[1] ADA-SCID is a rare inherited condition in which there is a change (mutation) in the gene needed to make an enzyme called adenosine deaminase (ADA).
[1] Because ADA is essential for maintaining healthy lymphocytes (white blood cells that fight off infections), the immune system of people with ADA-SCID does not work properly and without effective treatment they rarely survive more than two years.
[2] Strimvelis is indicated for the treatment of people with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
[8][9] Strimvelis, the first ex vivo autologous gene therapy approved by the EMA, has demonstrated remarkable efficacy and safety in clinical trials for the treatment of ADA-SCID.
[10] 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.
[16] The condition affects about 14 people per year in Europe and 12 in the U.S.[19] The price for the treatment was set at €594,000, twice the annual cost of enzyme replacement therapy injections.