Fondazione Telethon submits BLA for etuvetidigene autotemcel

Italian biomedical charity Fondazione Telethon, which is focused on rare genetic diseases, revealed it has submitted a Biologics License Application (BLA) for the gene therapy - etuvetidigene autotemcel – as a treatment of patients with Wiskott-Aldrich syndrome (WAS), a rare genetic disease of the immune system, to the US Food and Drug Administration (FDA).

The announcement follows closely on the heels of the submission of the marketing authorization request to the European Medicine Agency (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 (ADA-SCID)], these submissions for another advanced therapeutic medicinal product to both agencies confirm the Foundation's commitment to managing the production and distribution of medicinal products when they would not otherwise be made available by the pharmaceutical industry.

As the treatment for ADA-SCID, also etuvetidigene autotemcel gene therapy originated from research carried out by the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGER, Milan), a leader in the field of advanced therapies.