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

On December 10, 2025, Fondazione Telethon proudly announced that the U.S. Food and Drug Administration (FDA) has granted Biologics License Application (BLA) approval for Waskyra™ (etuvetidigene autotemcel). This ex vivo gene therapy marks a remarkable advancement in the treatment options available for Wiskott-Aldrich Syndrome (WAS), a rare but severe immunodeficiency that predominantly affects males. The approval is a celebration of decades of research, primarily conducted at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

Wiskott-Aldrich Syndrome is characterized by its significant health challenges, including recurrent infections, eczema, and low platelet counts. Affecting approximately 1 in every 250,000 male births, WAS results from mutations in the WAS gene, which leads to a deficiency in the WAS protein crucial for the normal functioning of blood and immune cells. This deficiency compromises the body's ability to mount effective immune responses and leads to serious health complications.

The newly approved therapy, Waskyra, offers new hope for affected patients who have struggled with limited treatment options. Prior to this innovation, the primary curative method available was hematopoietic stem cell transplantation, which required a compatible donor and came with considerable risks. Waskyra provides an alternative pathway; the therapy utilizes the patient's own hematopoietic stem and progenitor cells, which are genetically corrected to produce healthy WAS protein and then re-administered into the patient.

The approval by the FDA coincides with a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use, demonstrating a collaborative and comprehensive approach to regulatory processes surrounding gene therapies.

Ilaria Villa, CEO of Fondazione Telethon, expressed her excitement regarding the approval, noting that this achievement is a significant milestone not only for Italian research but also for the global rare disease community. She emphasized the transformative impact that this patient-centered model can have, converting extensive research into actionable treatments that improve lives.

Dr. Alessandro Aiuti, deputy director of clinical research at SR-Tiget and an authority on pediatric immunohematology, echoed these sentiments. He highlighted how years of dedication in scientific research have culminated in real therapeutic advancements that address the pressing needs of patients.

The clinical trials that led to the approval of Waskyra were conducted at the esteemed IRCCS Ospedale San Raffaele, which specializes in gene therapy for WAS. Through rigorous testing, Waskyra has been shown to reduce the frequency of severe bleeding events and serious infections in patients compared to periods prior to treatment. This advancement is particularly critical for patients lacking matchable living donors for transplantation.

The FDA approval not only reinforces Fondazione Telethon's commitment to excellence in medical research but also positions the organization as an international leader in the innovative treatment of rare genetic diseases. The organization has set a precedence as the first non-profit to independently lead an ex vivo gene therapy from research through to regulatory approval, marking a new era in the treatment of rare diseases where options have previously been scarce.

In summary, the approval of Waskyra™ for Wiskott-Aldrich Syndrome represents a new chapter in the lives of many patients and their families, offering hope and the possibility of a healthier future. As this therapy moves forward, it brings with it the promise of transforming the landscape of rare disease treatment, showcasing the potential of research-driven healthcare solutions.