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Skysona

elivaldogene autotemcel

Used for Inherited Metabolic Diseases

Skysona (elivaldogene autotemcel) is a gene therapy. It's used to treat a genetic condition called cerebral adrenoleukodystrophy (CALD) in boys ages 4 to 17 years old. As part of treatment, blood cells are taken from the body, changed into Skysona (elivaldogene autotemcel) in a lab, then infused back into the body. While Skysona (elivaldogene autotemcel) is given as a one-time infusion, the entire treatment process (including preparation and recovery) can take over 4 months. Some side effects of Skysona (elivaldogene autotemcel) includes mouth sores, nausea, and hair loss.

Reviewed by:

Ally Tsai, PharmD

Allison Gourley, PharmD

Last reviewed on October 31, 2023

Basics Side effects Pros & cons Pharmacist tips FAQs Warnings Dosage References Prices Medicare

What is Skysona (elivaldogene autotemcel)?

What is Skysona (elivaldogene autotemcel) used for?

Treatment of early stage CALD for boys between 4 to 17 years old

How Skysona (elivaldogene autotemcel) works

CALD is a genetic condition that leads to a build-up of fats in the brain and adrenal glands. As a result, CALD can affect how the brain works. This can also cause blindness, hearing loss, and a loss of communication.

Skysona (elivaldogene autotemcel) is a one-time gene therapy that's designed specifically for your child. Your child's provider will remove cells from your child's bone marrow. Then, these cells are shipped to a lab and made into Skysona (elivaldogene autotemcel) for your child. When it's finished, your child's provider will infuse Skysona (elivaldogene autotemcel) back into your child's body. There, this medication will start to break down the extra fats in their brain. This helps to slow down (or even prevent) brain problems in children with CALD.

Are you looking for information on Zynteglo (betibeglogene autotemcel) instead?

Drug Facts

Common BrandsSkysona

Drug ClassGene therapy

Controlled Substance ClassificationNot a controlled medication

Generic StatusNo lower-cost generic available

AvailabilityPrescription only

What are the side effects of Skysona (elivaldogene autotemcel)?

The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.

Common Side Effects

Lower blood cell counts (100%)
Mouth sores (92%)
Nausea (84%)
Vomiting (76%)
Fever related to low blood cell counts (73%)
Hair loss (72%)
No appetite (64%)
Belly pain (45%)
Constipation (42%)

Please note: People who receive Skysona (elivaldogene autotemcel) also receive chemotherapy as part of the treatment. Side effects listed might not be caused by Skysona (elivaldogene autotemcel) alone.

Other Side Effects

Diarrhea
Headache
Rash
Nosebleeds
Itching
Changes in skin color
Mouth and throat pain
Fast heartbeat

Serious Side Effects

Contact your healthcare provider immediately if you experience any of the following.

Seizures
Serious allergic reaction: trouble breathing, rash, swelling of lips, tongue, or throat
Severe bleeding: stomach cramps, black tarry stool, blood in the urine, any bleeding that's difficult to stop, tiredness, weakness
Serious infections: fever, cough, sore throat, tiredness, chills, body aches

Source: DailyMed

The following side effects have also been reported

Side effects that you should report to your care team as soon as possible:

Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
Bleeding—bloody or black, tar-like stools, vomiting blood or brown material that looks like coffee grounds, red or dark brown urine, small red or purple spots on skin, unusual bruising or bleeding
Infection—fever, chills, cough, or sore throat
Seizures

Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):

Constipation
Diarrhea
Loss of appetite
Headache
Nausea
Pain, redness, or swelling with sores inside the mouth or throat
Stomach pain
Vomiting

Pros and cons of Skysona (elivaldogene autotemcel)

Pros

The first and only gene therapy for CALD

Given as a single dose for one-time treatment

Safe to use in boys ages 4-17 years old

Cons

Risk of serious infections and bleeds

Treatment process can take months to complete

Might interact with vaccines or antiretroviral medications

Pharmacist tips for Skysona (elivaldogene autotemcel)

Tips for before starting Skysona (elivaldogene autotemcel) treatment:

Talk to your child's provider about all the stages of Skysona (elivaldogene autotemcel) treatment (including cell collection, conditioning, and recovery) so you and your child knows what to expect. Make sure you understand the risks and benefits of each stage before treatment starts.

Check in with your child's provider to see if your child is up-to-date on their vaccines before starting Skysona (elivaldogene autotemcel). Your child won't be able to receive vaccines starting at least 6 weeks before conditioning, which is stage of treatment where they're getting chemotherapy in order to prepare their body to receive gene therapy.

Let your child's provider know about all the medications your child is taking before they start Skysona (elivaldogene autotemcel). Some medications, such as anti-retroviral medications might affect how Skysona (elivaldogene autotemcel) works. If your child is taking these medications, their provider will likely want to hold off on treatment.

Tips for after Skysona (elivaldogene autotemcel) treatment:

Skysona (elivaldogene autotemcel) might raise your child's risk of blood cancer. To be safe, your child's provider will order blood tests at least every 6 months for 15 years after Skysona (elivaldogene autotemcel) treatment to check for changes in the blood. Talk with your child's provider if you've questions about labs and monitoring.

It might take a while for your child's platelet counts to recover after receiving Skysona (elivaldogene autotemcel). This can raise the risk for bleeding that's difficult to stop. Tell your child's provider right away if your child has symptoms of bleeding, such as abnormal bruising, blood in urine, black tarry stool, coffee ground vomit, or unusual belly pain.

Talk to a provider if you or your child has a fever, cough, chills, sore throat, runny or stuffy nose, or body aches after treatment with Skysona (elivaldogene autotemcel). This is because the medication can affect their immune system and raise your risk of developing serious infections, especially in the first 2 months.

After receiving Skysona (elivaldogene autotemcel), make sure your child doesn't donate blood, organs, tissues, or cells at any point in the future.

Frequently asked questions about Skysona (elivaldogene autotemcel)

How well does Skysona (elivaldogene autotemcel) work?

Skysona (elivaldogene autotemcel) is used in certain children with an early stage of CALD to slow down the worsening of their condition. If left untreated, CALD can result in blindness, trouble communicating, movement problems, and become life-threatening. A 2-year study showed that 72% of the children who received Skysona (elivaldogene autotemcel) didn't experience worsening of their condition to a major functional disability or death, compared to only 43% of the children in the placebo group. Please note that Skysona (elivaldogene autotemcel) isn't for everyone. If you have any questions about whether Skysona (elivaldogene autotemcel) is right for your child, follow up with your child's provider for more information.

Is Skysona (elivaldogene autotemcel) curative?

No, Skysona (elivaldogene autotemcel) isn't a cure for CALD. But it might help prevent brain problems in CALD that can typically lead to vision loss, needing tube feeding, or an inability to walk or speak. Depending on what other symptoms your child might have, their provider might recommend other treatments or medications to help relieve those symptoms. For example, your child's provider might prescribe corticosteroids to help replace the hormones if the adrenal glands aren't working properly. If you have concerns about symptoms of CALD, talk with your child's provider to determine the best treatment plan.

Is Skysona (elivaldogene autotemcel) approved by the FDA?

Skysona (elivaldogene autotemcel) was approved under the FDA's Accelerated Approval Program in September 2022. This program allows a new medication to be approved faster than usual if it fulfills an unmet need or if it treats serious or life-threatening conditions. These approvals are based on lab measurements that are thought to do a good job in predicting health benefits before the clinical trials are complete. After accelerated approval, the manufacturer still has to complete clinical studies to confirm the benefits of the medication. Depending on the results of these studies, the FDA can either give the medication a full approval or pull it off the market. Talk with your provider if you have any questions on Skysona (elivaldogene autotemcel) and its approval process.

What age is Skysona (elivaldogene autotemcel) for?

Skysona (elivaldogene autotemcel) is used in boys between 4-17 years old with mild or no symptoms of CALD. This is because CALD can get worse very quickly once symptoms are showing. Your child's provider will order a magnetic resonance imaging (MRI) to determine if this medication is right for your child.

What's the process for getting Skysona (elivaldogene autotemcel) treatment like?

Each infusion of Skysona (elivaldogene autotemcel) is made specifically for your child with their own cells. Your child's provider will first collect their stem cells about 2 months in advance. This process can sometimes take a week and they might have to go through it more than once. To be safe, their provider will also collect back-up stem cells in case there's a problem with the initial treatment. After the collection is completed, your child's stem cells are sent to a lab, where the cells are used to make Skysona (elivaldogene autotemcel) that's specific for your child. Once your child's Skysona (elivaldogene autotemcel) injection is ready, the provider will prepare your child for the infusion at the hospital.

What to expect when my child is getting Skysona (elivaldogene autotemcel)?

A few days before the infusion, your child will need to be admitted into the hospital to receive chemotherapy. This helps to make room in their bone marrow for the new cells. Then, they'll receive Skysona (elivaldogene autotemcel) as a one-time infusion through the veins. Consider planning for some extra time on the day of the infusion since the length of the infusion will depend on how many infusion bags your child's receiving. Each bag will be infused through the veins in less than 60 minutes. Your child might receive more than one bag to complete treatment. After the infusion, your child will recover in the hospital for about 2 months. This is so that their provider can make sure this medication is safe and is working for your child. Be sure to pack for your child's hospital stay after their infusion.

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What are the risks and warnings for Skysona (elivaldogene autotemcel)?

Skysona (elivaldogene autotemcel) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.

Possible risk of blood cancer

Risk factor: Receiving gene therapy (e.g., Skysona (elivaldogene autotemcel))

Some children have developed life-threatening blood cancer after treatment with Skysona (elivaldogene autotemcel). For your child's safety, the provider will monitor your child's blood work every 6 months for at least 15 years after treatment to check for blood cancer. In certain cases, the provider might also ask your child to get a bone marrow biopsy (procedure to remove a small sample of bone marrow). Speak with their provider if you have concerns about your child's risk of blood cancer. Let your child's provider know immediately if they have symptoms of blood cancer, such as feeling tired, frequent bleeding, and bruising.

Serious infection

Risk factor: Active, chronic (long-term), or recurrent infection

Your child will have a low white blood cell (WBC) count after treatment with Skysona (elivaldogene autotemcel). This can put them at risk for serious infections. Infections often occur within the first 3 months of Skysona (elivaldogene autotemcel) treatment. But in some rare cases, people have developed serious infections even 2 years after Skysona (elivaldogene autotemcel) infusion. Your child's provider will monitor your child for signs and symptoms of infection before and after treatment to make sure this medication is safe for them. Let your child's provider know if your child has developed an infection on or close to the date of their treatment; their provider might need to reschedule their infusion. Please tell your child's provider right away if you suspect that your child has an infection or if they have a fever, chills, sweat, belly pain, or sore throat.

Low blood cell counts

Skysona (elivaldogene autotemcel) can cause your child to have low blood cell counts, including platelets and WBCs. Sometimes, their blood cell counts can remain low for more than a year after receiving Skysona (elivaldogene autotemcel). This can put your child at risk for conditions such as bleeds and infections. Your child's provider will monitor their blood tests after treatment until they return to normal. In some cases, your child might receive rescue treatment with extra stem cells.

Let your child's provider know right away if your child experiences problems related to low blood cell counts, such as:

1) Low red blood cell (RBC) count: tiredness, weakness, trouble breathing, cold hands and feet
2) Low white blood cell (WBC) count: fever, headache, cough, sore throat, tiredness, body aches
3) Low platelet levels: blood in the urine, black tarry stool, coffee ground vomit, or unexplained bruising

Serious allergic reaction

Risk factor: Allergy to Rimso-50 (dimethyl sulfoxide)

Don't use Skysona (elivaldogene autotemcel) if your child has a history of an allergy to Rimso-50 (dimethyl sulfoxide), a medication used to treat painful bladder syndrome. Watch out for signs and symptoms of allergic reactions, such as hives, shortness of breath, swelling of your child's face, lips, or throat. Please get medical attention immediately if they have these signs and symptoms.

Interaction with vaccines and other medications

While we don’t completely know the effect Skysona (elivaldogene autotemcel) has on vaccines, it’s possible that it can interact with any vaccine your child gets. This is because Skysona (elivaldogene autotemcel) affects the immune system, which can make vaccines work less well. While receiving Skysona (elivaldogene autotemcel), avoid getting any vaccines at least 6 weeks before your child starts preparing for the infusion. Before your child plans to receive Skysona (elivaldogene autotemcel), talk with a provider or pharmacist about your child's vaccine history. Your child's provider can make sure all vaccines are up to date before treatment.

Certain medications for human immunodeficiency virus (HIV) should be avoided starting 1 month before and until after your child's stem cells are collected. This is because these medications can interfere with the way your child's stem cells are used in the lab to make Skysona (elivaldogene autotemcel). Typically, the provider might test your child for HIV and other viral infections before starting Skysona (elivaldogene autotemcel).

False positive for HIV

Skysona (elivaldogene autotemcel) can cause a false positive test result for HIV. This doesn't mean your child has HIV. If your child needs to have an HIV test, speak with their provider about the appropriate test to use to make sure your child gets an accurate result.

Skysona (elivaldogene autotemcel) dosage forms

Typical dosing for Skysona (elivaldogene autotemcel)

Skysona (elivaldogene autotemcel) is usually given at a qualified treatment center or in a hospital setting. Your child's provider will determine the dose of this medication based on your child's body weight. The treatment process takes several stages:

Cell collection and manufacturing: Stem cells are collected from the body and sent to a lab where they're made into Skysona (elivaldogene autotemcel) that's specific for your child. This process takes a little over 2 months.
Conditioning: Your child will receive chemotherapy to prepare their body to receive Skysona (elivaldogene autotemcel). This step takes about 1 week.
Infusion: Your child's provider will infuse Skysona (elivaldogene autotemcel) into the veins as a one-time infusion. The infusion takes about 2 hours.

After treatment, your child will need to stay in the hospital for about 2 months while their provider monitors their recovery and check for side effects.

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References

Best studies we found

bluebird bio, Inc. (2022). bluebird bio receives FDA Accelerated Approval for SKYSONA® gene therapy for early, active cerebral adrenoleukodystrophy (CALD). Business Wire.

bluebird bio, Inc. (2022). Treating early, active cerebral ALD.

bluebird bio, Inc. (2023). Skysona- elivaldogene autotemcel suspension [package insert]. DailyMed.

View All References (8)

Bryan, W. W. (2022). Accelerated BLA approval. U.S. Food and Drug Administration.

City of Hope. (2022). Blood cancer.

Genetic and Rare Diseases Information Center. (2024). X-linked cerebral adrenoleukodystrophy.

Khaddour, K., et al. (2023). Hematopoietic stem cell transplantation. StatPearls.

MedlinePlus. (2016). Stem cells.

MedlinePlus. (2022). X-linked adrenoleukodystrophy.

National Cancer Institute. (n.d.). Bone marrow biopsy.

National Organization for Rare Disorders. (2022). X-linked adrenoleukodystrophy.

GoodRx Health has strict sourcing policies and relies on primary sources such as medical organizations, governmental agencies, academic institutions, and peer-reviewed scientific journals. Learn more about how we ensure our content is accurate, thorough, and unbiased by reading our editorial guidelines.

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