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

Nov 27 (Reuters) - The U.S. Food and Drug Administration is weighing the need for regulatory action on bluebird bio's (BLUE.O), opens new tab gene therapy for a rare neurological disorder, it said on Wednesday, as the agency probes additional reports of blood cancers from its use.

Bluebird's Skysona was approved by the FDA in 2022 for the treatment of cerebral adrenoleukodystrophy (CALD) and was priced at $3 million at the time of its launch.

CALD, which affects about 1 in 20,000 to 50,000 people globally, typically occurs in boys aged between 3 and 12 years.

Skysona's prescribing information already includes 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.

Patients should look at alternative therapies such as stem cell transplant from a suitable donor, prior to deciding to treat a child with Skysona, the FDA said, adding that it was investigating the "known risks" associated with the gene therapy, which include hospitalization and death.

"Today's update from the...