The US FDA has approved the first autologous hematopoietic stem cell-based gene therapy, etuvetidigene autotemcel, for the treatment of adults and pediatric patients aged six months and older with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene. This therapy is indicated for patients for whom hematopoietic stem cell transplantation (HSCT) is appropriate and who do not have a suitable human leukocyte antigen (HLA)–matched related stem cell donor. Clinical evidence The safety and efficacy of etuvetidigene autotemcel were evaluated in two clinical trials involving 27 patients.
Across these trials, treatment reduced the rate of severe infections by 93% within six to 18 months following gene therapy, compared with the 12-month pre-treatment period. In addition, the therapy led to a 60% reduction in moderate and severe bleeding events during the first 12 months after…
