EUNew treatments and approvals
EU approves the gene therapy etuvetidigene autotemcel for Wiskott-Aldrich syndrome
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
The European Commission approved etuvetidigene autotemcel for Wiskott-Aldrich syndrome. The treatment is for patients aged 6 months and older who do not have a suitable related donor. The patient's own stem cells are corrected in the laboratory and given back once into a vein. In a total of 27 patients, severe infections fell from 2 to 0.12 per year. Moderate and severe bleeding also fell from 2 to 0.16 per year.
What does it mean for patients and families?
The treatment is given at a specialist center, and beforehand a drug is given that empties the bone marrow. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
Avrupa İlaç Ajansı (EMA) · source page
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Wiskott-Aldrich syndrome
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.