Bluebird Bio’s Zynteglo Receives CHMP’s Positive Opinion for Conditional Marketing for Patients with Transfusion-Dependent β-Thalassemia (TDT) without β0/β0 Genotype

Bluebird Bio’s Zynteglo Receives CHMP’s Positive Opinion for Conditional Marketing for Patients with Transfusion-Dependent β-Thalassemia (TDT) without β0/β0 Genotype

Shots:

  • The positive opinion is based on P-I/II HGB-205 & Northstar (HGB-204) and P-III Northstar-2 (HGB-207) & Northstar-3 (HGB-212) studies assessing Zynteglo in patients with transfusion-dependent β-thalassemia (TDT) who do not have β0/β0 genotype aged 12 yrs. and older
  • Collective data from studies: 80% achieved transfusion independence with median duration of 38 mos.; @6mos. median (min – max) total hemoglobin for patients was 11.9 g/dL
  • Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) is a gene therapy, formerly known as LentiGlobin targeted for TDT and reviewed under EMA’s PRIME and Adaptive Pathways programs with its expected European Commission approval in Q2’19

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