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Tech
News18 » Tech
1-min read

GSK Gets EU Nod for World's First Gene Therapy for Children

Called Strimvelis, it is designed for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID).

Reuters

Updated:May 29, 2016, 8:05 AM IST
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GSK Gets EU Nod for World's First Gene Therapy for Children
The GlaxoSmithKline building is pictured in Hounslow, west London, Britain, June 18, 2013. REUTERS/Luke MacGregor/File Photo
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London: The world's first life-saving gene therapy for children, developed by GlaxoSmithKline and Italian scientists, was approved for sale in Europe, in a step forward for the pioneering technology to fix faulty genes.

Called Strimvelis, it is designed for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID). SCID is sometimes known as “bubble baby” disease, since those born with it have immune systems so weak they must live in germ-free environments.

Strimvelis is the second gene therapy to be approved in Europe, after UniQure's Glybera, which treats a rare adult blood disorder.

Glybera made history in 2014 as the first drug to carry a $1 million price tag. GSK has not so far revealed how much its product will cost but a source close to the company said last month it would be “very significantly less than $1 million”.

The EU approval for GSK's treatment had been expected, following a positive opinion from the European Medicines Agency last month.

The US Food and Drug Administration has yet to approve any gene therapies but a growing number of US biotech companies have products in development.

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