Strimvelis, a product developed by the pharmaceutical company GlaxoSmithKline in partnership with the Italian Telethon and San Raffaele hospital in Milan, has been granted its Market Authorization (MA) by the European Medicines Agency (EMEA).
This product is used for a rare immune deficit in newborns: the adenosine deaminase severe combined immunodeficiency (ADA-SCID). This pathology affects nearly 15 children a year in Europe and accounts for 10% to 15% of all cases severe combined immunodeficiencies.
Strimvelis is a gene therapy treatment enabling the expression of the missing protein, adenosine deaminase, thanks to an autologous transplant of CD34+ modified stem cells.
All 12 children included in the pivotal study are still alive, three years after taking the treatment. In 2002, the first works which enabled to develop the treatment had been funded by the AFM-Téléthon as part of a joint call for proposals with the Italian Telethon. This MA is a first for a remedial gene therapy and is the outcome of many long years of research and clinical experimentation by researchers from the Italian TIGET (Telethon Institute for Gene Therapy), a laboratory created thanks to the Italian Telethon. It’s a new victory for gene therapy !