The FDA has approved Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening immune disorder.
Waskyra is indicated for pediatric patients 6 months and older, as well as adults with Wiskott-Aldrich syndrome who are eligible for stem cell transplant but lack a matched donor. The therapy uses a patient’s own genetically corrected blood stem cells to restore functional WAS protein expression, according to a Dec. 9 news release.
Approval was based on two single-arm clinical trials and an expanded access program involving 27 patients. Treated patients saw a 93% reduction in severe infections and a 60% reduction in moderate to severe bleeding within the first year after treatment, according to the FDA. Most patients had no moderate or severe bleeding events after four years.
The FDA said it had exercised regulatory flexibility in several areas, including trial design and manufacturing, to enable timely approval while requiring postmarket commitments. The product received Orphan Drug, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations.
