GSK's Strimvelis approved in Europe to treat very rare disease, ADA-SCID

31 May 2016

The European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency), from UK pharma major GlaxoSmithKline (LSE: GSK).

A child born with ADA-SCID does not have a healthy, fully-functioning immune system and as a consequence, is unable to fight off everyday infections. Strimvelis (autologous CD34+ cells transduced to express ADA) is the first corrective gene therapy for children to be awarded regulatory approval anywhere in the world. It is indicated for the treatment of patients with ADA-SCID for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.

ADA-SCID affects an estimated 15 children per year in Europe and following today’s approval, patients with the condition who are referred for treatment will be able to receive the gene therapy at Ospedale San Raffaele in Milan, Italy.

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