Source: European Medicines Agency (EU) Revision Year: 2026 Publisher: Fondazione Telethon ETS, Via Varese 16/B, 00185 Rome, Italy
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.
Waskyra must be administered in a qualified treatment centre by a physician with experience in haematopoietic stem cell transplantation (HSCT) and trained for administration and management of patients treated with the medicinal product.
Before mobilisation, apheresis and reduced intensity conditioning are initiated, it must be confirmed that haematopoietic stem cell (HSC) transplantation is appropriate for the patient.
Physicians should refer to the SmPCs of medicinal products used for pre-treatment, peripheral blood mobilisation and conditioning to ensure appropriate guidance to the patient.
Waskyra is intended for autologous use only and should be administered once (see section 4.4).
Treatment consists of a single dose for infusion containing a dispersion of viable CD34+ cells in one or more infusion bags.
The dose of Waskyra to be administered is defined based on the patient's body weight at the time of infusion.
The minimum recommended dose is 7 × 106 CD34+ cells/kg of body weight.
The maximum volume of Waskyra to be administered should remain <20% of the patient's estimated plasma volume (see section 4.4 and section 6.6).
See the accompanying Lot Information Sheet (LIS) for additional information pertaining to dose.
The treating physician should confirm that autologous HSPC gene therapy administration is clinically appropriate for the patient before rituximab and conditioning is initiated (see section 4.4).
Conditioning should not begin until the complete set of infusion bag(s) constituting the dose of Waskyra has been received and stored at the administration site, and the availability of the back-up collection is confirmed.
The autologous CD34+ cells are isolated from mobilised peripheral blood. This is achieved by apheresis procedure(s) following peripheral blood mobilisation.
To obtain CD34+ cells for medicinal product manufacturing and for autologous back up, patients are required to undergo haematopoietic stem and progenitor cell (HSPC) mobilisation with granulocyte-colony stimulation factor (G-CSF) and plerixafor followed by leukapheresis.
A total HSPC collection target of 40 × 106 CD34+ cells/kg is recommended for medicinal product manufacture. In addition to this, at least 3 × 106 CD34+ cells/kg should be collected as autologous back-up The back-up cells may be harvested either through mobilised peripheral blood apheresis or bone marrow harvest.
Busulfan and fludarabine are the recommended conditioning medicinal products.
| Days before the treatment infusion | Dosing regimen | Dose | |
| Rituximab | Day-22 (+/- 1 day) | The infusion of a single dose of IV Rituximab, a monoclonal antibody anti-CD20, is recommended at Day –22 (+/- 1 day) before the infusion of Waskyra, with the aim to deplete B cells pre-treatment. Infusion rate is in accordance with the summary of product characteristics (SmPC) To reduce the occurrence of potential adverse reactions, Rituximab infusion should be preceded by appropriate premedication with IV antihistaminic medicinal product, paracetamol, and steroids, following standard procedures. These should be repeated after 6 hours. | 375 mg/m² |
| Busulfan | Day-4 to Day-2 | Patients will receive body weight-based doses of IV busulfan. Patients are scheduled to receive a total of 8 doses, given every 6 hours from Day-4 to Day-2. If the target AUC of 48 000 ng/mL*h (± 10%) is not achieved with 8 doses, additional doses may be administered. Dosing will be stopped if the target AUC was reached prior to the eighth dose. Dose adjustment will be performed according to busulfan PK levels to achieve the target cumulative AUC of 48 000 ng/mL*h (± 10%). The Waskyra cell infusion will be scheduled to allow a washout time of at least 24 hours from the last dose of busulfan. | The starting dose of busulfan is based on patient's weight according to the following scheme: • 1 mg/kg/dose (<9 kg); • 1.2 mg/kg/dose (9 - 16 kg); • 1.1 mg/kg/dose (>16 - 23 kg); • 0.95 mg/kg/dose (>23 - 34 kg); • 0.8 mg/kg/dose (>34 kg). The subsequent doses of busulfan should be estimated from pharmacokinetics (PK) sampling and adjusted accordingly. The busulfan PK analysis should be performed by serial blood sampling before and after the infusion of at least two separate doses usually the first and fifth dose. Special attention should be kept on the busulfan dosing in order to achieve the target cumulative AUC of 48 000 ng/ml*h (± 10%). A dose adjustment should be performed if the total predicted AUC shows more than 10% deviation from the target (total predicted AUC is <43 200 ng/mL*h or >52 800 ng/mL*h). The number of doses of Busulfan may be decreased if the cumulative predicted AUC is too high. |
| Fludarabine | Day-4 and Day-3 | Fludarabine is administered IV once daily in Day-4 and Day-3. The infusion rate should be in accordance with the fludarabine Summary of Product Characteristics (SmPC). | 30 mg/m²/day |
AUC = area under the curve; IV=intravenous; PK=pharmacokinetic; m² = square meter
Prophylactic and empiric use of anti-infectives (bacterial, fungal, viral) should be considered for the prevention and management of infections especially during the neutropenic period following conditioning (see section 4.4). Infection control measures and isolation procedures should be employed during the hospitalisation according to local standards.
Antiallergic medicinal products, such as intravenous chlorpheniramine, are recommended be administered 15-30 minutes before the infusion of Waskyra to reduce the possibility of an allergic reaction to the infusion.
Waskyra has not been studied in patients >65 years of age.
Waskyra has not been studied in patients with HIV-1, HIV-2, active HBV, or active HCV. Patients should be screened for HIV-1, HIV-2, HBV, and HCV and any other infectious agents in accordance with local guidelines before collection of cells for manufacturing.
Waskyra has not been studied in patients with evidence of myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukemia, or other serious hematological disorders. Treatment with Waskyra is not recommended in these patients.
The safety and efficacy of Waskyra in patients < 6 months of age have not yet been established. No data are available.
Waskyra is for intravenous infusion only.
Prior to Waskyra infusion, it must be confirmed that the patient's identity matches the essential unique patient information on the Waskyra infusion bag(s) labels and accompanying documentation. When more than one bag of Waskyra is needed, only one bag of medicinal product should be infused at a time. The total number of infusion bags to be administered must also be confirmed with the patient specific information on the Lot Information Sheet (LIS) (see section 4.4).
Waskyra infusion will be scheduled to allow a washout time of at least 24 hours from the last dose of busulfan.
The timing of thaw and infusion of Waskyra should be coordinated. The infusion start time should be confirmed in advance and adjusted for thaw so that Waskyra is available for infusion when the patient is ready. To maintain product viability, as soon as thawing is complete, it is recommended that Waskyra is administered immediately. Administration must be completed within 2 hours from the time of thawing.
Administer the product as an intravenous infusion via a central venous catheter. When more than one bag of Waskyra is provided, one bag of medicinal product should be thawed at a time.
Each bag should be infused at an infusion rate which does not exceed 5 mL/kg/h, within approximately 30 minutes. The recommended administration set consists of a blood transfusion set equipped with a 200 μm filter.
Patients should be monitored closely prior to, during, and after infusion. Vital signs (blood pressure, heart rate, and oxygen saturation) and the occurrence of any symptom should be checked every ten minutes during the infusion and every hour, for 3 hours, after the infusion (see section 4.4).
Patients should be monitored frequently by complete blood count for at least 6 weeks after infusion or until recovery of haematopoiesis and infections managed according to standard guidelines and medical judgement (see section 4.4).
For detailed instructions on preparation, administration, measures to take in case of accidental exposure and disposal of Waskyra, see section 6.6.
No data from clinical trials are available regarding overdose of Waskyra.
6 months.
Once thawed, maximum 2 hours at room temperature (20°C - 25°C). For products formulated at 2 × 106 cells/mL, the maximum time at room temperature should be 45 minutes.
Waskyra infusion bags must be stored in the vapour phase of liquid nitrogen (< -130°C) and must remain frozen until the patient is ready for treatment to ensure viable cells are administered to the patient.
Keep the infusion bag(s) in the metal cassette(s). Do not unseal the overwrap bag before thawing. Do not re-freeze after thawing.
For storage conditions after thawing of the medicinal product, see section 6.3.
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.
Precautions to be taken before handling or administering the medicinal product:
Waskyra should be transported within the facility in closed, break-proof, leak-proof containers.
Definition of the dose to be administered:
Preparation for the infusion:
Checking prior to thawing:
Thawing:
Administration:
Precautions to be taken for the disposal of the medicinal product:
Accidental exposure:
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