FDA Approves First Gene Therapy Treatment For Rare Immune Disorder Wiskott-Aldrich Syndrome

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.

by Joji Xavier

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