CHMP recommends approval of Zynteglo for beta-thalassaemia

The Committee for Medicinal Products for Human Use (CHMP) on the basis of the draft Committee for Advanced Therapies opinion, adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Zynteglo (gene therapy), previously known as Lentiglobin, from bluebird bio, intended for the treatment of transfusion-dependent beta-thalassaemia (TDT). As Zynteglo is an advanced therapy medicinal product, the CHMP’s positive opinion is based on an assessment by the Committee for Advanced Therapies. Zynteglo was designated as an orphan medicinal product on 24 January 2013 and was reviewed under EMA’s accelerated assessment programme.

Zynteglo is a gene therapy medicinal product that adds functional copies of a modified beta-globin gene into the patients’ haematopoietic stem cells through transduction of autologous CD34+ cells with BB305 lentivirus vector, thereby addressing the underlying genetic cause of the disease. The full indication is: “treatment of patients 12 years and older with transfusion-dependent beta-thalassaemia (TDT) who do not have a beta0/beta0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.