bluebird bio submits BLA for single-dose gene therapy to treat beta-thalassemia
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The FDA received a completed rolling biologics license application for betibeglogene autotemcel, a gene therapy in development to treat patients with transfusion-dependent beta-thalassemia.
The application seeks approval of the therapy for adults and pediatric patients of all genotypes who require frequent red blood cell transfusions.
Betibeglogene autotemcel (bluebird bio) — also known as beti-cel — is an autologous gene therapy comprising CD34-positive hematopoietic stem cells from mobilized peripheral blood that are harvested by apheresis after plerixafor mobilization and transduced with a BB305 lentiviral vector. The one-time therapy adds functional copies of a modified form of the beta-globin gene into a patient’s hematopoietic stem cells.
“With this submission, we are one step closer to bringing a potentially transformative gene therapy to people living with [transfusion-dependent beta-thalassemia] and their families,” Andrew Obenshain, president of severe genetic diseases for bluebird bio, said in a company-issued press release.
“At bluebird bio, we have a deep understanding of gene therapies, built over a decade of research and development in severe genetic diseases,” Obenshain added. “We look forward to working with the FDA on its review of this BLA as we realize the promise that one-time gene therapies hold for patients.”
The FDA previously granted orphan drug and breakthrough therapy designations to beti-cel. If approved, it would be the first ex vivo hematopoietic stem cell gene therapy indicated for use in the United States, according to the manufacturer.
The BLA is based on results of two phase 3 trials (Northstar-2 and Northstar-3) and a phase 1/phase 2 study (Northstar). As of March 9, 63 patients have been treated on the trials and follow-up had reached 220 patient-years.
Data from all three studies presented at this year’s TCT Meetings Digital Experience showed most patients who received beti-cel achieved transfusion independence for at least 1 year.
Results from the phase 1/phase 2 study showed 77% of patients stopped receiving blood transfusions, and 64% of patients achieved transfusion independence for 1 year or longer. Eighty-nine percent of patients in the phase 3 trials stopped receiving blood transfusions, and 85% achieved transfusion independence.
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