Fondazione Telethon announced that it has submitted the Marketing Authorization Application (MAA) for the gene therapy – etuvetidigene autotemcel – for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the European Medicines Agency (EMA).
If approved, etuvetidigene autotemcel will provide a valid treatment option, with excellent results in terms of both safety and efficacy, to WAS patients for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
This milestone confirms Fondazione Telethon’s commitment to making gene therapies available to patients and builds upon the taking over of the EU and UK marketing authorizations for the gene therapy for the treatment of ADA-SCID, another rare immunodeficiency.
Read the full press release here: https://www.telethon.it/en/stories-and-news/news/from-telethon-foundation/fondazione-telethon-submits-eu-marketing-authorization-application-for-etuvetidigene-autotemcel-gene-therapy-for-the-treatment-of-wiskott-aldrich-syndrome/