FDA Approves First Cell-Based Gene Therapy for Beta-Thalassemia

MONDAY, Aug. 22, 2022 (HealthDay News) -- The U.S. Food and Drug Administration has approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
Zynteglo is a one-time, single-dose gene therapy product. Each dose of Zynteglo is customized and created using the patient's own bone marrow stem cells, which are genetically modified to produce functional beta-globin. The application was granted a rare pediatric disease voucher, as well as priority review, fast-track, breakthrough therapy, and orphan designations.
The approval was based on two multicenter clinical studies. Of 41 patients receiving Zynteglo, 89 percent achieved transfusion independence, defined as maintaining a predetermined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. The most common adverse reactions seen with Zynteglo included reduced platelet and other blood cell levels, mucositis, febrile neutropenia, vomiting, fever, alopecia, nosebleed, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and itch.
Given the potential risk for blood cancer associated with this treatment, patients receiving Zynteglo should have their blood monitored for at least 15 years for evidence of cancer. The FDA says patients should also be monitored for hypersensitivity reactions during Zynteglo administration and for thrombocytopenia and bleeding.
Approval of Zynteglo was granted to bluebird bio.
Related Posts
COVID-19-Related Financial Disruptions Linked to Child Mental Health
THURSDAY, March 16, 2023 (HealthDay News) -- COVID-19-policy-related financial...
Healthy Lifestyle Cuts Prostate Cancer Mortality Among High-Risk Men
FRIDAY, July 15, 2022 (HealthDay News) -- Adherence to a healthy lifestyle is...
Majority of Pregnant U.S. Women Were Already in Poor Health: Study
TUESDAY, Feb. 15, 2022 (HealthDay News) -- Once they're pregnant, women have a...
Outpatient Antibiotic Management of Appendicitis Feasible for Selected Adults
FRIDAY, July 15, 2022 (HealthDay News) -- Outpatient management seems safe for...