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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: etuvetidigene autotemcel
Trade Name: Waskyra
Date Designated: 04/30/2010
Orphan Designation: Treatment of Wiskott Aldrich syndrome
Orphan Designation Status: Designated/Approved
Fondazione Telethon ETS
Via Varese 16/B
Rome 00185
Italy

The sponsor address listed is the last reported by the sponsor to OOPD.

Marketing approved:

1 Generic Name: etuvetidigene autotemcel
Trade Name: Waskyra
Marketing Approval Date: 12/09/2025
Approved Labeled Indication: treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available
Exclusivity End Date: TBD  
*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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