Pharmacy Bulletin

Pharmacy Bulletin

We share important prescription drug information to help you stay informed about updates concerning particular prescription medicines.

VativoRx Bottle update


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.