Class Name(s):

• 26:12 Gene Therapy

Brand Name(s):

• Waskyra ^®

WHY is this medicine prescribed?

Etuvetidigene autotemcel injection is used for the treatment of Wiskott-Aldrich Syndrome (WAS; a genetic disorder that causes immune system dysfunction) in patients who are candidates for hematopoietic stem cell transplant but do not have a matched related donor. Etudetidigene autotemcel is in a class of medications called gene therapies. It works by taking a patient's own blood stem cells and genetically modifying it in a lab to correct the gene that causes the immune system dysfunction and then infusing the corrected cells back into the patient so that healthy immune cells can be produced.

HOW should this medicine be used?

Etuvetidigene autotemcel injection comes as a suspension to be given intravenously (into the vein) by a doctor or a nurse at a healthcare facility. It is given as a single dose. You will be given medications prior to the infusion to prevent adverse reactions to etuvetidigene autotemcel injection from occurring.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

Are there OTHER USES for this medication?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

What SPECIAL PRECAUTIONS should I follow?

Before receiving etuvetidigene autotemcel injection,

• tell your doctor or pharmacist if you are allergic to this medication, any part of this medication, or any other medications, foods or substances. Tell your doctor or pharmacist about the allergy and what symptoms you had.
• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take while taking etuvetidigene autotemcel. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
• tell your doctor or pharmacist if you are taking the following medications or have stopped taking them within the past month: antiretroviral medications used for prevention or treatment of infection with human immunodeficiency virus (HIV). Ask your doctor or pharmacist if you are not sure if a medicine you have taken or are taking is an antiretroviral medication.
• tell your doctor if you have ever had a hematopoietic stem cell transplant or have ever received hematopoietic stem cell gene therapy. Your doctor may tell you not to receive etuvetidigene autotemcel injection.
• tell your doctor if you have or have ever had infection with or exposure to HIV.
• tell your doctor if you or your partner are pregnant or plan to become pregnant. You or your partner should not be pregnant during and for 6 months after you receive etuvetidigene autotemcel injection. You may have to take a pregnancy test before starting treatment. You and your partner should use effective birth control to prevent pregnancy during your treatment and for 6 months after. Talk to your doctor about what birth control would work best for you and your partner.
• tell your doctor if you are breastfeeding. You likely will be told to stop breastfeeding before receiving etuvetidigene autotemcel injection.
• impact of etuvetidigene autotemcel on ability to father or conceive a child is unknown. Talk to your doctor about fertility risks and whether cryopreservation (freezing) of sperm or ovaries (eggs) before receiving etuvetidigene autotemcel injection is warranted.
• do not have any vaccinations without talking to your doctor.
• you should know that etuvetidigene autotemcel injection may cause infections and secondary malignancies after infection. Talk to your doctor about the risks of receiving etuvetidigene autotemel.
• you should know that you should not donate blood, tissue, cells or organs after receiving etuvetidigene autotemcel injection at any time in the future.

What SPECIAL DIETARY instructions should I follow?

Unless your doctor tells you otherwise, continue your normal diet.

What SIDE EFFECTS can this medicine cause?

Etuvetidigene autotemcel injection may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• diarrhea
• vomiting
• sores and inflammation in the mouth, tongue, gums, cheeks or palate
• headache
• sore throat
• cough, runny nose, wheezing
• rash
• petechiae (tiny pinpoint brown, red or purple spots under the skin caused by bleeding from the capillaries)
• bruising
• nose bleeds
• fever
• pain, swelling, or bruising at site of infusion

Some side effects can be serious. If you experience any of these symptoms, call your doctor immediately or get emergency medical treatment:

• yellowing of skin or whites of the eyes, lower abdominal pain, pale stools, dark urine, loss of appetite, extreme tiredness
• fever, extreme tiredness, productive cough, irritated eyes, swollen lymph nodes, ear pain, chest pain or shortness of breath, white lesion in the mouth, difficulty or painful urination, lower back pain
• blood in the stool, vomiting blood, pale skin, prolonged bleeding (taking longer to stop bleeding), unexpected bleeding (bleeding from gums, nose bleeds, blood in urine)
• rash, hives, itching, swelling of mouth or lips or throat, difficulty breathing or wheezing or shortness of breath, weakness

Etuvetidigene autotemcel injection may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What OTHER INFORMATION should I know?

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to etuvetidigene autotemcel.

Keep a written list of all of the prescription and nonprescription (over-the-counter) medicines, vitamins, minerals, and dietary supplements you are taking. Bring this list with you each time you visit a doctor or if you are admitted to the hospital. You should carry the list with you in case of emergencies.

This report on medications is for your information only, and is not considered individual patient advice. Because of the changing nature of drug information, please consult your physician or pharmacist about specific clinical use.

The American Society of Health-System Pharmacists, Inc. represents that the information provided hereunder was formulated with a reasonable standard of care, and in conformity with professional standards in the field. The American Society of Health-System Pharmacists, Inc. makes no representations or warranties, express or implied, including, but not limited to, any implied warranty of merchantability and/or fitness for a particular purpose, with respect to such information and specifically disclaims all such warranties. Users are advised that decisions regarding drug therapy are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and the information is provided for informational purposes only. The entire monograph for a drug should be reviewed for a thorough understanding of the drug's actions, uses and side effects. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information is not a substitute for medical care.

AHFS^® Patient Medication Information™ Copyright, 2026. The American Society of Health-System Pharmacists^®, 4500 East-West Highway, Suite 900, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.

Selected Revisions: January 15, 2026.