FDA investigating serious risk of hematologic malignancy following Skysona

The FDA stated: “Since approval, the Food and Drug Administration has received additional reports of hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia, following treatment of early, active cerebral adrenoleukodystrophy – CALD- patients with Skysona, or elivaldogene autotemcel, an autologous hematopoietic stem cell-based gene therapy. The reported cases appear to be related to treatment with Skysona. Reports were received from clinical trials, with cases diagnosed between 14 to 92 months post-treatment. FDA is investigating the known risk of hematologic malignancies with serious outcomes, including those such as hospitalization, the requirement for allogeneic hematopoietic stem cell transplantation, and death, and is evaluating the need for further regulatory action.” Skysona is marketed by Bluebird Bio (BLUE).