dm+d

19822411000001108

New Medicines

Chenodeoxycholic acid Leadiant Cerebrotendinous xanthomatosis (CTX) or inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency

Information

Chenodeoxycholic acid Leadiant
New molecular entity
Leadiant Biosciences
Leadiant Biosciences

Development and Regulatory status

Launched
Launched
Phase III Clinical Trials
July 2019
Yes
Yes
Jul 19Available in the UK. Price 1 x 100 Tablets = £14000 [12].
Jan 19Still to be launched in the EU [11].
Jan 19Currently PIII in US [10].
Jan 19NICE is helping to develop an NHS England specialised clinical commissioning policy for chenodeoxycholic acid. A scope was published in Jan 18 [9].
Apr 17European Commission grants a marketing authorisation valid throughout the European Union [8].
Sep 16EU positive opinion for treatment of inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis) in infants, children and adolescents aged 1 month-18 years and adults [7].
Nov 15Filed in EU via centralised procedure [6].
Mar 15Xenbilox listed as NDA/preparation in the US in the Sigma-Tau pipeline; EU plans not stated [4].
Dec 14Granted orphan drug status in the EU (EU/3/14/1406) for treatment of inborn errors in primary bile acid synthesis [5].
Feb 10Filed in the US [1].
Jul 07Orphan drug designation granted by the US FDA [1].

Category

A cholesterol inhibitor, reducing the cholesterol concentration of bile. Chenodeoxycholic acid is one of the main bile acids produced by the liver.
CTX is a rare autosomal metabolic disorder due to mutation of the gene CYP27A1 encoding sterol 27-hydrolase. This enzyme is essential in turning cholesterol into bile acids and if not working properly, cholesterol and precursors of bile acids accumulate in various tissues resulting in xanthomatous swelling of tendons, early formation of cataract and progressive neurological symptoms, including dementia, seizures, hallucinations and depression [1]. Prevalence 0.2 in 10,000 people in the EU [2].
Cerebrotendinous xanthomatosis (CTX) or inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency
Oral

Trial or other data

Mar 15Chenodeoxycholic acid is used off-label in the UK for CTX [3].

Evidence based evaluations