FDA's breakthrough therapy designation to LentiGlobin for treatment of beta-thalassemia major

By William Chin, PhD, Scientific Coordinator, EUCRAF

Yesterday, the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to LentiGlobin® BB305 for the treatment of transfusion-dependent patients with beta-thalassemia major. LentiGlobin BB305 works by inserting a functional human beta-globin gene into the patient's own hematopoietic stem cells ex vivo and then returning those modified cells to the patient through an autologous stem cell transplantation. It is a drug product from the company bluebird bio, Inc that has operations in Cambridge, Massachusetts, Seattle, Washington, and Paris, France. 

The FDA's Breakthrough Therapy designation is supported by data from the ongoing global clinical study for the treatment of beta-thalassemia major Phase 1/2 study, called the Northstar Study; and a single-center Phase 1/2 study in France (HGB-205).

Thalassaemia major is a disorder of the haemoglobin molecule inside the red blood cells. It is an inherited genetic disease and approximately 7% of the global population is a carrier for Haemoglobin disorders.


1. bluebird bio News Release, 2 February 2015, FDA Grants Breakthrough Therapy Designation to LentiGlobin for Treatment of Beta-Thalassemia Major

2. Thalassaemia International Federation (TIF)