Abstract
The Committee for Human Medicinal Products has recently recommended marketing authorization for retrovirus-mediated ex vivo gene therapy of patients' autologous CD34+ cells transduced with human ADA complementary DNA for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).1 If confirmed by the European Commission, the product, called Strimvelis, would be the third approved gene therapy in the European Union. According to the CHMP opinion, “Strimvelis is indicated for the treatment of patients with severe combined immune deficiency due to ADA deficiency (ADA-SCID), for whom no suitable matched related stem cell donor is available.” The positive opinion includes a request to establish a post-treatment registry and long-term follow-up of the patients.
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