Bluebird Bio's Lentiglobin gene therapy nears approval in Europe

The European Medicines Agency's (EMA) Committee on Human Medicinal Products (CHMP) recommended a conditional marketing approval for Bluebird Bio's Lentiglobin BB305 gene therapy for β‑thalassaemia. If approved, the new medicinal product will be marketed under the brand name Zynteglo, and will be intended for the treatment of adolescent and adult patients with transfusion-dependent β-thalassaemia (TDT) and a non-β0/β0 genotype. The treatment with Zynteglo involves the transplantation of autologous CD34+ stem cells transduced ex vivo with the BB305 lentiviral βA-T87Q-globin vector, enabling patients with TDT to produce haemoglobin at sufficient levels to allow lifelong independence from blood transfusions [ClinicalTrials.gov Identifier: NCT02906202]. Bluebird Bio's Lentiglobin BB305 gene therapy is the first ever to be recommended for approval in Europe for TDT. Final approval depends on the European Commission and is anticipated in the second quarter of 2019. A comprehensive account on the impact of this latest development on patients' lives has been discussed recently by Dr. Carsten W. Lederer, TIF Expert Advisor, here.

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