bluebird bio gets CHMP nod for gene therapy Zynteglo

A European Medicines Agency advisory panel has recommended a conditional approval for Zynteglo, bluebird bio’s lead gene therapy for thalassemia, a rare blood disorder. The Committee for Medicinal Products for Human Use (CHMP) confirmed on Friday that it had issued a positive opinion for the therapy – previously known as LentiGlobin – as a treatment for patients aged 12 years and older with transfusion-dependent beta-thalassemia (TDT) who do not have a specific β0/β0 genotype. The announcement came after a couple of medical charities jumped the gun and prematurely issued notices of the panel’s endorsement last week, hailing it as a major advance for patients with TDT who typically need frequent blood transfusions to manage the disease. The positive opinion for Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) puts bluebird bio on course for its first gene therapy approval in the coming weeks and could give thalassaemia patients a new one-shot treatment option that tackles the underlying genetic cause of the disease.