EMA accepts bluebird bio MAA

US biotech bluebird bio (Nasdaq: BLUE) has had its marketing authorization application (MAA) accepted by the European Medicines Agency (EMA) for its investigational LentiGlobin gene therapy for the treatment of adults and adolescents with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.

LentiGlobin was previously  granted an accelerated assessment by the Committee for Medicinal Products for Human Use of the EMA in July of this year, potentially reducing the agency’s active review time of the MAA from 210 days to 150 days.

TDT is an inherited blood disorder which can lead to serious complications and organ damage. LentiGlobin is a potential one-time gene therapy that could address the underlying genetic cause of TDT.