GlaxoSmithKline’s Strimvelis receives provisional NICE recommendation

GlaxoSmithKline's rare disease therapy Strimvelis has been recommended for routine NHS use in new draft guidance from the National Institute for Health and Care Excellence (NICE).

The regulator has offered a provisional approval for the drug to be used in the treatment of children with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an ultra-rare inherited immunodeficiency condition.

This marks the first time that NICE has applied its new higher cost-effectiveness limits for treatments for very rare conditions, with Strimvelis recommended when no suitable matched related stem cell donor is available.

ADA-SCID are extremely vulnerable to infection and usually live in isolation to minimise the risk. It affects only around three babies a year in England, but if left untreated, affected infants usually die before they reach school age.

Stem cell transplants are the current standard of care, but these carry risks, as well as being complicated by the difficulty of finding a good match. It is hoped that Strimvelis could help children to attend school and socialise without constant fear of catching an infection.

Professor Carole Longson, director of the centre for health technology assessment at NICE, said: "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."

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