On December 9, 2025, the FDA announced the approval of Waskyra (etuvetidigene autotemcel), making it the first cell-based gene therapy authorized for the treatment of Wiskott-Aldrich syndrome (WAS).

The indication covers pediatric patients six months of age and older, as well as adults with a mutation in the WAS gene for whom hematopoietic stem cell transplantation (HSCT) is clinically appropriate, provided there is no HLA-matched related family donor.

The FDA described this decision as a transformative milestone because, for the first time, an approved therapy is available that uses the patient's own genetically corrected stem cells to address the underlying cause of the disease.

WHOM DOES IT IMPACT?

The direct impact is felt by pediatric and adult patients with severe WAS, clinical teams, and the advanced therapy ecosystem.

CONCLUSION:

The approval of Waskyra represents a high-impact milestone because it offers a transformative therapeutic option for a group of patients with WAS who previously had no optimal curative alternative, and it sets a relevant precedent for future gene therapies in rare diseases.