Zynteglo FDA Approval History

Last updated by Judith Stewart, BPharm on Aug 18, 2022.

FDA Approved: Yes (First approved August 17, 2022)
Brand name: Zynteglo
Generic name: betibeglogene autotemcel
Dosage form: Suspension for Intravenous Infusion
Company: Bluebird Bio, Inc.
Treatment for: Beta Thalassemia

Zynteglo (betibeglogene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

• Beta-thalassemia is a rare, genetic blood disease caused by mutations in the beta-globin gene and characterized by significantly reduced or absent adult hemoglobin production. Patients with the most severe form of beta-thalassemia experience severe anemia and lifelong dependence on regular red blood cell transfusions, a lengthy process that patients typically undergo every 2-5 weeks.
• Zynteglo is a first-in-class, one-time ex-vivo LVV gene therapy that works by adding functional copies of a modified form of the beta-globin gene (β^A-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells to enable the production of a modified functional adult hemoglobin (HbA^T87Q). Once a patient has the β^A-T87Q-globin gene, they have the potential to increase Zynteglo-derived adult hemoglobin (HbA^T87Q) and total hemoglobin to normal or near normal levels that can eliminate the need for regular red blood cell transfusions. 
• Zynteglo is made specifically from a patient's own blood stem cells, which are collected by a process called mobilization and apheresis. The blood stem cells are then sent to a manufacturing site where they are used to make Zynteglo.
• Zynteglo is administered via intravenous infusion. Each infusion bag is administered over a period of less than 30 minutes.
• Warnings and precautions associated with Zynteglo include delayed platelet engraftment, risk of neutrophil engraftment failure, risk of insertional oncogenesis, and hypersensitivity reactions.
• Common non-laboratory adverse reactions include mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nose bleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus (itch). Common Grade 3 or 4 laboratory abnormalities include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.

Development timeline for Zynteglo

Further information

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