Leadiant GmbH, a fully owned subsidiary of Leadiant Biosciences Limited, has been granted Marketing Authorization by the European Commission for Chenodeoxycholic Acid Leadiant for the treatment of inborn errors of primary bile acid synthesis due to sterol-27-hydroxylase deficiency, presenting as cerebrotendinous xanthomatosis (CTX) in infants, children and adolescents aged 1 month to 18 years, and adults.1
Chenodeoxycholic Acid Leadiant is also listed in the EC Register of Orphan Medicinal Products. The orphan drug designation provides this product with a 10-year market exclusivity for the licensed indication.
CTX is an orphan inherited bile acid disorder that results in progressive neurological decline with severe motor and cognitive impairment and peripheral neuropathy that have a significant impact on patients’ life expectancy and quality. It is also associated with non-neurological manifestations that include neonatal cholestasis, diarrhoea and juvenile cataracts. CTX is estimated to affect a total of 200 patients in Europe.i
The Marketing Authorization application was supported by findings from two retrospective clinical studies carried out in major CTX treatment centres in Europe, one in The Netherlands and one in Italy. Both studies showed improvements in biochemical abnormalities and disease signs and symptoms resolved, improved or stabilized in a majority of patients over the course of the study.
The side effects seen with Chenodeoxycholic Acid Leadiant are constipation and abnormal liver test values; however, their frequency could not be reliably estimated from the available limited data. They were mild or moderate in severity and did not last.ii
“The license of Chenodeoxycholic Acid Leadiant will result in its consistent availability for CTX patients
and represents a necessary step to ensure patient access to this treatment in the EU”, said Dr. Antonio Gama da Silva, Managing Director at Leadiant GmbH and CEO of Leadiant Biosciences Ltd.
“Leadiant will be working alongside patient groups, healthcare professionals and regulatory bodies to make this a reality.”
For further information, please email info@leadiantbiosciences.com
About Leadiant Biosciences Ltd
Leadiant Biosciences is a research-based global pharmaceutical company that dedicates considerable resources to the development, registration and commercialization of novel and effective therapies that address patient needs and improve quality of life.
The company’s headquarters are based in London, UK.
i Orphanet E. Rare diseases in numbers [Available from: http://ec.europa.eu/health/archive/ph_threats/non_com/docs/rdnumbers.pdf.]
iI http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/004061/WC500226787.pdf
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1Leadiant GmbH (2017) Chenodeoxycholic Acid Leadiant Capsules SmPC. Available at: https://www.ema.europa.eu/en/documents/product-information/chenodeoxycholic-acid-leadiant-epar-product-information_en.pdf
