Skip to main content
Premium Trial:

Request an Annual Quote

SCID Treatment Funded in UK

The UK's National Institute for Health and Care Excellence (NICE) has approved GlaxoSmithKline's gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), Reuters reports.

For GSK's Strimvelis, patients' CD34+ cells are reprogrammed to express ADA to treat the condition. Patients with ADA-SCID are kept in isolation to protect them from infections. The European Commission approved the therapy, which is to be a one-time treatment, in 2016.

Reuters notes that the UK's NICE approved the therapy despite its cost of nearly €600,000 (US$700,000). As the Times reports, NICE has placed a cap on what it would pay for treatments for rare diseases, saying that it wouldn't pay for therapies that topped £100,000 for a year of good-quality life. But the agency estimates that the treatment gives 14 years of good-quality life, bringing its cost under that limit.

 "It's an extremely exciting time for gene therapy," Bobby Gaspar, a researcher at Great Ormond Street, tells the Times

"We are getting to the point where we have a number of standard genetic medicines and that will only encourage the development of others," he adds.