FDA Issues Priority Review Voucher for Rare Pediatric Disease Treatment
February 22, 2026
February 22, 2026
WASHINGTON, Feb. 22 (TNSFR) -- The U.S. Department of Health and Human Services Food and Drug Administration announced the issuance of a priority review voucher to Fondazione Telethon ETS for the development of a treatment targeting a rare pediatric condition.
WASKYRA (etuvetidigene autotemcel) received approval on Dec. 9, 2025. The gene therapy is indicated for adults and pediatric patients aged 6 months and older who have Wiskott-Aldrich Syndrome (WAS). This specific patient popul . . .
WASKYRA (etuvetidigene autotemcel) received approval on Dec. 9, 2025. The gene therapy is indicated for adults and pediatric patients aged 6 months and older who have Wiskott-Aldrich Syndrome (WAS). This specific patient popul . . .