Posted by Michael Wonder on 04 Feb 2025
Fondazione Telethon submits EU marketing authorisation application for etuvetidigene autotemcel gene therapy for the treatment of Wiskott-Aldrich syndrome
3 February 2025 - Fondazione Telethon announced that it has submitted the marketing authorisation application for the gene therapy - etuvetidigene autotemcel - for the treatment of patients with Wiskott-Aldrich syndrome, a rare genetic disease of the immune system to the EMA.
After positioning itself as the first charity in the world to assume responsibility for the production and distribution of a drug – the gene therapy for the treatment of adenosine deaminase, this submission to the EMA represents an extraordinary new milestone that confirms the Foundation's commitment to making therapies discovered through the work of its researchers available to patients who need them.
