EU regulators reviewing bluebird bio's filing for LentiGlobin gene therapy in beta thalassaemia

Bluebird bio said Friday that the European Medicines Agency accepted a marketing application seeking approval of its experimental LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassaemia (TDT) and a non-β0/β0 genotype. The company noted that the filing will be reviewed under accelerated assessment, which was granted in July.

David Davidson, chief medical officer of bluebird bio, commented "the acceptance of our marketing authorisation application for LentiGlobin is a milestone that advances us toward our goal of providing to patients the first one-time gene therapy that addresses the underlying genetic cause of TDT."

The application for LentiGlobin is based on data from two Phase I/II studies, as well as available results from the Phase III Northstar-2 trial and a long-term follow-up study. Data reported in June showed that in the Northstar-2 study, seven of eight patients with TDT and non-β0/β0 genotypes exhibited HbAT87Q levels of at least 7.6 g/dL and total haemoglobin levels of 11.1 g/dL to 13.3 g/dL by six months. At the time, the drugmaker added that all seven patientshad remained transfusion-free for 4.7 months to 15.1 months.

LentiGlobin is designed to be a one-time gene therapy to address the underlying genetic cause of TDT, which "could eliminate or reduce the need for blood transfusions," the company said.

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