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.

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