was developed at San Raffaele Telethon Institute for Gene Therapy and developed by GlaxoSmithKline (GSK) through a 2010 collaboration with Fondazione Telethon and Ospedale San Raffaele. 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 (EMA) 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. As of 2016, the only site approved to manufacture the treatment was MolMed. In 2017, GSK announced it was looking to sell off Strimvelis, and in March 2018, GSK sold Strimvelis to Orchard Therapeutics Ltd.; as of that time there had been only five sales of the product. Society and culture The condition affects about 14 people per year in Europe and 12 in the U.S. Economics The price for the treatment was set at , twice the annual cost of enzyme replacement therapy injections. Enzyme replacement therapy for ADA requires weekly injections and costs about for one patient over ten years. Names Strimvelis is the brand name. The common name is autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence. References Further reading Applied genetics Biotechnology Emerging technologies Gene therapy Medical genetics Medical treatments Orphan drugs Stem cells