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Beta-thalassemia

FDA-approved Zynteglo: Cell-based gene therapy for beta-thalassemia

  • August 22, 2022
  • < 1 min read

The FDA approved the first cell-based therapy on 17 August 22 for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. 

Beta-thalassemia is a kind of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells (RBCs) in the blood through mutations in the beta-globin subunit, resulting in insufficient delivery of oxygen to the body. The decreased levels of red blood cells can lead to several health problems such as dizziness, weakness, fatigue, bone abnormalities, and more severe complications. 

The medicine Zynteglo contains betibeglogene autotemcel, an autologous hematopoietic stem cell-based gene therapy. Zynteglo is prepared from the patient’s HSCs (hematopoietic stem cells) and collected by the apheresis procedure. This medicine comes in the form of cell suspension single-dose infusion bags for intravenous infusion. 

Zynteglo works by adding functional copies of a modified β-globin gene into patients’ hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells with BB305 LVV. After ZYNTEGLO infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate to produce RBCs containing biologically active βA-T87Q-globin that will combine with α-globin to produce functional adult Hb containing βA-T87Q-globin (HbAT87Q). 

The common side-effects of Zynteglo are: 

  • Reduced platelet and other blood cell levels and mucositis
  •  Febrile neutropenia
  •  Vomiting, pyrexia (fever)
  •  Alopecia (hair loss)
  •  Epistaxis (nosebleed)
  •  Abdominal pain
  •  Musculoskeletal pain
  •  Cough
  •  Headache
  •  Diarrhea
  •  Rash
  •  Constipation
  •  Nausea
  •  Decreased appetite
  •  Pigmentation disorder
  •  Pruritus (itch)

There is a potential risk of blood cancer related to this treatment; however, no cases have been seen in the studies of Zynteglo. One should monitor the blood of patients who receive Zynteglo for at least 15 years for any evidence of cancer. Patients should also be monitored for hypersensitivity reactions during Zynteglo administration, thrombocytopenia, and bleeding. 

Source: https://www.fda.gov/media/160991/download

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