Alexis Thompson, MD, MPH, on Qualifying Treatment Centers for Beti-Cel in Thalassemia

“Many of these qualified treatment centers are in fact, the centers with substantial experience in collecting stem cells from individuals with hemoglobin disorders like thalassemia. We believe that that really is going to be key to see success. Clearly, we'd like as many patients to benefit as possible. I think that there may very well be a point in time where more centers join that group.”

The FDA has approved bluebird bio’s ZYNTEGLO (betibeglogene autotemcel (beti-cel), a gene therapy for adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions (transfusion dependent thalassemia; TDT). The therapy will become available for treatment at qualifying treatment centers (QTCs) that are experts with stem cells, TDT, and gene therapies.

Beti-cel is an autologous gene-edited, lentiviral cell therapy that delivers a modified form of the β-globin gene into patients’ hematopoietic stem cells and then back into the patient. The therapy enables normalized production of hemoglobin without regular RBC transfusions.

The approval is based off positive data from multiple studies, including the ongoing phase 3 HGB-207 study (NCT02906202) in which 20 of 22 evaluable patients (91%), including 6 of 7 patients (86%) younger than 12 years of age, reached transfusion independence.

CGTLive spoke with Alexis Thompson, MD, MPH, primary investigator and chief, division of hematology, Children’s Hospital of Philadelphia, to learn more about the QTCs and the required expertise needed to successfully administer beti-cel.

REFERENCE

FDA approves first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News release. FDA. August 17, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-treat-adult-and-pediatric-patients-beta-thalassemia-who?utm_medium=email&utm_source=govdelivery