LentiGlobin for the treatment of transfusion-dependent beta-thalassemia and a non-beta0/beta0 genotype, was granted an accelerated assessment by the CHMP.- bluebird bio Inc.

bluebird bio, Inc. announced that its investigational LentiGlobin gene therapy for the treatment of adolescent and adult patients with transfusion-dependent beta-thalassemia (TDT) and a non-beta0/beta0 genotype, was granted an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), for its upcoming marketing authorization application (MAA).

LentiGlobin is a potential one-time gene therapy that may address the underlying genetic cause of TDT. bluebird bio intends to file an MAA for LentiGlobin in TDT with the EMA in 2018. Accelerated assessments can reduce the active review time of an MAA from 210 days to 150 days once it has been validated by the EMA. An accelerated assessment is granted to products deemed by the CHMP to be of major interest for public health and represent therapeutic innovation.