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Overview: Waskyra is a gene therapy that is given as a one-time infusion into a blood vessel. It is used to treat certain people with Wiskott-Aldrich syndrome who have a faulty gene. Waskyra is made from the person’s own stem cells. The faulty cells are “corrected” and then infused back into the body, which helps the body make new blood cells and certain proteins properly. Common side effects may include infections, swelling, or bleeding where the medicine is infused, other infections, diarrhea, vomiting, mouth sores, cough or runny nose, and rash. Get help right away if you get a fever, trouble breathing, dizziness, or unusual bleeding or bruising. Be sure to keep all appointments with your health care provider before and after receiving Waskyra.

Uses

What is Waskyra used for?

Waskyra (etuvetidigene autotemcel) is used to treat certain people with Wiskott-Aldrich syndrome (WAS). WAS is a rare inherited condition that affects the immune system and the body’s ability to make certain blood cells, including platelets. Platelets help to form blood clots. People with WAS can have bleeding, infections, eczema, and other problems.

Learn more about Waskyra for Wiskott-Aldrich Syndrome.

Waskyra may also be used for other conditions as determined by your health care provider.

How does Waskyra work (mechanism of action)?

Waskyra is a gene therapy. It is for certain people with Wiskott-Aldrich syndrome who have a faulty gene. Waskyra uses the person’s own stem cells that are collected from the blood. These cells are changed to include healthy, working copies of the faulty gene. The “corrected” cells are then infused back into the body, which helps the body make new blood cells and certain proteins properly.

How is Waskyra supplied (dosage forms)?

Waskyra is available in an injectable form that is given by a health care provider.

How should I store Waskyra?

Waskyra is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Waskyra?

The most common side effects of Waskyra are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Infection or other problems (such as swelling or bleeding) where the medicine is infused
  • Infections (see below)
  • Diarrhea
  • Vomiting
  • Mouth sores
  • Liver problems
  • Runny nose or stuffy nose
  • Cough
  • Rash
  • Tiny red, flat spots on the skin (petechiae)
  • Allergic reactions (see below)
  • Blood disorders (see below)
  • Nosebleeds
  • Fever

There may be other side effects of Waskyra that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Waskyra?

While less common, the most serious side effects of Waskyra are described below, along with what to do if they happen.

Severe Allergic Reactions. Waskyra may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Infusion Reactions. Waskyra can cause reactions that can be serious when it is given to you. Your health care provider will likely give you medicine before your dose to help reduce the risk of a reaction. Tell your health care provider right away if you have any of the following symptoms during and after getting your infusion.

  • Fever or chills
  • Joint or muscle pain
  • Redness of your face and neck
  • Trouble breathing
  • Chest pain
  • Hives
  • Nausea or vomiting

Blood Disorders. Waskyra can cause blood disorders, such as low red blood cell levels (anemia), low neutrophil levels (neutropenia), and low platelet levels (thrombocytopenia). These disorders can happen for several weeks after receiving Waskyra and may be severe. Keep all appointments with your health care provider. Get help right away if you have any of the following symptoms of blood disorders.

  • Fever
  • Shortness of breath
  • Pale or yellowish skin
  • Easy bruising or bleeding
  • Frequent infection
  • Unusual weakness or tiredness
  • Dizziness, lightheadedness, or feeling like you are about to pass out
  • Headache
  • Fast or abnormal heartbeat

Infections. You may have a higher risk of infections, including serious infections, after receiving Waskyra. Call your health care provider right away if you have an infection or any of the following symptoms.

  • Burning or stinging when you pee 
  • Coughing, or coughing up blood
  • Fever, sweats, chills, sore throat, or muscle aches
  • Shortness of breath
  • Diarrhea or stomach pain
  • Weight loss
  • Tiredness
  • Swollen glands

Risk of Infection. The process to make Waskyra involves the use of human and cow-based substances. It is possible that these substances may carry germs that can cause infections. While steps are taken to reduce this risk, there is still a small chance you could get an infection. Some infections may not cause symptoms. Keep all appointments after receiving Waskyra so your health care provider can check your health. Talk with your health care provider about any concerns you may have.

Liver Problem. Some people who have received Waskyra have had a liver problem called hepatic veno-occlusive disease. This happens when small blood vessels in the liver get blocked. Keep all appointments for blood work to check the health of your liver. Call your health care provider right away if you have any of the following symptoms.

  • Stomach or belly pain or swelling
  • Sudden weight gain
  • Nausea or vomiting
  • Weakness or unusual tiredness
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Cancer. Waskyra may increase the risk of a new cancer to develop or cause a previously treated cancer to recur. Your health care provider will likely watch you over your lifetime for possible cancer development. Tell your health care provider about any new symptoms that you may get.

Warnings & Precautions

Who should not use Waskyra?

Allergies to Ingredients. People who are allergic to any of the following should not use Waskyra.

  • Etuvetidigene autotemcel
  • Waskyra
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Waskyra.

Prior Stem Cell Transplant. Waskyra should not be used if you have had a hematopoietic stem cell transplant in the last 6 months or if you still have donor stem cells in your body. 

Prior Stem Cell Gene Therapy. Waskyra is a gene therapy. You should not use Waskyra if you have been treated in the past with a hematopoietic stem cell gene therapy.

Unable to Complete Preparation Steps. You will need to take certain steps before receiving Waskyra. For example, you will need to have stem cells taken from your blood (apheresis). You will also need to receive certain medicines before receiving Waskyra. Talk to your health care provider about the steps you need to take.

What should I know about Waskyra before using it?

Waskyra is usually given by a health care provider in a hospital or clinic. It is given as a one-time infusion into a vein (IV).

It is very important to keep all appointments with your health care provider before and after receiving Waskyra. These appointments help your health care provider make sure the treatment is safe and working well for you.

Tell all of your health care providers that you have received Waskyra. Waskyra may affect the results of certain lab tests. Also, if you need a blood transfusion after receiving Waskyra, the blood may need to be treated with a special process first to make it safer for you.

You should not donate blood, organs, cells, or tissues at any time after receiving Waskyra. You should receive a Patient Alert Card with this information. Talk with your health care provider if you have concerns.

If you can get your partner pregnant, you should use an effective method of birth control before you take the steps needed to receive Waskyra and for at least 6 months afterwards. See the Pregnancy section for more information.

Before receiving Waskyra, talk to your health care provider if you or your child may want to become pregnant or have a partner become pregnant in the future. 

There is a small risk that the “corrected” stem cells may not grow properly in your body. Your health care provider will likely keep a back-up supply of your stem cells before you receive Waskyra in case they are needed later. If you have any questions, ask your health care provider.

What should I tell my health care provider before using Waskyra?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Waskyra is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

Vaccines. Talk with your health care provider about any vaccines that you may need. You should not receive certain vaccines right before or after receiving Waskyra. Tell your health care provider about any vaccines you have recently received or have scheduled.

Other Medicines and Supplements. Waskyra may interact with other medicines and supplements. Before receiving Waskyra, tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Pregnancy. People who can become pregnant are not usually prescribed Waskyra. It is not known if or how Waskyra could affect pregnancy or harm an unborn baby. If you can get pregnant, you should have a test to make sure you are not pregnant before you take the steps needed to receive Waskyra. You should also use effective birth control before you take the steps needed to receive Waskyra and for at least 6 months afterwards. Tell your health care provider right away if you are pregnant, think you may be pregnant, or plan to become pregnant.

Breastfeeding. People who can breastfeed are not usually prescribed Waskyra. It is not known if Waskyra passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should receive Waskyra while breastfeeding.

Interactions

Does Waskyra interact with foods or drinks?

There are no known interactions between Waskyra and foods or drinks.

It is unknown if drinking alcohol will affect Waskyra.

Does Waskyra interact with other medicines (drug interactions)?

Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

In particular, make sure that you discuss if you are taking any medicines for HIV before or after receiving Waskyra.

This may not be a complete list of medicines that can interact with Waskyra. Always check with your health care provider.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Waskyra?

If you or someone else has used too much Waskyra, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Waskyra?

Waskyra is usually given by a health care provider in a hospital or clinic. If you miss your appointment to receive Waskyra, call to make another appointment as soon as possible.