FDA Approves Gene Therapy Waskyra for Wiskott-Aldrich Syndrome

Fondazione Telethon announced FDA approval of Waskyra™ (etuvetidigene autotemcel), marking a significant milestone for the treatment of Wiskott-Aldrich Syndrome (WAS). WAS primarily affects males and is a rare immunodeficiency disorder. The approved therapy, developed over decades at the San Raffaele Telethon Institute for Gene Therapy in Milan, offers a new therapeutic option for patients lacking a suitable stem cell donor.

The approval follows a favorable opinion from the European Medicines Agency, underlining Waskyra's potential to address severe bleeding and infection issues in WAS patients. Fondazione Telethon highlights this development as evidence of their commitment to transforming research into viable treatments for rare diseases.

Waskyra uses genetically modified autologous stem cells, ensuring reduced infection rates and improved patient outcomes. Current treatment alternatives include risky hematopoietic stem cell transplantation, highlighting Waskyra's importance in offering a safer gene therapy option when donor matches are unavailable.