NEJM Publishes Promising Bluebird Bio Gene Therapy Data in Beta Thalassemia

Rare Daily Staff

Interim data published in the New England Journal of Medicine from two separate studies of Bluebird Bio’s LentiGlobin, a gene therapy to treat transfusion-dependent beta thalassemia, showed the majority of patients have not required transfusions for two years or longer following treatment.

Transfusion-dependent beta thalassemia is a severe genetic disease characterized by reduced or absent hemoglobin levels that results in severe anemia and ineffective red blood cell production. People with the condition require a lifelong regimen of chronic blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions.

LentiGlobin is being developed to treat both transfusion-dependent beta thalassemia and severe sickle cell disease.

The publication included data from two studies. The first study HGB-204, which recently was completed, and HGB-205, which is ongoing. Both are evaluating the safety and efficacy of one-time treatment with LentiGlobin gene therapy and the interim results showed that a majority of the 22 patients in the two phase 1/2 studies followed for two years or longer remained free from transfusions.

“These interim data demonstrate the potential of LentiGlobin gene therapy to address the underlying genetic cause of TDT and increase production of functional red blood cells,” said Dave Davidson, chief medical officer, Bluebird Bio. “Nearly all patients in the two studies with a non-β⁰/β⁰genotype achieved freedom from chronic blood transfusions and, importantly, several of these patients reached normal or near-normal total hemoglobin levels and sustained those levels throughout the interim study period.”

The U.S. Food and Drug Administration and the European Medicines Agency granted LentiGlobin Orphan Drug status for the treatment of beta thalassemia and sickle cell disease. The FDA granted Breakthrough Therapy designation to LentiGlobin for the treatment of transfusion-dependent patients with β-thalassemia major and Fast Track designation for the treatment of beta-thalassemia major and for the treatment of certain patients with severe sickle cell disease.

Bluebird bio is participating in the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines. The EMA granted Priority Medicines (PRIME) eligibility to LentiGlobin for the treatment of transfusion-dependent beta thalassimia.

April 18, 2018
Photo: Dave Davidson, chief medical officer, Bluebird Bio