Fondazione Telethon announced that FDA has approved its Biologics License Application (BLA) for Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy, for the treatment of patients with Wiskott-Aldrich syndrome (WAS).

Waskyra consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells that are transduced with a lentiviral vector encoding the WAS gene. Once corrected, the stem cells are reinfused into the patient undergoing chemotherapy prior to treatment, to prepare the bone marrow to receive them.¹

The treatment has also displayed its ability to reduce the frequency of severe and moderate bleeding events along with serious infections in patients with WAS compared to the period prior to a patient’s treatment.

WAS is seen almost exclusively in males and affects both blood cells and cells of the immune system, and is caused by abnormalities in the gene that produces the WAS protein found in blood cells and certain immune cells.¹ Those diagnosed with WAS lack a functional WAS protein, resulting in their immune cells and blood cells not developing or functioning normally.

The approval of Waskyra makes Fondazione Telethon the first non-profit organization to have successfully led the full pathway of an ex vivo gene therapy from laboratory research to regulatory approval.¹ The company achieved this feat through its collaborations with industry partners bringing gene therapies from discovery to patients.

"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," said Ilaria Villa, CEO of Fondazione Telethon. "It confirms the value of a patient-centered model that turns research into real treatments, especially where the market fails to act."

What was FDA’s approval of Waskyra’s BLA based on?

FDA's approval of the BLA for Waskyra is based on positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for Waskyra just a few weeks earlier.

Back in November this year, CHMP based its positive opinion on findings gathered from a clinical development program of 27 patients diagnosed with WAS, where its group of experts for cell- and gene-based medicines deemed that benefits of Waskyra were greater than its potential risks in WAS patients who had no suitable donor available for a required haematopoietic stem cell transplantation.

The treatment was developed through decades of research at the San Raffaele Telethon Institute for Gene Therapy in Milan. Waskyra represents a major scientific and clinical achievement, offering new hope for patients affected by this condition.

"The approval of this gene therapy represents a decisive step forward and a tangible response to the needs of patients," commented 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. "Seeing years of scientific research and dedication translate into real therapeutic opportunities for people gives profound meaning to our work."

Sources

1. Fondazione Telethon Announces FDA Approval of Waskyra (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome Fondazione Telethon December 10, 2025 https://www.prnewswire.com/news-releases/fondazione-telethon-announces-fda-approval-of-waskyra-etuvetidigene-autotemcel-a-gene-therapy-for-the-treatment-of-wiskott-aldrich-syndrome-302637868.html
2. Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 10-13 November 2025 European Medicines Agency November 14, 2025 https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-10-13-november-2025