Gene Therapy Receives Recommendation from European Committee

March 29, 2019 – bluebird bio has announced that the company received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending conditional marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) for patients 12 years and older with Transfusion-Dependent β-Thalassemia (TDT) who do not have a β00 genotype. Final approval depends on the European Commission, which generally follows recommendations from the Committee on Human Medicinal Products (CHMP). If approved by the European Commission, ZYNTEGLO, formerly referred to as LentiGlobin™ for TDT, will be the first gene therapy to treat TDT.

According to a document referencing highlights from the CHMP meeting, “Since Zynteglo addresses an unmet medical need, it benefited from support within the PRIME scheme, EMA’s platform for early and enhanced dialogue with developers of promising new medicines. This interaction led to a more robust application package which allowed accelerated assessment of Zynteglo in 150 days, the fastest review time for an ATMP to date. Zynteglo was designated as an orphan1 medicine during its development.” (These highlights from the meeting can be accessed by clicking

CAF will continue to monitor this situation and inform the thalassemia community of new developments.

Secured By miniOrange