WASHINGTON (dpa-AFX) - The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of a rare, genetic immune disorder Wiskott-Aldrich syndrome, or WAS.
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 is appropriate and no suitable human leukocyte antigen-matched related stem cell donor is available.
'Today's approval is a transformative milestone for patients with Wiskott-Aldrich syndrome, offering the first FDA-approved gene therapy that uses the patient's own genetically corrected hematopoietic stem cells to treat the disease,' said Vinay Prasad, Chief Medical and Scientific Officer and Director of the FDA's Center for Biologics Evaluation and Research. 'The FDA continues to exercise flexibility in the regulatory approach for rare diseases by considering all available data sources, including as appropriate data from expanded access programs, to facilitate the advancement of life-changing treatments while ensuring scientific requirements are satisfied.'
WAS is a rare, life-threatening 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. Until today, treatment options for patients with WAS have been limited to symptomatic management and allogeneic hematopoietic stem cell transplantation, with the latter being most effective when performed early in life and feasible only when matched donors are available.
Waskyra consists of the patient's own blood stem cells, which have been genetically modified to include functional copies of the WAS gene. Following reduced-intensity conditioning, the gene-corrected cells are infused intravenously to restore blood cell production. Waskyra restores functional WAS protein expression in affected cells, addressing the underlying cause of the disease.
The safety and effectiveness of Waskyra was assessed based on multinational clinical studies on 27 patients with severe WAS. It returned positive results, with significant reductions in the primary disease manifestations that drive morbidity and mortality.
Copyright(c) 2025 RTTNews.com. All Rights Reserved
Copyright RTT News/dpa-AFX
© 2025 AFX News