Zynteglo first gene therapy approved for transfusion-dependent β-thalassemia

The European Commission (EC) has granted conditional marketing authorization for gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene).

It is indicated for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

The drug’s developer, USA-based bluebird bio (Nasdaq: BLUE) - whose share rose as much as 4% on the news, says it will continue the country-by-country reimbursement process to help ensure access to Zynteglo (previously known as LentiGlobin) for appropriate patients in Europe.