FDA probes blood cancer risk from bluebird’s gene therapy, weighs regulatory action

(Reuters) -The U.S. Food and Drug Administration is weighing the need for a regulatory action on bluebird bio’s gene therapy for a rare neurological disorder, it said on Wednesday, as the agency probes additional reports of blood cancers from its use.

Skysona was approved by the FDA in 2022 for the treatment of cerebral adrenoleukodystrophy.

The therapy’s prescribing information included a warning for blood cancers, including leukemia and myelodysplastic syndromes, which are a group of cancers that occur when the bone marrow produces immature blood cells instead of healthy ones.

The FDA said considering the risk of such cancers, patients should consider alternative therapies.

(Reporting by Christy Santhosh in Bengaluru; Editing by Shilpi Majumdar)