Fondazione Telethon announced that the US Food and Drug Administration (FDA) has approved the Biologics Licence Application (BLA) for Waskyra, an ex-vivo gene therapy for patients with Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening immunodeficiency.

The FDA's approval of the BLA of Waskyra follows a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use for the same product just a few weeks earlier.

Developed through decades of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Waskyra represents a major scientific and clinical achievement, offering new hope for patients affected by this condition.

"The FDA's approval of Waskyra is an extraordinary achievement – not only for Italian research and for Fondazione Telethon, but for the global rare disease community. It confirms the value of a patient-centered model that turns research into real treatments, especially where the market fails to act," said Ilaria Villa, CEO, Fondazione Telethon.

The clinical trial phase was conducted at IRCCS Ospedale San Raffaele, a Centre of Excellence (CoE) in gene therapy for WAS and other diseases.

"The approval of this gene therapy represents a decisive step forward and a tangible response to the needs of patients. Seeing years of scientific research and dedication translate into real therapeutic opportunities for people gives profound meaning to our work," commented Dr Alessandro Aiuti, Deputy Director—Clinical Research, SR-Tiget, Chief of Paediatric Immunohematology at IRCCS Ospedale San Raffaele and Full Professor of Pediatrics at Università Vita-Salute San Raffaele.