Frequently Asked Questions (FAQs)

Yes, ZYNTEGLO uses a patient’s own cells to treat beta-thalassemia.

ZYNTEGLO works by adding working copies of the beta-globin gene to a patient’s own cells to treat beta-thalassemia.

You will need to speak to your doctor about getting started on ZYNTEGLO. In the meantime, you can find resources at my bluebird support, where you can connect with a Patient Navigator to help support you in the treatment journey.

ZYNTEGLO is for adult and pediatric patients. In the phase 3 studies, the safety and efficacy of ZYNTEGLO was studied in patients aged 4–34 years.

The safety and efficacy of ZYNTEGLO in children less than 4 years of age have not been established. No data are available.

ZYNTEGLO treats people with beta-thalassemia who require regular red blood cell transfusions. This includes pediatrics & adults and all genotypes—β⁰/β⁰, where no beta-globin is produced, and non-β⁰/β⁰, where a reduced amount of beta-globin is produced. To find out if you are eligible for ZYNTEGLO, talk to your doctor. The safety and efficacy of ZYNTEGLO was studied in clinical trials that enrolled patients between the ages of 4 and 34 years old.

ZYNTEGLO (betibeglogene autotemcel) is indicated for the treatment of patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

The most common side effects of ZYNTEGLO on the day of treatment are increased heart rate and abdominal pain. The most common side effects of ZYNTEGLO following treatment for up to 6 months are low level of platelets, which may reduce the ability of blood to clot and may cause bleeding, low level of white blood cells, which may make you more susceptible to infection, and pain in arms or legs.

It is also important to note that these may not be all of the side effects you may experience.

There are no available data with ZYNTEGLO administration in pregnant women and there are risks associated with conditioning on pregnancy and fertility. ZYNTEGLO should not be administered to women who are pregnant and pregnancy after ZYNTEGLO infusion should be discussed with your doctor. Women of childbearing potential must use an effective method of contraception from start of cell collection through at least 6 months after administration of ZYNTEGLO. Please consult your doctor to learn more.

After receiving the chemotherapy, it may not be possible for you to become pregnant. You should consider discussing options for fertility preservation with your doctor before treatment.

The overall timing of the entire treatment process for ZYNTEGLO will vary based on coordinating patient and Qualified Treatment Center availability; for an estimated treatment timeline, visit the treatment journey page.

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 help identify potential sources of support for eligible and underinsured patients.

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.

ZYNTEGLO can only be administered at a select specialized hospital called a Qualified Treatment Center (QTC). At these hospitals, there are care teams available comprised of doctors and nurses who are experienced in treating patients with beta-thalassemia and administering bone marrow transplants, and who have been specifically trained to administer ZYNTEGLO.

The current list of Qualified Treatment Centers for ZYNTEGLO is available using the QTC Locator Tool.

The current list of Qualified Treatment Centers for ZYNTEGLO is available using the QTC Locator Tool.