NICE approves gene therapy for rare 'bubble baby syndrome'

The UK medicines cost-effectiveness watchdog the National Institute for Health and Care Excellence (NICE) has today published draft guidance recommending the first ex-vivo stem cell gene therapy Strimvelis, from pharma giant GlaxoSmithKline (LSE: GSK), as an option for treating, an ultra-rare immune deficiency condition.

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. Their quality of life is affected by developmental delay, chronic diarrhoea, failure to thrive and recurrent infections. People with ADA-SCID who aren’t receiving treatment need to be kept in isolation to avoid infections – hence the colloquial term for the condition: ‘bubble baby syndrome’