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Bluebird Bio gene therapy for blood disorder gets priority review in Europe

Bluebird Bio Inc. said the European Medicines Agency granted accelerated assessment to LentiGlobin, its gene therapy for a certain blood disorder.

The designation from the EMA's Committee for Medicinal Products for Human Use covers the treatment of adolescents and adults with transfusion-dependent beta thalassemia, or TDT, and a certain non-beta genotype.

"Receiving accelerated assessment for LentiGlobin helps support our goal of delivering the first gene therapy to patients with TDT," Bluebird Bio Chief Medical Officer David Davidson said in a news release.

Bluebird Bio plans to file LentiGlobin's marketing authorization application with the EMA in 2018. Accelerated assessment can reduce the application's review from 210 days to 150 days.

The Cambridge, Mass.-based company is evaluating LentiGlobin in phase 2 and phase 3 studies.

LentiGlobin previously received priority medicines and orphan medicinal product designations from the EMA. The U.S. Food and Drug Administration has also granted LentiGlobin orphan drug status and breakthrough therapy status.

Patients with TDT cannot produce enough oxygen-carrying hemoglobin, and require a lifetime of blood transfusions, which could cause complications such as organ failure from iron overload. LentiGlobin is meant to be a one-time treatment to address TDT's underlying genetic cause.