Lifelong transfusions treat the symptoms of beta-thalassemia but do not address the underlying genetic cause
ZYNTEGLO is a gene therapy that uses gene addition to help your body produce functional adult hemoglobin, potentially eliminating the need for regular transfusions.
Specifically made for each person, ZYNTEGLO uses a person’s own blood stem cells and adds working copies of the beta-globin gene (needed to create functional adult hemoglobin) to the person’s cells. This may allow the person to produce sufficient hemoglobin.
Who is ZYNTEGLO for?
ZYNTEGLO is a one-time gene therapy to treat beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions
- β0/β0: no beta-globin is produced
- Non-β0/β0: a reduced amount of beta-globin is produced
*In the phase 3 studies, the safety and efficacy of ZYNTEGLO was studied in patients aged 4–34 years.
†Made using the patient’s own stem cells.
Topics to explore:
In two phase 3 studies, about 9 out of 10 (89%) patients treated with ZYNTEGLO stopped transfusions
Transfusion independence (TI) meant that patients achieved a weighted average hemoglobin of ≥9 g/dL without any transfusions for a continuous period of ≥12 months at any time during the study after infusion of ZYNTEGLO.
The majority of patients achieved transfusion independence (89%; 32/36 patients) and had a normal or near-normal median total hemoglobin of 11.5 g/dL.‡
‡Normal Hb ranges for healthy individuals from the American College of Clinical Pharmacy:
- 2–9 years: 11.5–14.5 g/dL
- Males 10–17 years: 12.5–16.1 g/dL
- Females 10–17 years: 12–15 g/dL
- Male adults (≥18 years): 13.5–18 g/dL
- Female adults (≥18 years): 12.5–16 g/dL
The phase 3 studies evaluated the efficacy of ZYNTEGLO in 41 patients aged 4 to 34 years. Of these 41 patients, 36 were evaluable for transfusion independence.
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