NEW YORK – Fondazione Telethon is seeking EU marketing authorization for etuvetidigene autotemcel as a gene therapy to treat Wiskott-Aldrich syndrome, a rare X-linked immunodeficiency.
The Italian charity earlier this week said it has submitted a regulatory application to the European Medicines Agency seeking marketing authorization for the gene therapy, which comprises autologous CD34-positive stem and progenitor cells that are modified with a lentiviral vector encoding the WAS gene. Wiskott-Aldrich syndrome is caused by a mutation in the WAS gene, which leads to immune system and blood clotting problems.
So far, researchers have data on the gene therapy's activity from 30 patients who have received it, 27 within a clinical trial and three through an early-access scheme in Italy.
The gene therapy was developed by researchers at San Raffaele-Telethon Institute for Gene Therapy, who aimed to show that the treatment could be an option for patients who don't have a matched family donor for hematopoietic stem cell transplantation. Etuvetidigene autotemcel was known as OTL-103 when it was licensed to Orchard Therapeutics. However, when Orchard decided to seek strategic alternatives for its primary immunodeficiency programs in 2022, including this gene therapy, Fondazione Telethon reacquired it.
In a statement, Ilaria Villa, the director general for Fondazione Telethon, said the organization is also planning to seek approval for etuvetidigene autotemcel in this setting in the US.