On Aug. 17, 2022, the U.S. Food and Drug Administration (FDA) approved bluebird bio’s Zynteglo® (betibeglogene autotemcel, beti-cel). It’s a personalized stem cell-based gene therapy to treat adult and pediatric patients who have transfusion-dependent beta-thalassemia. The drug is manufactured using the patient’s own stem cells, which are collected and altered to include a modified form of the β-globin gene. Following bone-marrow conditioning therapy, Zynteglo is administered as a single intravenous (IV) infusion by specially trained providers in a Qualified Treatment Center (QTC), which are certified transplant centers that have experience with beta-thalassemia and with delivering cell and gene therapies. bluebird bio announced that it will price Zynteglo at $2.8 million. Full prescribing information can be found here.
At a Glance
- Brand Drug: Zynteglo (betibeglogene autotemcel)
- Manufacturer: bluebird bio, Inc.
- Date Approved: Aug. 17, 2022
- Indication: beta-thalassemia that requires regular red blood cell transfusions
- Dosage Forms Available: IV (one time infusion)
- Launch Date: The first patient apheresis is expected in 4th quarter 2022
- Estimated Cost: $2.8 million (one-time infusion)
- Beta-thalassemia is a rare, genetic disease caused by a mutation in the beta-globulin gene, causing ineffective red blood cell production which can result in anemia.
- Patients who have its most severe form may require blood transfusions every two weeks to five weeks to maintain normal hemoglobin levels.
- However, chronic transfusions also carry risks, including iron overload, which can result in organ damage.
- bluebird bio estimates that approximately 1,300 to 1,500 patients in the U.S. have transfusion-dependent beta-thalassemia. About 850 of them may be eligible to receive this gene therapy.
- Manufacturing Zynteglo requires time, taking 70 to 90 days from when the patient’s stem cells are collected (via apheresis) to when it is ready to infuse into the patient.
- In clinical trials that included 41 patients who had transfusion-dependent beta-thalassemia that was treated with Zynteglo, 89% of patients no longer needed blood transfusions to maintain a hemoglobin level of at least 9g/dL when followed for at least 12 months.
- Among the side effects experienced by 20% or more of patients were fever, hair loss, headaches, itching and vomiting. Around one-half of patients had low levels of blood components such as red and white blood cells, platelets and lymph.
- Zynteglo is a designated Breakthrough Therapy and Orphan Drug. It also received a Priority Review Voucher, which can be used to accelerate approval of a future FDA submission.