Quick answers
- What is Etuvetidigene autotemcel used for?
- The FDA-approved label (Waskyra, Fondazione Telethon ETS, July 23, 2026) says: “WASKYRA ® is indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. …” This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- What are the side effects of Etuvetidigene autotemcel?
- The “Adverse Reactions” section of the FDA label (Waskyra, Fondazione Telethon ETS, July 23, 2026) begins: “The most common adverse reactions (incidence ≥ 20%) are catheter related infections, bacterial and viral infections, diarrhea, vomiting, stomatitis, liver injury, head injury, rhinitis, cough, rash, petechiae, hypersensitivity, anemia, febril neutropenia, epistaxis, pyrexia, catheter site complications.(6.1) To report SUSPECTED ADVERSE REACTIONS, contact Fondazione Telethon ETS at toll-free phone 1-888-212-6928 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.” … This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- What are the drug interactions of Etuvetidigene autotemcel?
- The “Drug Interactions” section of the FDA label (Waskyra, Fondazione Telethon ETS, July 23, 2026) begins: “No formal drug interaction studies have been performed. WASKYRA ® is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters. 7.1 Effect of Vaccines on WASKYRA ® The safety and effectiveness of vaccination during or following WASKYRA ® treatment have not been studied.” … This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- Does Etuvetidigene autotemcel have a boxed warning?
- No. None of the 2 FDA labels we found for this medicine has a boxed warning.
- Is there a shortage of Etuvetidigene autotemcel?
- No current shortage is listed by the FDA for the products of this medicine (FDA data from October 30, 2026).
- Has Etuvetidigene autotemcel been recalled?
- No recall linked to the products of this medicine appears in the FDA enforcement reports we use.
- Who makes Etuvetidigene autotemcel?
- 2 companies list prescription products of Etuvetidigene autotemcel in the FDA NDC Directory, including Orphan Therapies LLC and Fondazione Telethon ETS.
What Etuvetidigene autotemcel is used for
Quoted from the “Indications and Usage” section of the FDA-approved label.
WASKYRA ® is indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. …
— FDA label: Waskyra, Fondazione Telethon ETS, effective July 23, 2026. Read the full label on DailyMed →
Label quoted: the one for the most common form (injection, intravenous), Waskyra, Fondazione Telethon ETS; it covers 1 product in the FDA NDC Directory. The side effects, interactions, dosage and pregnancy quotes below come from this same label.
The FDA has 2 labels for products of this medicine (different makers and forms); uses can differ between them. Check the label that comes with your own medicine.
This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
Boxed warning
A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.
The label quoted above has no boxed warning.
0 of 2 FDA labels for this medicine have a boxed warning.
Etuvetidigene autotemcel side effects
Quoted from the “Adverse Reactions” section of the FDA-approved label. Tell your doctor about any side effect that worries you.
The most common adverse reactions (incidence ≥ 20%) are catheter related infections, bacterial and viral infections, diarrhea, vomiting, stomatitis, liver injury, head injury, rhinitis, cough, rash, petechiae, hypersensitivity, anemia, febril neutropenia, epistaxis, pyrexia, catheter site complications.(6.1) To report SUSPECTED ADVERSE REACTIONS, contact Fondazione Telethon ETS at toll-free phone 1-888-212-6928 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
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The safety data described in this section reflects exposure to WASKYRA ® in two clinical studies, Study 1 (201228) and Study 2 (OTL-103-4) as well as patients in expanded access program (EAP). A total of 27 patients received a single infusion of WASKYRA ® at a dose range of 7 to 31×10 6 CD34 + cells/kg (median dose: 16.90×10 6 CD34 + cells/kg). The median duration of the follow up was 5.67 years (range: 0.37-13.26 years) [see Clinical Studies (14)]. During the above-mentioned clinical trials no adverse reactions attributable to the gene therapy were reported. There were 45 serious adverse reactions reported in 21 patients including catheter-related infections (n=11), bacterial and viral infections (n=10), pyrexia (n=3), prolonged neutropenia (n=2), vomiting (n=2), veno-occlusive disease (n=1), aspergillus infection (n=1). One patient died approximately 4.5 months after WASKYRA ® infusion due to neurological decompensation. Table 1: Adverse Reactions Occurring in ≥10% of Patients (N=27) Adverse Reactions Any Grade n (%) Grade 3 or higher n (%) Infections and Infestations - - Catheter related infection 16 (59) 14 (52) Respiratory tract infection Is a composite that includes multiple related terms. 18 (67) 4 (15) Conjunctivitis * 10 (37) 0 Cytomegalovirus infection * 8 (30) 4 (15) Epstein-Barr virus infection 5 (19) 1 (4) Gastroenteritis 5 (19) 2 (7) Ear infection 4 (15) 0 Oral candidiasis 4 (15) 0 Urinary tract infection * 5 (19) 3 (11) Balanoposthitis 3(11) 0 Gastrointestinal disorders - - Diarrhea * 16 (59) 3 (11) Vomiting 12 (44) 5 (19) Liver injury * 16 (59) 3 (11) Stomatitis 6 (22) 3 (11) Hematochezia 5 (19) 0 Abdominal pain 3 (11) 0 Constipation 3 (11) 0 Nervous System disorders - - Head Injury* 10 (37) 1 (4) Headache 4 (15) 2 (7) Respiratory, thoracic and mediastinal disorders - - Rhinitis 9 (33) 0 Cough * 8 (30) 0 Wheezing 4 (15) 0 Oropharyngeal pain 3 (11) 1 (4) Skin and subcutaneous tissue disorders - - Rash Rash includes eczema, rash, urticaria, dermatitis, dry skin, erythema. 23 (85) 9 (33) Petechiae 16 (59) 1 (4) Immune system disorders - - Hypersensitivity * 6 (22) 4 (15) Lymphadenopathy 4 (15) 1 (4) Blood and lymphatic system disorders - - Anemia * 11 (41) 4 (15) Immune thrombocytopenia 4 (15) 4 (15) Febrile neutropenia 7 (26) 6 (22) Epistaxis 9 (33) 0 Mouth hemorrhage 3 (11) 0 Traumatic hematoma * 4 (15) 0 General disorders and administration site conditions - - Pyrexia 11 (41) 6 (22) Catheter site complications Catheter site complications includes Catheter site inflammation, Catheter site hemorrhage, Unintentional medical device removal. 10 (37) 1 (4) Note: Adverse reactions are defined as adverse events occurred during conditioning and year 1 following WASKYRA ® administration.
Table from the label
| Laboratory Abnormality | All Grade n (%) | Grade 3 or 4 n (%) |
|---|---|---|
| Neutrophils decreased | 10 (37) | 9 (33) |
| Eosinophils increased | 7 (26) | 1 (4) |
| Electrolyte imbalanceElectrolyte imbalance includes hypokalemia, hyperkalemia, hyponatremia, hypomagnesemia | 11 (41) | 6 (22) |
| Creatinine increased | 3 (11) | 0 |
| Calcium decreased | 3 (11) | 0 |
— FDA label: Waskyra, Fondazione Telethon ETS, effective July 23, 2026. This is part of the section. Read the full label on DailyMed →
Etuvetidigene autotemcel drug interactions
Quoted from the “Drug Interactions” section of the FDA-approved label. Tell your pharmacist about every medicine, vitamin and herbal product you take.
No formal drug interaction studies have been performed. WASKYRA ® is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters. 7.1 Effect of Vaccines on WASKYRA ® The safety and effectiveness of vaccination during or following WASKYRA ® treatment have not been studied.
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Vaccination with live virus vaccines is not recommended until immune reconstitution is completed following treatment with WASKYRA ®. 7.2 Effect of Anti-retrovirals on WASKYRA ® Anti-retroviral medications may interfere with the manufacturing of WASKYRA ®. Patients should not take anti-retroviral medications for at least one month prior to mobilization until at least 7 days after WASKYRA ® infusion. If a patient requires anti-retroviral treatment following exposure to HIV/HTLV, initiation of WASKYRA ® treatment should be delayed until an HIV/HTLV western blot and viral load assay have been performed at 6 months post-exposure.
— FDA label: Waskyra, Fondazione Telethon ETS, effective July 23, 2026. Read the full label on DailyMed →
Etuvetidigene autotemcel dosage
Quoted from the “Dosage and Administration” section of the FDA-approved label. Your own dose is the one your doctor prescribes: do not change it on your own.
For autologous use only. For single-dose intravenous use only. For autologous use only. For intravenous use only. Patients are required to undergo hematopoietic stem and progenitor cell (HSPC) mobilization followed by apheresis to obtain CD34 + cells for WASKYRA ® manufacturing. (2.2) Dosing of WASKYRA ® is based on the number of CD34 + cells in the infusion bag(s) per kg of body weight at the time of infusion.
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(2.1) The minimum recommended dose is 7 x 10 6 CD34 + cells per kg. Reduced-intensity conditioning is required before infusion of WASKYRA ®. (2.2) Prior to WASKYRA ® infusion, confirm that the patient’s identity matches the essential unique patient information on the infusion bag(s). (2.3) 2.1 Recommended Dose The minimum recommended dose of WASKYRA ® is 7 × 10 6 CD34 + cells/kg based on patient’s body weight at the time of infusion. The maximum volume of WASKYRA ® to be administered should remain < 20% of the patient’s estimated plasma volume. 2.2 Patient Preparation before WASKYRA ® Infusion Mobilization and apheresis Mobilize hematopoietic stem and progenitor cells (HSPC) using G-CSF with plerixafor according to established protocols. Perform apheresis to collect CD34+ cells required for WASKYRA ® manufacturing following the mobilization procedure. Collect and cryopreserve a back-up supply of CD34 + stem cells containing at least 3 x 10⁶ CD34 + cells/kg from the patient. Complete this collection before initiating reduced intensity conditioning and WASKYRA ® infusion. Store the back-up collection for potential rescue treatment in the following scenarios: WASKYRA ® compromise after reduced intensity conditioning initiation but before scheduled infusion, primary engraftment failure, or prolonged bone marrow aplasia following WASKYRA ® treatment. Pre-treatment and conditioning Administer rituximab (anti-CD20 monoclonal antibody) approximately 22 days before WASKYRA ® administration to deplete autoreactive B-cells and provide pre-emptive treatment for potential lymphoproliferative disorder due to Epstein Barr Virus infection which is a risk factor in WAS patients. Administer busulfan and fludarabine for reduced intensity conditioning before infusion of WASKYRA ® to promote engraftment of the genetically modified autologous CD34 + cells. Do not begin conditioning until the complete set of infusion bag(s) constituting the dose of WASKYRA ® has been received and stored at the administration site. Confirm availability of the back-up collection. Premedication Administer intravenous chlorpheniramine (0.2 mg/kg, max. dose 10 mg), or an equivalent 15-30 minutes before the infusion of WASKYRA ® to reduce the possibility of an allergic reaction to the infusion. 2.3 Administration Receipt WASKYRA ® is shipped in the vapor phase of liquid nitrogen (<-130°C; -202°F) from the manufacturing site to the qualified treatment center for the infusion. The Lot Information Sheet and the Chain of Custody documentation travels with the cryoshipper. Upon arrival check the temperature on the display of the cryoshipper, open it using the proper DPI, and confirm the patient code, batch number and number of bags against product label(s).
— FDA label: Waskyra, Fondazione Telethon ETS, effective July 23, 2026. This is part of the section. Read the full label on DailyMed →
Etuvetidigene autotemcel in pregnancy
Quoted from the pregnancy part of the FDA-approved label. Ask your doctor before you take any medicine while pregnant or planning a pregnancy.
Risk Summary There are no clinical data from the use of WASKYRA ® in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with WASKYRA ® to assess whether it can cause fetal harm when administered to a pregnant woman. WASKYRA ® must not be administered during pregnancy because of the risk associated with conditioning. Pregnancy after WASKYRA ® infusion should be discussed with the treating physician.
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In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.
— FDA label: Waskyra, Fondazione Telethon ETS, effective July 23, 2026. Read the full label on DailyMed →
When Etuvetidigene autotemcel should not be used
Quoted from the “Contraindications” section of the FDA-approved label.
Hypersensitivity to the active substance or to any of the excipients [see Hypersensitivity and Infusion Related Reactions (5.1)]. Previous treatment with HSCT within 6 months prior to treatment or HSCT with evidence of residual donor cells. Previous treatment with hematopoietic stem cell gene therapy. Contraindications to the mobilization and the conditioning regimen. Hypersensitivity to the active substance or to any of the excipients.
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(4) Previous treatment with HSCT within 6 months prior to screening or HSCT with evidence of residual donor cell. (4) Previous treatment with hematopoietic stem cell gene therapy. (4) Contraindications to the mobilization and the conditioning regimen. (4)
— FDA label: Waskyra, Fondazione Telethon ETS, effective July 23, 2026. Read the full label on DailyMed →
Forms and how it is given
| Dosage forms | Injection |
|---|---|
| Route | Intravenous |
| Prescription products | 2 in the FDA NDC Directory |
| Brand names | Waskyra |
Companies that list this medicine
Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.
- Orphan Therapies LLC1 product
- Fondazione Telethon ETS1 product
FDA approval history
- BLA: Biologics License Application: how a biologic medicine (made from living cells) is approved.
| Application | Sponsor | Approved | Later changes approved |
|---|---|---|---|
| BLA125846 | — | Date not on file | — |
Shortage status
The FDA drug shortage list does not show these products.
Recalls
No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.
Official identifiers
| RxCUI (RxNorm) | 2747661, 2747667 |
|---|---|
| SPL set ID of the quoted label | 45929c87-685d-b21c-e063-6394a90a3818 |
| FDA labels (SPL) | 2 |
| FDA applications | BLA125846 |
| Active ingredients (FDA) | ETUVETIDIGENE AUTOTEMCEL |
Next steps
- Find a pharmacy near youAsk which price applies to you and whether your product is affected by a recall
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- Orphan Therapies LLCMaker or distributor
- Fondazione Telethon ETSMaker or distributor
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Sources
- FDA-approved label on DailyMed (National Library of Medicine) — Waskyra, Fondazione Telethon ETS, effective July 23, 2026; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
- NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
- Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →