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Strimvelis


Strimvelis is 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), a rare disorder caused by the absence of an essential protein called adenosine deaminase (ADA), which is required for the production of lymphocytes. Children born with ADA-SCID do not develop a healthy immune system so cannot fight off everyday infections, which results in severe and life-threatening illness. Without prompt treatment, the disorder often proves fatal within the child’s first year of life. ADA-SCID is estimated to occur in approximately 15 patients per year in Europe.

The treatment was developed at San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and developed by GlaxoSmithKline (GSK) through a 2010 collaboration with Fondazione Telethon and Ospedale San Raffaele (OSR). GSK, working with the biotechnology company MolMed S.p.A, developed a manufacturing process that was previously only suitable for clinical trials into one demonstrated to be robust and suitable for commercial supply.

In April 2016, a committee at the European Medicines Agency recommended marketing approval for its use in children with adenosine deaminase deficiency, for whom no matched HSC donor is available, on the basis of a clinical trial that produced a 100% survival rate; the median follow-up time was 7 years after the treatment was administered. 75% of people who received the treatment needed no further enzyme replacement therapy. Efforts had begun 14 years before. The total number of children treated was reported as 22 and 18. Around 80% of patients have no matched donor. Strimvelis was approved by the European Commission on 27 May 2016.


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