INDICATION1
ZYNTEGLO is indicated for the treatment of patients 12 years and older with TDT who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)–matched related HSC donor is not available.
Full common name: A genetically modified autologous CD34+ cell–enriched population that contains haematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene.1
*In clinical trials, transfusion independence was defined as a weighted average haemoglobin (Hb) of ≥9 g/dL with no red blood cell (RBC) transfusions for a continuous period of ≥12 months at any time during the study after infusion of ZYNTEGLO.1
†As the Phase 3 trial is ongoing, data are for patients who are currently evaluable for assessment of transfusion independence.1