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FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients With Beta-Thalassemia Who Require Regular Blood Transfusions


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On August 17, the U.S. Food and Drug Administration (FDA) approved betibeglogene autotemcel (Zynteglo), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. 

“[This] approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” said Peter Marks, MD, PhD, Director of the FDA’s Center for Biologics Evaluation and Research. “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting development of innovative therapies for patients who have limited treatment options.” 

Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood, through mutations in the beta-globin subunit, leading to insufficient delivery of oxygen in the body. The reduced levels of red blood cells can lead to a number of health issues, including dizziness, weakness, fatigue, bone abnormalities, and more serious complications. Transfusion-dependent beta-thalassemia, the most severe form of the condition, generally requires lifelong red blood cell transfusions as the standard course of treatment. These regular transfusions can be associated with multiple health complications of their own, including problems in the heart, liver, and other organs due to an excessive build-up of iron in the body. 

Betibeglogene autotemcel is a one-time gene therapy product administered as a single dose. Each dose of betibeglogene autotemcel is a customized treatment created using the patient’s own bone marrow stem cells that are genetically modified to produce functional beta-globin (a hemoglobin component). 

The safety and effectiveness of betibeglogene autotemcel were established in two multicenter clinical studies that included adult and pediatric patients with beta-thalassemia requiring regular transfusions. Effectiveness was established based on achievement of transfusion independence, which is attained when the patient maintains a predetermined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. Of 41 patients receiving betibeglogene autotemcel, 89% achieved transfusion independence.   

The most common adverse reactions associated with betibeglogene autotemcel included reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia, alopecia, epistaxis, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus. 

There is a potential risk of blood cancer associated with this treatment; however, no cases have been seen in studies of betibeglogene autotemcel. Patients who receive betibeglogene autotemcel should have their blood monitored for at least 15 years for any evidence of cancer. Patients should also be monitored for hypersensitivity reactions during administration and should be monitored for thrombocytopenia and bleeding. 

This application was granted a rare pediatric disease voucher, in addition to receiving Priority Review, Fast Track, Breakthrough Therapy, and Orphan designations. 

The FDA granted approval of Zynteglo to bluebird bio, Inc.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
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