FDA investigates bluebird’s Skysona over cancer risk

The US Food and Drug Administration has received new reports of hematologic malignancies, including life-threatening cases of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), following treatment of early, active cerebral adrenoleukodystrophy (CALD) patients with Skysona (elivaldogene autotemcel).

Reported cases appear to be related to treatment with the bluebird bio (Nasdaq: BLUE) drug, the FDA says.

A study published in The New England Journal of Medicine last month detailed seven cases of blood cancer in patients treated with Skysona, including MDS and AML, revealing that they were likely triggered by the viral vector used in the therapy.