On 13 November in Milan, EMA’s expert committee backs Italy’s first home‑grown gene therapy for people in Europe living with Wiskott–Aldrich Syndrome.

First Italian-made gene therapy for Wiskott-Aldrich Syndrome

European Medicines Agency (EMA)

On 13 November, the European Medicines Agency (EMA) announced the Committee for Medicinal Products for Human Use (CHMP) positive opinion recommending marketing authorisation for Etuvetidigene autotemcel (Waskyra®). This is an ex vivo hematopoietic stem cell gene therapy intended to treat adults and children aged older than 6 months with Wiskott-Aldrich syndrome (WAS), a rare and life-threatening immunodeficiency. The therapy reduces the frequency of bleeding events and serious infections in patients, especially in cases where transplantation from a compatible family donor is not possible.

Waskyra® is the result of decades of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and of a clinical development program conducted at IRCCS Ospedale San Raffaele in Milan, Italy. Data obtained from this program demonstrated a significant reduction in the annualised rate of severe health events following the treatment.

Waskyra® was supported through an “EMA pilot” program, which offers enhanced support to academic and non-profit developers of ATMPs addressing unmet medical needs.

This milestone highlights the growing impact of ATMPs in changing the lives of people with WAS in the EU, providing them with new hope.

Importantly, the applicant of the marketing authorisation is Fondazione Telethon ETS, the first non-profit marketing authorization holder in the European Union. In fact, in 2023, they obtained the marketing authorization holder of another advanced therapy for an ultra-rare disease, adenosine deaminase severe combined immunodeficiency (ADA-SCID).

Next steps include the European Commission final marketing authorisation to make it available for EU WAS patients. The gene therapy is also under review for authorisation to market by the U.S. Food and Drug Administration (FDA). You can read more information by following these links:

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