Fondazione Telethon, an Italy-based non-profit biomedical organisation, announced on Friday that it has received positive opinion from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP), recommending marketing authorisation for Waskyra in the European Union.

Waskyra is an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency. The disease manifests from early childhood with recurrent and persistent infections, bleeding episodes, eczema, and an increased risk of developing autoimmune diseases and lymphomas.

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

According to Fondazione Telethon, it is the first non-profit organisation to have successfully led the full pathway from laboratory research to regulatory approval, collaborating with industry partners when available to bring gene therapies from discovery to patients.

The therapy will be made available to patients at IRCCS Ospedale San Raffaele, a recognised centre of excellence in gene therapy for WAS and other diseases, where the clinical trial phase was conducted. The BLA for the same gene therapy for WAS is also under review by the US Food and Drug Administration (FDA).