Waskyra (etuvetidigene autotemcel)

OrphanATMPAdditional monitoring

Region: 🇪🇺 EU
Active substance: etuvetidigene autotemcel
Company: Fondazione Telethon ETS
Status: Authorised
Therapeutic area: Wiskott-Aldrich Syndrome
CHMP opinion: Thu 13 Nov 2025
EU approval: Fri 09 Jan 2026
Designations: Orphan, ATMP, Additional monitoring
Application: EMEA/H/C/006525
ATC: B06
Official record: www.ema.europa.eu

Indication

Waskyra is indicated for the treatment of patients aged 6 months and older with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom haematopoietic stem cell (HSC) transplantation is appropriate and no suitable human leukocyte antigen (HLA)-matched related haematopoietic stem cell donor is available.

Regulatory milestones

Fri 09 Jan 2026🇪🇺 EU approval (EC decision)

Waskyra (etuvetidigene autotemcel) approved in the EU

Marketing authorisation to Fondazione Telethon ETS; Orphan, ATMP, Additional monitoring · source

Thu 13 Nov 2025🇪🇺 CHMP positive opinion

Waskyra (etuvetidigene autotemcel): CHMP positive opinion

Fondazione Telethon ETS; Orphan, ATMP, Additional monitoring · source

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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.