Overview
- On 13 November 2025, the CHMP adopted a positive opinion recommending EU marketing authorisation for Waskyra.
- The CAT found the benefits outweighed risks for patients eligible for HSCT who lack an HLA‑matched related donor, and the CHMP agreed.
- Across 27 treated patients, overall survival reached 96% with steep declines in annualised severe infections and moderate‑to‑severe bleeding.
- Waskyra is a one‑time autologous CD34+ stem cell gene therapy using a lentiviral vector to restore WAS protein expression, delivered in qualified HSCT centres.
- The most common adverse events were procedure‑related, including effects from mobilisation, conditioning and infusion devices; the applicant is Fondazione Telethon ETS, with orphan status due for COMP review and national pricing decisions to follow any Commission approval.