Italian scientists along with SmithGlaxoKline, are likely to have gene therapy approved for use in Europe and many children with faulty genes will benefit. Strimvelis has been endorsed by the European Medicine Agency and will help those not obtaining a bone marrow match. Each year in Europe 15 children are born unable to produce white blood cells, and two years are all they can expect to live without a bone marrow transplant. Such is the degree of weakness that the child will often be confined to a bubble in order to remain totally germ-free.
It is expected that within a couple of months, this will become only the second therapy that Europe is happy to accept. The other – Glybera from UniQure treats adults rather than children. No gene therapies have yet been approved by the U.S. Food and Drug Administration, but Bluebird Bio is hopeful that their products will pass on to development and BristolMyersSquibb are looking at becoming involved.
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This research goes back a quarter of a century and since the death of a patient in the US in 1999 and some poor trial results, there have been setbacks. It is hoped that the future is brighter. Rare Diseases Unit at GlaxoSmithKlines’ head Martin Andrews as high hopes for the future. He accepts there are challenges as the bone marrow has to be taken, altered and then returned.
Pricing is going to be problematic as the market is limited and while GSK will not comment on the cost, once marketed, Strimvelis would be priced well below $1 million.