NICE Recommends New Treatment for Rare Liver Condition

Dr Sheena Meredith

January 14, 2022

Editor's note: This article has been altered to add a comment from the Children's Liver Disease Foundation

A novel drug for a rare inherited liver disease was today approved for use in England by the National Institute for Health and Care Excellence (NICE). The drug, odevixibat, offers a new treatment option for people with progressive familial intrahepatic cholestasis (PFIC).

NICE’s final draft highly specialised technologies guidance recommends odevixibat in line with its marketing authorisation as an option for people aged 6 months and older with all types of PFIC.

The autosomal recessive condition results from mutations in genes encoding proteins involved in transporting bile out of the liver, reducing or stopping the outflow of bile acids and causing metabolic cholestasis that can lead to serious liver damage. Symptoms and signs most often start during the first months of life and include jaundice, growth impairment and severe pruritus.

Progression is at varying rates depending on the type of PFIC, but usually cirrhosis develops within the first decade of life and most patients develop progressive liver fibrosis and ultimately end stage liver disease requiring liver transplantation. In some forms there is a high risk of liver tumour development. The condition is fatal if untreated. Its prevalence in England is unknown, but worldwide estimates are between 1 per 50,000 to 1 per 100,000 live births.

First Licensed Treatment for PFIC

Previous treatment options included nutritional support and anti-pruritics, followed by biliary diversion procedures in selected patients and ultimately transplantation. Other medications including ursodeoxycholic acid, rifampicin, and cholestyramine have sometimes been used off-label to combat cholestatic pruritus, but odevixibat is the first licensed treatment for PFIC.

Odevixibat (Bylvay, Albireo Pharma) is a selective inhibitor of ileal bile acid transporter (IBAT), a protein in the intestine that transports bile acid from the small intestine back to the liver. It thereby increases their excretion through the colon and thus lowers hepatic and serum bile acid levels and reduces subsequent liver damage.

The drug received a marketing authorisation within the EU in July 2021 on the basis of initial trial data suggesting it may delay disease progression and the need for surgery and/or liver transplantation in PFIC. Odevixibat is also in clinical development for the treatment of other cholestatic diseases, including Alagille syndrome and biliary atresia.

Today’s positive recommendation by NICE follows an improved commercial offer from the company, with a discount on the drug price. In addition the assessment committee felt that some of the company’s economic assumptions underestimated the cost effectiveness of odevixibat and did not capture all benefits, including the drug’s innovative nature and the invasive features of other treatments.

Taking all this into consideration, the committee agreed that odevixibat is cost-effective use of NHS resources within the context of a highly specialised service. It is available as 200µg, 400µg, 600µg and 1200µg hard capsules, and its approximate average annual list price per patient is £255,000. 

A spokesperson for the Children’s Liver Disease Foundation told Medscape UK that today’s announcement was "brilliant news". Chief Executive, Alison Taylor described it as a "watershed moment". She said: "When the final stage of the drug approval process is concluded, this ground-breaking drug has the potential to transform the lives of children and young people living with PFIC."

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