Bluebird bio Inc. said the European Medicines Agency accepted the company's marketing authorization application for gene therapy LentiGlobin, which treats a blood disorder.
The Cambridge, Mass.-based clinical-stage biotechnology company has applied to market LentiGlobin as a treatment for adolescents and adults with transfusion-dependent beta-thalassemia and a non-beta0/beta0 genotype. Beta-thalassemia is an inherited blood disorder that reduces the production of hemoglobin, the protein in red blood cells that transports oxygen throughout the body. The disorder, which is caused by a mutation in the beta-globin gene, can lead to severe anemia.
Bluebird is developing LentiGlobin as a one-time gene therapy to address the underlying genetic cause of transfusion-dependent beta-thalassemia, with an aim to eliminate or reduce the need for blood transfusions.
In July, the EMA's Committee for Medicinal Products for Human Use granted accelerated assessment to LentiGlobin, potentially shortening the application's review to 150 days from 210 days.
The application was based on data from several completed, or in-progress studies of the therapy, including the phase 3 Northstar-2 HGB-207 study and the long-term follow-up study LTF-303.