5th May 2015 09:20
LONDON (Alliance News) - GlaxoSmithKline PLC, Italian biomedical charity Fondazione Telethon, and San Raffaele University and research hospital said Tuesday they have submitted a marketing application to the European Medicines Agency for gene therapy GSK2696273.
The submission is for the use of GSK2696273 for the treatment of adenosine deaminase severe combined immunodeficiency syndrome in patients for whom no suitable human leukocyte antigen matched related stem cell donor is available. This is a rare disease in which patients cannot make a certain type of white blood cell, and as a result have a severely deficient immune system.
The application follows on from a strategic alliance formed by the three in 2010.
"I am very pleased that we are now at a stage to file for approval of this gene therapy for ADA-SCID ? a devastating rare disease which drastically limits and shortens patients? lives," said President of Pharmaceuticals Research and Development at Glaxo Patrick Vallance in a statement.
"We believe this marks a significant milestone, showing the potential of gene therapy as an important additional modality for tackling the underlying cause of serious diseases," Vallance added.
Shares in GlaxoSmithKline are trading up 2.0% at 1,541.00 pence Tuesday morning.
By Hana Stewart-Smith; [email protected]; @HanaSSAllNews
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