FDA grants priority review to betibeglogene autotemcel for beta-thalassemia
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The FDA granted priority review to betibeglogene autotemcel, a gene therapy in development to treat patients with transfusion-dependent beta-thalassemia.
If approved, the investigational therapy would be the first ex vivo hematopoietic stem cell gene therapy indicated for use in the United States.
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.
“The FDA’s acceptance of our [biologics license application] for beti-cel brings us one step closer to potentially providing a one-time treatment that can address the underlying cause of beta-thalassemia and offer patients freedom from regular transfusions,” Andrew Obenshain, chief executive officer at bluebird bio, said in a company-issued press release.
The FDA previously granted orphan drug and breakthrough therapy designations to beti-cel.
Its priority review designation is based on results of a long-term follow-up study (LTF-303), two phase 3 trials (Northstar-2 and Northstar-3), and a phase 1/phase 2 study (Northstar). As of March 9, 63 patients had been treated on the trials with follow-up of 220 patient-years.
Data from all three Northstar 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. Eighty-nine percent of patients in the phase 3 trials stopped receiving blood transfusions, and 85% achieved transfusion independence.
“For too long, people with beta-thalassemia who rely on regular transfusions have had to live with extraordinary burdens associated with their disease,” Anne-Virginie Eggimann, chief regulatory officer at bluebird bio, said in the release. “Beti-cel works uniquely to help patients produce adult hemoglobin at normal or near-normal levels, which can eliminate their need for chronic transfusions and chelation that only temporarily relieve the symptoms of anemia and are associated with serious health risks and reduced quality of life.”
Reference:
Hongeng S, et al. Abstract 1. Presented at: The 2021 TCT Meetings Digital Experience (virtual meeting); Feb. 8-12, 2021.
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