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EC approves GSK's Strimvelis

May 28, 2016 1:05 AM UTC

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) said the European Commission approved gene therapy Strimvelis ( GSK2696273) to treat patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) for whom no suitable HLA-matched related stem cell donor is available. GSK said the approval is the first granted to a corrective gene therapy for children.

Strimvelis is a retroviral vector delivering ADA for ex vivo hematopoietic stem cell therapy. The EC based its approval on data from 18 patients, including 12 participants in a pivotal Phase I/II study. GSK said all 18 patients remain alive after an average follow-up period of seven years. GSK said ADA-SCID affects 15 patients annually in Europe (see BioCentury Extra, May 5, 2015). ...