Zynteglo (betibeglogene autotemcel) is the first cell-based gene therapy for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood cell transfusions approved by the US Food and Drug Administration (FDA).
Beta-thalassemia is a type of hereditary blood disorder that causes a decrease in the blood of normal hemoglobin and red blood cells, through mutations in the beta-globin subunit, which causes insufficient oxygen in the body to result. insufficient. Reduced levels of red blood cells can lead to a variety of health problems, including weakness, dizziness, tiredness, bone changes, and more serious complications.
Transfusion-dependent beta thalassemia is the most severe form, and those affected usually require lifelong red blood cell transfusions as standard treatment. The problem is that these continuous transfusions can lead to an excessive accumulation of iron in the body that is associated with multiple health complications, including problems with the heart, liver, and other organs.
Of 41 patients with beta-thalassemia who received Zynteglo, 89% achieved transfusion independence
“Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in people who require continuous red blood cell transfusions,” said Dr. Peter Marks, director of the FDA’s Center for Biological Evaluation and Research. “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting the development of innovative therapies for patients who have limited treatment options.”
Zynteglo, a personalized treatment of beta-thalassemia
Zynteglo is administered once in a single dose, and each dose of Zynteglo is a personalized treatment made from stem cells from the patient’s own bone marrow, genetically modified to produce functional beta-globin (a component of hemoglobin).
To verify the safety and efficacy of this gene therapy, two multicenter clinical trials were conducted that included adult and pediatric patients with beta-thalassemia who required regular transfusions. The efficacy of the product was determined based on the achievement of transfusion independence, which is achieved when the patient maintains a predetermined level of hemoglobin without requiring red blood cell transfusions for a minimum of 12 months. Of 41 patients who received Zynteglo, 89% achieved transfusion independence.
Regarding the most common adverse effects related to Zynteglo, reduced levels of platelets and other blood cells, as well as mucositis, febrile neutropenia, vomiting, fever, alopecia (hair loss), epistaxis (nosebleed), abdominal pain, musculoskeletal pain were observed. , cough, headache, diarrhoea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus (itching).
Although there is a potential risk of developing blood cancer associated with this therapy, there have been no cases in studies of Zynteglo, but it is necessary to monitor the blood of patients receiving the treatment for at least 15 years to detect any signs Of cancer. In addition, patients should be monitored for hypersensitivity reactions during Zynteglo administration and should be monitored for thrombocytopenia and bleeding.
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