EC authorization for Waskyra for Wiskott-Aldrich syndrome

Italian non-profit organization Fondazione Telethon today announced the European Commission’s decision of January 9, to grant marketing authorization for Waskyra (etuvetidigene autotemcel), 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.