February 18, 2021
Bluebird Bio has temporarily suspended two clinical trials of its betibeglogene autotemcel (LentiGlobin) gene therapy to treat sickle cell disease (SCD) after reports of cancer diagnoses in two patients who participated in the trial and received the investigational treatment. One patient who received the treatment more than five years ago was diagnosed with acute myeloid leukemia (AML), while a second patient developed myelodysplastic syndrome. The company is investigating both cases to determine if there is any relationship to the use of BB305 lentiviral vector in the manufacture of LentiGlobin.
In the European Union and United Kingdom, betibeglogene autotemcel (marketed as Zynteglo) is approved to treat transfusion-dependent beta-thalassemia. While there have been no reports of hematologic malignancies in patients with beta-thalassemia, Bluebird has temporarily suspended marketing of Zynteglo while the AML case is assessed because it is manufactured using the same BB305 lentiviral vector.
Bluebird Bio has advised the independent safety review board monitoring the company’s studies of these cases, as well as the Food and Drug Administration and the European Medicines Agency. AABB will continue to monitor news regarding betibeglogene autotemcel and provide updates as they are available.