Waskyra (etuvetidigene autotemcel)

BL 125846

Waskyra (etuvetidigene autotemcel)

BL 125846

U.S. License Holder:

Fondazione Telethon ETS

Date of License:

December-09-2025

Last Update:

August-08-2026

approved_indications FDA-Approved Indications

WASKYRA (etuvetidigene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.

Methodology

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