FDA Approval of Waskyra for Wiskott-Aldrich Syndrome

Waskyra (etuvetidigene autotemcel) is the first FDA‑approved cell‑based gene therapy for Wiskott‑Aldrich syndrome (WAS), offering a disease‑modifying option that immediately expands treatment for eligible patients with this rare immuno‑hematologic disorder.

Care previously relied on supportive measures and hematopoietic stem cell transplantation, constrained by donor availability, graft complications, and transplant morbidity. Waskyra uses a patient’s own hematopoietic stem cells that are genetically corrected to express functional WAS protein and are returned after reduced‑intensity conditioning—targeting the underlying defect and supporting potential durable clinical benefit.

This therapy option was studied in two open‑label, single‑arm multinational trials plus an expanded‑access program (27 patients total). Primary assessments of severe infections and bleeding showed a 93% reduction in severe infections in the six to 18 months after treatment compared with the 12 months before treatment, and a 60% reduction in moderate‑to‑severe bleeding events at 12 months post‑treatment. At four years after infusion most patients reported no moderate‑to‑severe bleeding.