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Yes. SKYSONA is a type of gene therapy called gene addition.

SKYSONA is made specifically for each patient using the patient’s own blood stem cells, and adds functional copies of the ABCD1 gene to their cells. This addition allows the child’s body to make ALD protein (ALDP), which helps their body to break down very long–chain fatty acids (VLCFAs) in the brain. This process slows the progression of damage to the brain and slows the decline in neurologic function.

Yes. SKYSONA was approved by the FDA on September 18, 2022.

The most common side effects of SKYSONA on the day of treatment are nausea, vomiting, decreased appetite, constipation, abdominal pain, headache, and rash. The most common side effects of SKYSONA following treatment are low blood counts, which may lead to a risk of bleeding and/or infection, blood cancer, life-threatening infections, and inflamed and painful mouth. It is important to note that these may not be all of the side effects you may experience.

SKYSONA may cause cancer of the blood and bone marrow, which can be life-threatening and lead to death. Blood cancer has resulted from treatment with SKYSONA because cancer-causing genes have been turned on by the gene therapy. Patients have developed cancer as early as one year after SKYSONA administration, prior to SKYSONA having time to potentially help their CALD. Blood cancer can also take years to develop, and has been diagnosed as late as 7.5 years later. Because SKYSONA was approved while patients were still being evaluated for the risk of cancer, the percent of patients who will develop cancer and the maximum timeframe when cancer caused by SKYSONA could develop is unknown. Blood cancer is usually treated with chemotherapy and may require stem cell transplant. Stem cell transplant using cells from a donor have been used to treat children diagnosed with blood cancer that was caused by SKYSONA. Blood cancers, including those following SKYSONA treatment, will lead to death if not treated. Treatment of blood cancer has led to death due to complications. Because of the risk of cancer caused by SKYSONA, your doctor may recommend that you are evaluated by a hematologist to determine if you have underlying risk factors that could further increase your risk for cancer and change whether SKYSONA is appropriate for you.

Before you are treated with SKYSONA, you should have a detailed discussion with your doctor about the risks and benefits of SKYSONA and alternative treatment options. Alternative treatment may include an allogeneic hematopoietic stem cell transplant. Discuss with your physician the possibility of a stem cell transplant using cells from a suitable and willing matched sibling donor if one is available.

Because of the risk of cancer, it is important for you to be monitored lifelong. At a minimum we recommend blood tests every 3 months for 15 years. Blood tests will look at your blood cell counts and the locations in your blood cells where the gene therapy is inserted. If your blood counts are too low or too high, or if you have lots of cells with the same gene therapy insertion sites, additional testing may be recommended. Additional testing might include more frequent blood tests to watch you more closely for changes in your blood. Additional testing could also include a bone marrow evaluation, which can tell your doctor more than blood tests about the health of your bone marrow and if there is cancer forming.

If blood cancer develops quickly or if you have not been having the recommended blood or bone marrow tests, you might experience symptoms of cancer before it is diagnosed. You or your caregiver should call your healthcare provider right away for any of these signs or symptoms:

• Abnormal bruising or bleeding (including nosebleed)
• Blood in urine, stool, or vomit
• Coughing up blood
• Severe headache
• Unusual stomach or back pain
• Fever (100.4°F/38°C or higher)
• Swollen glands
• Abnormal tiredness

If you are diagnosed with a cancer, have your treating physician contact bluebird bio at 1-833-999-6378.

SKYSONA may cause life-threatening allergic reactions as it contains DMSO, a commonly used preserving agent. Please inform your healthcare provider if you have been told that your child has a DMSO allergy or has experienced a reaction after receiving a DMSO-containing product.

It is important that you or your caregiver tell your healthcare providers that you have received SKYSONA.

Do not donate blood, organs, tissues or cells.

There is a risk of blood cancer following treatment with SKYSONA which will require lifelong monitoring. You will be monitored at least every 3 months for a minimum of 15 years for this.

Possible or reasonably likely side effects when treated with SKYSONA are:

• While receiving chemotherapy to prepare your body for SKYSONA:
• Nausea, vomiting, decreased appetite, constipation, abdominal pain
• Headache
• Rash
• On the day of treatment with SKYSONA:
• Life-threatening allergic reaction
• Nausea, vomiting
• Following treatment:
• Blood cancer. Refer to “What is the most important information I or my caregiver should know about SKYSONA?”
• Low blood counts leading to a risk of bleeding and/or infection. Until your blood counts (platelets, white blood cells, red blood cells) return to safe levels, you may be treated with blood and platelet transfusions and other medicines that prevent bleeding and infection by increasing your blood counts. Most patients’ blood counts return to safe levels in about one month after treatment with SKYSONA. Some patients’ blood counts may not recover for >1 year. Tell your healthcare provider right away if you get a fever, are feeling tired, or have easy bleeding or bruising.
• Life-threatening infections. Patients treated with SKYSONA may experience serious or life-threatening infections, including infections of the bloodstream by bacteria or viruses. Infections often occur in the first 1 or 2 months after treatment with SKYSONA, but can occur >1 year later. Tell your healthcare provider right away if you develop fever, chills, or any signs or symptoms of an infection.
• Inflamed and painful mouth (this typically occurs during the first 2 months after SKYSONA treatment)
• Nausea, vomiting, decreased appetite, constipation, abdominal pain, diarrhea
• Headache
• New onset seizures

These are not all the possible side effects of SKYSONA. Your healthcare providers may give you other medicines to treat your side effects. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or to bluebird bio at 1-833-999-6378.

The one-time treatment process for SKYSONA is carefully coordinated at a Qualified Treatment Center and includes a pre-treatment, treatment, and post-treatment period.

Yes. SKYSONA is a one-time gene therapy treatment.

The current list of Qualified Treatment Centers for SKYSONA is available using the QTC Locator Tool. We are currently working to activate and train more centers on administering SKYSONA, so please check for updates.

my bluebird support can help support you through a benefits investigation. This explores your coverage options and what out-of-pocket costs you may be responsible for, including copays. my bluebird support can also collaborate with your health insurance provider and your doctor’s office staff by offering guidance and answers to coverage questions.

my bluebird support can help support you through a benefits investigation. This explores your coverage options and what out-of-pocket costs you may be responsible for, including copays. my bluebird support can also collaborate with your health insurance provider and your doctor’s office staff by offering guidance and answers to coverage questions.

Establish regular check-ins with the child’s doctor to keep monitoring their cerebral ALD. As a caregiver, you can talk to your child’s doctor about how your child’s recovering, focusing on both their physical and mental recovery.

PHYSICAL RECOVERY
The goal is that children will live an active life after treatment with SKYSONA. That said, it’s important to always discuss with your child’s doctor when they may be able to return to physical activity and what physical activity would be appropriate. Ask your provider if your child should stay away from certain sports where head injuries are more common.

MENTAL RECOVERY
Keep in mind that your child’s mental health is also important when receiving gene therapy for cerebral ALD. Please talk with your child’s doctor about concerns you may have regarding their mental health.

LIFELONG MONITORING
Your child will also have lifelong monitoring for blood cancer and will continue to be monitored for cerebral adrenoleukodystrophy.

Work with your child’s doctor to establish regular follow-up care. As a caregiver, you can talk with your child’s doctor about how your child is recovering, focusing on both their physical and mental recovery.

15-YEAR REGISTRY
By enrolling your child in the 15-year registry study, your child will be monitored. You are encouraged to continue lifelong follow-up monitoring at least every six months for your child after the registry ends.

Cerebral ALD treatments have relied on the transplantation of donor blood stem cells. The idea is that the donor cells give a body functioning copies of the ABCD1 gene that will allow the body to produce ALD protein (ALDP), the protein needed to prevent the buildup of VLCFAs. However, donor blood stem cell transplants require something very important—the availability and willingness of a donor.

The best option for a stem cell donor is a child’s sibling, called a matched sibling donor (MSD). The better the match from a sibling donor, the less risk of immunologic complications (infection, graft-versus-host disease). However, MSDs are only available for about 11% of children, leaving many people having to find a donor through the bone marrow registry.

Cerebral adrenoleukodystrophy (ALD) is a genetic disease that progresses from ALD and affects many important functions of a person’s brain. If left untreated, cerebral ALD can result in major functional disabilities (MFDs) and early death.

ALD is caused by a genetic mutation in a specific gene labeled ABCD1. This mutation prevents a person’s body from producing a specific protein that’s needed to break down very long-chain fatty acids (VLCFAs).

Major functional disabilities are a categorization of 6 severe disabilities commonly attributed to cerebral ALD (loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement).

VLCFAs are the building blocks of fat and contribute to important body functions like the forming of the skin barrier or assisting how kidneys operate. If VLCFAs are not broken down, a harmful buildup occurs in the brain, which can destroy the protective covering around nerve cells and cause damage to the brain. When a person progresses from ALD to cerebral ALD it is caused by a buildup of VLCFAs in the brain.