In two phase 3 studies, about 9 out of 10 (89%; 32/36 patients) treated with ZYNTEGLO achieved transfusion independence and had a normal or near-normal median total hemoglobin of 11.5 g/dL.*†
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Who is ZYNTEGLO for?
ZYNTEGLO is for adult and pediatric patients with beta-thalassemia who require regular blood 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. ZYNTEGLO was evaluated in 41 patients aged 4 to 34 years. Of these 41 patients, 36 were evaluable for TI. Patients were evaluable if they had completed 24 months of follow-up in the phase 3 study, or achieved TI, or won’t achieve TI in the phase 3 study. Total hemoglobin levels presented about ZYNTEGLO reflect weighted average total hemoglobin during transfusion independence, which is defined as the average across hemoglobin levels taken at each patient visit during the clinical study, factoring in a weighting based on the time duration between study visits.†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*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. ZYNTEGLO was evaluated in 41 patients aged 4 to 34 years. Of these 41 patients, 36 were evaluable for TI. Patients were evaluable if they had completed 24 months of follow-up in the phase 3 study, or achieved TI, or won’t achieve TI in the phase 3 study. Total hemoglobin levels presented about ZYNTEGLO reflect weighted average total hemoglobin during transfusion independence, which is defined as the average across hemoglobin levels taken at each patient visit during the clinical study, factoring in a weighting based on the time duration between study visits.†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
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