In a statement by the FDA, the U.S. Food and Drug Administration has authorized Waskyra (etuvetidigene autotemcel), the first gene therapy designed to treat Wiskott-Aldrich syndrome (WAS), a rare and life-threatening immune disorder. This approval represents a major advancement for patients who previously had limited options.
Who Can Receive It:
The therapy is intended for children over six months and adults with confirmed WAS gene mutations who are eligible for stem cell transplantation but lack a matched donor.
How It Works:
Waskyra uses a patient’s own blood stem cells, which are genetically corrected to restore normal WAS protein function. After conditioning, these modified cells are infused back into the body, targeting the root cause of the disease rather than just symptoms.
Clinical Results:
Studies involving 27 patients showed dramatic improvements. Severe infections dropped by more than 90%, and bleeding complications were significantly reduced within the first year. Many participants remained stable for years after treatment.
Safety Considerations:
Reported side effects include rash, infections, gastrointestinal issues, liver injury, and low blood counts.
Regulatory Significance:
The FDA applied flexible standards for this rare condition, granting Waskyra multiple designations such as Orphan Drug and Regenerative Medicine Advanced Therapy. The product was developed by Fondazione Telethon ETS, marking the first time a non-profit organization has received approval for a cell and gene therapy.
This milestone offers new hope for individuals living with WAS, enabling them to pursue normal activities and reducing the burden of a previously devastating disease.
