Fondazione Telethon wins FDA approval for first gene therapy for Wiskott-Aldrich

The U.S. FDA has approved Italian nonprofit Fondazione Telethon’s ex vivo gene therapy, branded Waskyra, for patients with Wiskott-Aldrich syndrome (WAS), a rare and life-threatening immunodeficiency.

The approval marks the first gene therapy for WAS and the first approved cell and gene therapy product from a nonprofit applicant.

Waskyra is indicated for pediatric patients six months and older and adults with WAS who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.

The approval was based on two open-label, single-arm, multinational clinical studies and an expanded access program totaling 27 patients with severe WAS, which demonstrate substantial and sustained clinical benefit for patients with severe WAS, with significant reductions in the primary disease manifestations that drive morbidity and mortality

WAS is a genetic disease caused by mutations in the WAS gene. The condition is characterized by bleeding, eczema, recurrent infections, and increased susceptibility to autoimmunity and lymphoreticular malignancies. Treatment options have been limited to symptomatic management and early in life allogeneic hematopoietic stem cell transplantation.

Waskyra consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector encoding the WAS gene. By restoring functional WAS protein expression in affected cells, Waskyra addresses the underlying cause of the disease.