NEW YORK – Fondazione Telethon on Tuesday said that it has submitted a biologics license application for its Wiskott-Aldrich syndrome (WAS) gene therapy to the US Food and Drug Administration seeking marketing authorization in the US.
Rome-based Fondazione Telethon filed a marketing authorization application for the gene therapy, etuvetidigene autotemcel, with the European Medicines Agency in the EU last month.
"After the EMA submission, it was essential for us to also approach the US market in order to make WAS gene therapy available in another important area of the world," Fondazione Telethon CEO Ilaria Villa said in a statement. As a charity, Fondazione Telethon aims to commercialize rare disease drugs that might not otherwise be made available due to small market opportunity.
Etuvetidigene autotemcel was developed based on research at the San Raffaele-Telethon Institute for Gene Therapy in Milan. It was originally licensed to Orchard Therapeutics, where it was known as OTL-103, but the gene therapy was later acquired by Fondazione Telethon in 2023.
The gene therapy comprises autologous CD34-positive stem and progenitor cells that are modified with a lentiviral vector encoding the WAS gene, mutations in which cause Wiskott-Aldrich syndrome, a rare X-linked immunodeficiency. In Wiskott-Aldrich syndrome, a mutation in the WAS gene leads to immune system and blood clotting problems.
Thus 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.