GSK has received good news from NICE as it is revealed that Strimvelis, its gene therapy treatment for adenosine deaminase deficiency (ADA-SCID) or ‘bubble boy’ syndrome, has been given a positive recommendation by NICE despite the extremely high cost associated with it.  

The therapy carries a heavy price tag of €594,000 per administration, making it one of the most expensive drugs in the world, though only one treatment is required and the benefits are thought to be life-long. On this basis, it was judged that the drug is actually cost-effective.

ADA-SCID is a serious immune condition under which sufferers are at high risk of recurrent infections and severe symptoms if they are not isolated, capable of killing children before they reach school age.

"Strimvelis represents an important development in the treatment of ADA-SCID, offering the potential to cure the immune aspects of the condition and avoid some of the disadvantages of current treatments," explained Carole Longson, Director of NICE's Centre for Health Technology Assessment. "This means that children born with ADA-SCID will now have a better chance of being able to lead as near normal a life as possible." 

GSK expects that the treatment will be given to only one person per year, though this rate may vary.