Posted by Michael Wonder on 10 Dec 2025
FDA approves first gene therapy treatment for Wiskott-Aldrich syndrome
9 December 2025 - Agency exercises regulatory flexibility to address unmet need for rare, life-threatening disease.
The US FDA today approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
Waskyra is indicated for pediatric patients six months and older and adults with WAS who have a mutation in the WAS gene and for whom haematopoietic stem cell transplantation is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
