Fondazione Telethon announced that it has submitted the Biologics License Application (BLA) 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 Food and Drug Administration (FDA).
The announcement follows closely on the heels of the submission of the same 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 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-TIGET, Milan), a leader in the field of advanced therapies.
“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. The FDA registration can therefore offer a wider opportunity to meet the need of more access to the therapy for WAS affected patients. We are hopeful that this step can be completed in a timely fashion”, said Foundation CEO Ilaria Villa.
About Wiskott-Aldrich Syndrome (WAS)
WAS is a rare immunodeficiency which manifests itself from early childhood with recurrent and relapsing infections, bleeding, eczema, increased risk of developing autoimmune diseases and lymphomas. It almost exclusively affects males, with an incidence of 1/250,000 male live births.
The current treatment options of affected children consist of supportive treatments managing and preventing clinical manifestations. The potentially curative treatment is a hematopoietic stem cell transplant, which is, however, only feasible in the presence of an adeguate donor and still carries potential complications.
About etuvetidigene autotemcel
In cases where transplantation from a matched family donor is not possible, gene therapy can be a viable treatment option, with excellent results in both safety and efficacy.
Etuvetidigene autotemcel consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells transduced by a lentiviral vector encoding for the WAS gene.
To date, a total of 30 patients with WAS have been treated, 27 within the clinical development programme and 3 within an early access scheme which makes as of today, etuvetidigene autotemcel accessible in Italy according to the AIFA determination of 2 August 2023, which, pursuant to Law 648/96, indicates it for the treatment of patients with WAS from 6 months onwards without a HLA matched-related donor.
