Children's Health

GSK gets EU approval for first gene therapy for children

The GlaxoSmithKline building in Hounslow, west London

The GlaxoSmithKline building in Hounslow, west London  (Copyright Reuters 2016)

The world's first life-saving gene therapy for children, developed by GlaxoSmithKline and Italian scientists, was approved for sale in Europe on Friday, 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.

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