FDA approves Zynteglo for patients with transfusion-dependent beta-thalassemia
Click Here to Manage Email Alerts
The FDA approved betibeglogene autotemcel for treatment of patients with beta-thalassemia who require routine red blood cell transfusions.
Betibeglogene autotemcel (Zynteglo, bluebird bio), also known as beti-cel, is a one-time ex vivo gene therapy that adds functional copies of a modified form of the beta-globin gene into a patient’s hematopoietic stem cells. The therapy received FDA priority review in November.
A final evidence report released last month by the nonprofit Institute for Clinical and Economic Review concluded that adequate evidence existed to show beti-cel confers a net health benefit vs. standard clinical management for patients with beta-thalassemia. The therapy also meets commonly accepted value thresholds at a $2.1 million estimated annual price with an 80% payback option for patients who do not remain transfusion-independent over a 5-year period, cost-effectiveness modeling showed.
“Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in an FDA press release. “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.”
Two multicenter clinical trials established the safety and effectiveness of the therapy among adults and children with transfusion-dependent beta-thalassemia, the most severe form of the condition. Among 41 patients who received beti-cel, 89% achieved transfusion independence, according to the press release.
The most frequently reported adverse events associated with beti-cel included reduced platelet and other blood cell levels, mucositis, febrile neutropenia, vomiting, pyrexia, alopecia, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash and nausea.
The FDA noted a potential risk for blood cancer associated with beti-cel, although studies have not shown any cases. Patients who receive the gene therapy should undergo blood monitoring for at least 15 years for any sign of cancer, as well as monitoring for hypersensitivity reactions during treatment, thrombocytopenia and bleeding, the release states.
Click Here to Manage Email Alerts