Source: European Medicines Agency (EU) Revision Year: 2026 Publisher: Fondazione Telethon ETS, Via Varese 16/B, 00185 Rome, Italy
Waskyra 2-10 × 106 cells/mL dispersion for infusion.
| Pharmaceutical Form |
|---|
|
Dispersion for infusion. A clear to slightly cloudy, colourless to yellow or pink dispersion. |
Waskyra (etuvetidigene autotemcel) is a genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human Wiskott-Aldrich Syndrome (WAS) gene.
Each patient-specific infusion bag of Waskyra contains etuvetidigene autotemcel at a batch-dependent concentration of genetically modified autologous CD34+ cell enriched population.
The medicinal product is packaged in one or more infusion bags overall containing a dispersion for infusion of 2–10 × 106 cells/mL of viable CD34+ enriched cell population suspended in a cryopreservative solution.
Each patient-specific infusion bag contains 10 to 20 mL of dispersion for infusion.
The quantitative information of medicinal product, including the batch dependent concentration and the number of infusion bags to be administered, is presented in the Lot information sheet (LIS) accompanying the medicinal product for treatment.
Excipients with known effect:
This medicinal product contains 3.5 mg sodium per mL and 55 mg dimethylsulfoxide (DMSO) per mL (see section 4.4).
For the full list of excipients, see section 6.1.
| Active Ingredient |
|---|
|
Etuvetidigene autotemcel is an ex vivo genetically modified autologous CD34+ haematopoietic stem and progenitor cell gene therapy. The genetically modified cells are capable of expressing the functional WAS protein. Following administration, the genetically modified cells engraft and repopulate the haematopoietic compartment. These genetically modified cells containing the corrected WAS protein (WASP) differentiate and produce biologically active lymphoid and myeloid progenitors whose progeny express WAS protein. |
| List of Excipients |
|---|
|
Dimethyl sulfoxide |
50 mL ethylene vinyl acetate (EVA) infusion bag(s) with two available spike ports, packed in an EVA overwrap bag placed inside a metal cassette.
Waskyra is shipped from the manufacturing facility to the treatment centre storage facility in a cryoshipper, which may contain multiple metal cassettes intended for a single patient.
Each metal cassette contains one infusion bag of Waskyra.
Fondazione Telethon ETS, Via Varese 16/B, 00185 Rome, Italy
EU/1/25/1996/001
| Drug | Countries | |
|---|---|---|
| WASKYRA | Lithuania, United States |
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