FDA Approves First Gene Therapy for Wiskott–Aldrich Syndrome, Milestone for Non-Profit Research

Published December 19, 2025 The U.S. Food and Drug Administration (FDA) has approved etuvetidigene autotemcel (brand name Waskyra) as the first gene therapy for the treatment of Wiskott–Aldrich syndrome (WAS), a rare, life-threatening immunodeficiency disorder.

Developed by Fondazione Telethon ETS, Waskyra is indicated for pediatric patients aged 6 months and older and adults with a confirmed WAS gene mutation who are eligible for hematopoietic stem cell transplantation (HSCT) but lack a suitable related donor. The ex vivo cell-based therapy uses patients’ own hematopoietic stem cells genetically engineered to express a functional WAS gene, addressing the underlying cause of the disease.

The FDA’s decision marks a major advance for the rare disease community, representing both the first gene therapy approved for WAS and the first gene therapy approval in the U.S. led by a non-profit sponsor. Fondazione Telethon’s decade-plus research and collaboration with clinical partners culminated in this regulatory milestone, offering a potential therapeutic option for patients historically reliant on supportive care or higher-risk donor transplants.

Beyond its impact on Wiskott–Aldrich syndrome, the approval of Waskyra signals broader momentum for gene therapy development in rare diseases, particularly those affecting ultra-small patient populations. It demonstrates that sustained non-profit-driven research models, combined with regulatory engagement and academic-industry collaboration, can successfully translate into approved therapies. This milestone may help de-risk future gene therapy programs, encourage investment in rare and ultra-rare indications, and expand the pipeline of curative-intent treatments across the rare disease landscape.

Wiskott–Aldrich syndrome primarily affects males and is characterized by immunodeficiency, low platelet counts, severe infections, and bleeding. Traditional treatment options have been limited, underscoring the significance of this approval for affected families, clinicians, and the broader rare disease research community.

References:

1. FDA press announcement: FDA Approves First Gene Therapy Treatment for Wiskott–Aldrich Syndrome (U.S. FDA)

2. PR Newswire: Fondazione Telethon Announces FDA Approval of Waskyra™ (etuvetidigene autotemcel)

3. FDA approval context and non-profit milestone coverage (U.S. GovDelivery)

4. Drug details and clinical implications (Drugs.com summary)