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August 18, 2022

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

The approval is based on findings from two Phase III clinical trials and a long-term follow-up study of Zynteglo.

The US Food and Drug Administration (FDA) has granted approval for bluebird bio’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta‑thalassemia in adult and paediatric patients.

A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.

The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study.

The open-label, single-arm, 24-month Phase III trials enrolled 41 subjects of the age four to 34 years with non-β0/β0 and β0/β0 genotypes.

Findings showed that 89% of evaluable subjects attained transfusion independence (TI). 

TI is described as no longer requiring RBC transfusions for a minimum of 12 months while continuing to have a weighted average total haemoglobin of a minimum of 9g/dL. 

An ex-vivo lentiviral vector (LVV) gene therapy, Zynteglo’s every dose is made by genetically modifying the bone marrow stem cells of the patient to produce functional beta-globin.

bluebird bio CEO Andrew Obenshain said: “The FDA approval of Zynteglo offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation and unlocks new possibilities in their daily lives. 

“After more than a decade of research and clinical development, and through the perseverance of clinicians, patients and their families, the approval of Zynteglo marks a watershed moment for the field of gene therapy.”

A genetic blood ailment, beta-thalassemia is caused by beta-globin gene mutations and results in a substantial decline or lack of production of adult haemoglobin. 

In June 2019, the European Commission (EC) granted conditional marketing authorisation for Zynteglo to treat transfusion-dependent beta-thalassemia.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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