Waskyra
What Waskyra is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
Key facts
- Brand
- Waskyra
- Active substance
- etuvetidigene autotemcel
- Disease area
- Wiskott-Aldrich Syndrome
- Medicine group
- Other hematological agents
- ATC code
- B06
- Marketing authorisation holder
- Fondazione Telethon ETS
- Authorised in the EU since
- 2026
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Waskyra used for?
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.
What is the active substance in Waskyra?
Waskyra contains etuvetidigene autotemcel.
Who makes Waskyra?
The EU marketing authorisation holder is Fondazione Telethon ETS.
Is Waskyra an orphan medicine?
Yes. Waskyra is designated an orphan medicine in the EU, for a rare disease (Wiskott-Aldrich Syndrome).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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