In March 2019, the Committee for Medicinal Product 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 authorization for the medicinal product Zynteglo, intended for the treatment of transfusion-dependent b-thalassemia (TDT). Since b-thalassaemia is a rare disease, Zynteglo was designed as “orphan medicine” on 24 January 2013 and was reviewed under EMA’s accelerated assessment programme. The applicant for this medicinal product is bluebird bio (Netherlands) B.V. Zynteglo has been shown to be effective at reducing the need for blood transfusion in 2 main studies in patients with beta thalassaemia who required regular blood transfusions. In these studies, out of the 14 patients who did not completely lack beta-globin and were given Zynteglo, 11 of them had sufficiently high levels of red blood cells so that they did not need blood transfusions for at least 1 year after treatment.
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