BLA for Beti-Cel in B-thalassemia Accepted for Priority Review by FDA

The newly accepted FDA approval application for betibeglogene autotemcel is supported by 5 studies of the agent across all ages of patients. Eighty-nine percent of evaluable patients achieved transfusion independence.

The FDA has accepted the biologics license application for betibeglogene autotemcel (beti-cel), a one-time gene therapy for patients with B-thalassemia who require regular red blood cell transfusions, according to a press release by Bluebird bio, Inc.

Beti-cel’s mechanism of action is adding functional copies of a modified form of B-globin in a patient’s stem cells in order to correct deficiently of adult hemoglobin. Once the B-globin gene is acquired at certain levels, it may eliminate the need for transfusions.

“For too long, people with β-thalassemia who rely on regular transfusions have had to live with extraordinary burdens associated with their disease. 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,” said Anne-Virginie Eggimann, chief regulatory officer, of bluebird bio, in a press release. “This BLA acceptance represents the culmination of contributions from many, including the patients involved in the clinical program, their caregivers, and the study investigators. We look forward to working closely with the FDA to bring this treatment to patients in need.”

The application is supported by data from 5 separate studies, the phase 3 Northstar-2 (NCT02906202) and Northstar-3 (NCT03207009) studies, and the phase 1/2 studies Northstar (NCT01745120) and HGB-205 (NCT02151526), and the long-term LTF-303 (NCT02633943). As of March 9, 2021, 63 pediatric, adolescent, and adult patients. Additionally, long-term efficacy and safety results are available in 2 patients who more than 7 years of follow-up.

In the phase 3 studies, 89% of evaluable patients, including pediatric patients and those with the most severe genotypes, achieved transfusion independence. Transfusion independence was defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average hemoglobin of at least 9 g/dL.

“The FDA’s acceptance of our BLA for beti-cel brings us one step closer to potentially providing a one-time treatment that can address the underlying cause of β-thalassemia and offer patients freedom from regular transfusions,” said Andrew Obenshain, chief executive officer of bluebird bio, in a press release. “It’s also a critical milestone for bluebird bio as an independent severe genetic disease company. We are moving forward with great discipline and exceptional care to deliver on our commitments to patients and achieve our near-term goal of launching three first-in-class gene therapies in the United States.”

A prescription drug user fee act goal data of May 20, 2022 has been set. Additional data will be presented at the 63rd American Society of Hematology Annual Meeting and Exposition.

bluebird bio announces FDA priority review of biologics license application for beti-cel gene therapy for patients with β-thalassemia who require regular red blood cell transfusions. News release. bluebird bio. November 22, 2021. Accessed November 23, 2021.