Posted by Michael Wonder on 30 Mar 2019
bluebird bio receives positive opinion from CHMP for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia who do not have β0/β0 Genotype
29 March 2019 - First gene therapy recommended for approval in the EU for transfusion-dependent β-thalassemia.
- bluebird bio announced today that the CHMP of the EMA adopted a positive opinion recommending conditional marketing authorization for Zynteglo (autologous CD34+ cells encoding β A-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (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. If approved, Zynteglo formerly referred to as LentiGlobin™ for TDT, will be the first gene therapy to treat TDT.
The positive CHMP opinion is supported by efficacy, safety and durability data from the Phase 1/2 HGB-205 study and the completed Phase 1/2 Northstar (HGB-204) study as well as available data from the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies, and the long-term follow-up study LTF-303.
