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FDA Approval of Waskyra™: A New Era for Wiskott-Aldrich Syndrome

On December 10, 2025, Fondazione Telethon celebrated a significant milestone with the announcement of the U.S. Food and Drug Administration's (FDA) approval of Waskyra™ (etuvetidigene autotemcel). This groundbreaking gene therapy is designed for the treatment of Wiskott-Aldrich Syndrome (WAS), a rare immunodeficiency disorder that primarily affects males.

Wiskott-Aldrich Syndrome, which affects approximately 1 in 250,000 live male births, is characterized by a triad of symptoms: eczema, increased susceptibility to infections, and low platelet counts. Patients face a lifelong battle against these severe health challenges, making Waskyra's approval a beacon of hope for many.

Transformational Treatment

Waskyra is an innovative ex vivo gene therapy that works by correcting the underlying genetic defect associated with WAS. Developed by the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan over decades of dedicated research, this therapy involves collecting a patient’s CD34+ stem cells. These cells are then modified using a lentiviral vector to express the corrected WAS gene before being reinfused into the patient after a preparatory chemotherapy session.

The clinical data demonstrated that Waskyra significantly decreases both severe and moderate bleeding episodes and serious infections in patients with WAS. This is especially crucial given that patients often lack viable options for treatment, as hematopoietic stem cell transplantation is not always feasible due to the challenges of finding a compatible donor.

Expert Endorsements

“The FDA's approval of Waskyra represents a groundbreaking achievement,” remarked Ilaria Villa, CEO of Fondazione Telethon. “It signifies not only a triumph for Italian research but also a major step forward for the global community focusing on rare diseases. This approval exemplifies our commitment to translating research into tangible treatments for patients who need them the most.”

Dr. Alessandro Aiuti, a prominent researcher in the field, echoed these sentiments, stating, “Seeing years of scientific inquiry culminate in effective therapeutic options for patients is profoundly rewarding. The approval of Waskyra is not just a regulatory success; it’s a testament to our unwavering commitment to improving patient outcomes.”

The Path Ahead

The FDA's recognition of Waskyra follows a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use, highlighting the therapy’s anticipated impact in Europe as well. Fondazione Telethon's achievement as the first non-profit to navigate a gene therapy from conception to regulatory approval showcases its leadership in advancing research on rare genetic conditions.

With this approval, Fondazione Telethon fortifies its pivotal role in the realm of gene therapies and sets a precedent for future innovations in treating rare genetic diseases. It expands opportunities for pediatric and adult patients suffering from Wiskott-Aldrich Syndrome, offering them a fighting chance at a healthier life.

About Waskyra™ and WAS

Waskyra is aimed at pediatric patients aged six months and older and adults who possess a mutation in the WAS gene. For those suited for hematopoietic stem cell transplantation but lacking a suitable donor, Waskyra provides an alternative that could lead to significant health improvements.

During clinical trials, no adverse reactions attributed directly to Waskyra were observed, although preparatory treatments carried their own risks. Participants were encouraged to report any negative side effects to the FDA, reinforcing the importance of ongoing safety monitoring in innovative therapies.

A Commitment to Rare Diseases

For over three decades, Fondazione Telethon has been at the forefront of research into rare genetic disorders, devoted to funding high-impact scientific work that leads to new treatments and improved quality of life for those affected. With the approval of Waskyra, the future looks brighter for patients battling Wiskott-Aldrich Syndrome.

For more information on Waskyra and its clinical implications, stakeholders and patients can refer to the comprehensive prescribing information available through the FDA’s website.