The European Commission’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025. The therapy was also approved by the U.S. FDA for the U.S. market in December 2025.
Fondazione Telethon today announced the European Commission’s decision of 9 January 2026 to grant marketing authorisation for Waskyra™, an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS), a rare and severe genetic immunodeficiency.
Developed thanks to decades of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, the therapy represents a significant scientific and clinical milestone and a new hope for patients.
Wiskott-Aldrich syndrome causes severe immune deficiencies, recurrent infections, bleeding, eczema, and an increased risk of autoimmune diseases and cancer. Gene therapy offers an alternative for patients who cannot undergo hematopoietic stem cell transplantation due to the absence of a compatible family donor.
The European Commission’s decision follows the positive opinion issued in November 2025 by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), which recommended the granting of marketing authorisation. The same therapy was also approved for the U.S. market by the U.S. Food and Drug Administration (FDA) in December 2025, confirming the effectiveness and uniqueness of a research and development model that has become truly one-of-a-kind at international level.
Fondazione Telethon is the first non-profit organization to successfully complete the development of a gene therapy, taking it from laboratory research to regulatory approval in both the United States and the European Union.
“The European Commission’s decision represents the completion of a long and rigorous regulatory process, making this therapy officially approved in Europe, following the FDA’s approval in the United States last December. This result demonstrates the strength of a model built over time, capable of taking a discovery born in non-profit research all the way to approval by the highest international regulatory authorities. This milestone reinforces our commitment to translating research on rare genetic diseases into concrete therapeutic solutions, delivering meaningful clinical impact and sustainable treatment perspectives for patients and their families” said Ilaria Villa, CEO of Fondazione Telethon.
“The authorisation for marketing in Europe represents a concrete recognition of the scientific rigor, the quality of the clinical data, and the multidisciplinary work that made this achievement possible,” added Dr. Alessandro Aiuti, Deputy director clinical research at SR-Tiget, Chief of Pediatric Immunohematology at IRCCS Ospedale San Raffaele and Full Professor of Pediatrics at Università Vita-Salute San Raffaele. “It demonstrates that translational research, supported by a long-term vision, can generate therapies that sustainably redefine treatment standards for rare genetic diseases.”
