NICE final draft backs gene therapy for rare 'bubble baby syndrome'

The UK medicines cost-effectiveness watchdog the National Institute for Health and Care Excellence (NICE) today published final draft guidance recommending gene therapy Strimvelis, from UK pharma major GlaxoSmithKline (LSE: GSK), as an option for treating an extremely rare immune deficiency condition, confirming an October preliminary opinion.

Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is an inherited genetic condition. It affects the body’s white blood cells, leaving people with the condition without a properly functioning immune system and therefore with a high risk of developing life-threatening, recurrent infections. Around three babies a year in England are born with ADA-SCID. If left untreated, infants die before school age.

Strimvelis is only the second gene therapy for an inherited disease to be licensed anywhere in the world. The current treatment for the condition is a stem cell transplant, which can restore the immune system if successful. However, closely matched stem cell donors are hard to find or may not be available. Also, stem cell transplants may not be successful in all cases and they carry a risk of mortality and graft versus host disease. The draft guidance recommends Strimvelis when no suitable matched related stem cell donor is available.