The FDA approved labeling changes for elivaldogene autotemcel (eli-cel; Skysona), a one-time gene therapy for patients with early, active cerebral adrenoleukodystrophy (CALD), to reflect new safety information about hematologic malignancy risk, the agency announced Friday.

The update restricts the eli-cel indication to patients without an available human leukocyte antigen (HLA)-matched allogeneic hematopoietic stem cell donor. Given the risk of blood cancer, eli-cel should be used only in CALD patients without suitable alternative treatment options, the agency said.

The notice follows an FDA investigation of reports of blood cancers -- including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia -- in patients with early, active CALD who were treated with

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