LONDON (Alliance News) - GlaxoSmithKline PLC, along with Fondazione Telethon and Ospedale San Raffaele, said Friday that gene therapy Strimvelis has been granted marketing authorisation in the European Union for the treatment of rare disease ADA-SCID.
This followed the release of long-term data of the treatment earlier this week of eighteen children treated with the hematopoietic stem cell gene therapy between 2000 and 2010 at the San Raffaele Telethon Institute for Gene Therapy.
ADA-SCID - or adenosine deaminase deficiency - is a very rare inherited gene disorder, in which a child does not develop a healthy immune system that often proves fatal in the child's first year of life.
"Today's approval is the result of many years' work with our collaborators in Milan and is the next step towards bringing life-changing treatment to patients with ADA-SCID and their families. This is the start of a new chapter in the treatment of rare genetic diseases and we hope that this therapeutic approach could also be used to help patients with other rare diseases in the future," said Head of Glaxo's Rare Disease Unit Martin Andrews in a statement.
Shares in GlaxoSmithKline were down 0.3% at 1,453.00 pence Friday.
By Hana Stewart-Smith; hanassmith@alliancenews.com; @HanaSSAllNews
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