Powered By
Waskyra (etuvetidigene autotemcel) is a prescription gene therapy approved by the U.S. Food and Drug Administration (FDA) to treat children 6 months and older and adults with Wiskott-Aldrich syndrome. WAS is a rare genetic condition that affects the blood and immune system.
Waskyra is a gene therapy. It works by adding working copies of the WAS gene into a person’s own blood-forming stem cells. These cells are changed outside the body.
After the cells are given back, they can settle in the bone marrow. The bone marrow is the soft tissue inside bones that makes blood cells.
The changed cells can make blood cells with WAS protein. This protein helps blood and immune cells work properly.
Doctors prescribe Waskyra when a child 6 months or older or an adult has Wiskott-Aldrich syndrome with a mutation in the WAS gene, a stem cell transplant is appropriate, and there is no suitable matched related stem cell donor.
Waskyra is given as a one-time intravenous (IV) infusion. Before the infusion, doctors collect the person’s stem cells and prepare them. The person also receives treatment to help the new cells settle in the body.
Waskyra is given at a qualified treatment center.
The minimum recommended dose of Waskyra is 7 × 10⁶ CD34+ cells per kilogram of body weight.
This means the dose is based on how much the person weighs at the time of infusion.
This information is based on the prescribing information, but your healthcare provider may tailor your treatment plan. Always follow their guidance.
In clinical studies of Waskyra for Wiskott-Aldrich syndrome, side effects were reported in 27 people who received one infusion of Waskyra. The most common adverse reactions occurred in 20 percent or more of people and included:
Waskyra can cause serious side effects that may require immediate medical attention. These include:
Get medical help right away if you think you are having a serious reaction.
Fondazione Telethon ETS, the manufacturer of Waskyra, does not offer a traditional copay card program because Waskyra is a one-time gene therapy administered at specialized treatment centers. Instead, patient support programs are available to help people navigate insurance coverage and access to treatment.
These support programs may offer assistance with insurance approvals and prior authorizations. Financial counselors at treatment centers may also help eligible people identify financial assistance options, including hospital-based financial support programs, grants, or other funding resources.
To learn more about available support services, speak with your healthcare provider or call Fondazione Telethon ETS at 888-212-6928.
Before starting Waskyra, your doctor will check that you are not pregnant if you can become pregnant. Waskyra must not be given during pregnancy.
Vaccination with live virus vaccines is not recommended until your immune system has recovered after Waskyra treatment.
Tell your doctor if you have any allergies to Waskyra or any ingredients in Waskyra. Do not receive Waskyra if you have had an allergic reaction to Waskyra or any ingredients in Waskyra.
Tell your doctor about all medicines you take, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements. Antiretroviral medicines may interfere with making Waskyra. Your doctor may tell you to stop these medicines before treatment.
Before Waskyra, doctors will collect your stem cells. They will also collect and store backup stem cells in case they are needed. You will receive other treatments before Waskyra to help prepare your body.
People treated with Waskyra should not donate blood, organs, tissues, or cells for transplantation at any time in the future.
After Waskyra, some HIV tests may give a false-positive result. This can happen with PCR-based HIV tests. Tell your healthcare team that you received Waskyra before having an HIV test.
You also should not receive Waskyra if you had a stem cell transplant within the past six months, have donor cells left from a prior transplant, had prior hematopoietic stem cell gene therapy, or cannot receive the mobilization or conditioning treatment needed before Waskyra.
Males who can father a child and females who can become pregnant should use effective birth control from the start of mobilization through at least 6 months after Waskyra is given.
Breastfeeding should be stopped during conditioning. Talk with your doctor if you are pregnant, planning to become pregnant, or breastfeeding.
These answers are fact-checked by our editorial staff.
How effective is Waskyra for Wiskott-Aldrich syndrome?
In studies of people with Wiskott-Aldrich syndrome, severe infections decreased after treatment with Waskyra. They went from two infections per patient-year before treatment to 0.2 infections per patient-year during months 6 to 18 after treatment.
Moderate and severe bleeding events also decreased. They went from 2 events per patient-year before treatment to 0.8 events per patient-year during the first 12 months after treatment.
Gene-corrected cells were seen starting one month after Waskyra treatment. They were seen during follow-up in all people who were evaluated, with follow-up lasting up to nine years in some people.
How long does Waskyra take to work for WAS?
The study results do not give one exact time when Waskyra starts to work. In studies, gene-corrected cells were seen starting one month after Waskyra treatment.
Severe infections were measured during months 6 to 18 after treatment. Moderate and severe bleeding events were measured during the first 12 months after treatment.
The studies also found that the body made WAS protein after treatment. More than 65 percent of lymphocytes, a type of white blood cell, expressed WAS protein at one year. More than 74 percent of platelets expressed WAS protein at 30 days.
What precautions and monitoring are needed with Waskyra for WAS?
Before Waskyra, tell your doctor about all medicines you take, including antiretroviral medicines, because they may interfere with making Waskyra. Your doctor will also check for pregnancy if you can become pregnant.
After Waskyra, do not get live virus vaccines until your immune system has recovered. Do not donate blood, organs, tissues, or cells for transplantation at any time in the future.
Tell your healthcare team that you received Waskyra before having an HIV test, because some HIV tests may give a false-positive result.
Your doctor will monitor you for serious risks, including allergic reactions, low blood cell counts, infections, liver problems, and engraftment failure. Liver function tests are monitored for one month after treatment, and blood counts are monitored for at least eight weeks.
You may also need long-term monitoring for cancer risk after treatment.
On myPIteam, people share their experiences with primary immunodeficiency, get advice, and find support from others who understand.
Learn more about Waskyra and primary immunodeficiency care:
More resources from myPIteam that mention Waskyra
Become a member to get even more
Join
Your Privacy Choices
