose should be increased to 10 mg once daily to have optimal response.

Price

Obeticholic acid 5 mg or 10 mg costs £2,384.04 per 30‑tablet pack. Costs may vary in different settings because of negotiated procurement discounts.

The company has agreed a patient access scheme with the Department of Health. This scheme provides a simple discount to the list price of obeticholic acid, with the discount applied at the point of purchase or invoice. The level of the discount is commercial in confidence. The Department of Health considered that this patient access scheme does not constitute an excessive administrative burden on the NHS.

  • National Institute for Health and Care Excellence (NICE)