CHENODEOXYCHOLIC ACID LEADIANT (chenodeoxycholic acid), bile-acid based medicinal product

Low clinical benefit in cerebrotendinous xanthomatosis but no clinical benefit demonstrated within the therapeutic strategy

• CHENODEOXYCHOLIC ACID LEADIANT has MA in the treatment of congenital primary bile acid synthesis deficiency due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)

• Three retrospective analyses of the medical records of patients treated with chenodeoxycholic acid show a reduction in blood cholestanol concentration (biological criterion), with conflicting results on the clinical symptoms.

• The use of chenodeoxycholic acid is established in this disease.

• The safety profile appears to be favourable.