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LONDON (Alliance News) - GlaxoSmithKline PLC, along with Fondazione Telethon and Ospedale San Raffaele, Wednesday released safety and efficacy data from their long term analysis of children being treated with stem cell gene therapy for ADA-SCID, a rare genetic disorder.
The data is from the analysis 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.
The key efficacy endpoint was survival; all eighteen patients were alive after a median follow-up of 6.9 years.
Additionally, intervention free survival and infection rates were also measured, and fifteen of the eighteen did not require interventions. The absolute number of severe infections after the therapy was reduced when compared to a pre-treatment period.
At the time of reporting twelve out of fourteen patients surveyed were attending pre-school or school as appropriate for their age, Glaxo said.
"We are delighted to publish this formal update on the long-term safety and efficacy of Strimvelis," said Jonathan Appleby, medicines development leader at Glaxo.
Shares in GlaxoSmithKline were up 0.5% at 1,452.00 pence Wednesday morning.
By Hana Stewart-Smith; hanassmith@alliancenews.com; @HanaSSAllNews
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