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 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.
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 agency was not prompted by any new cases of malignancy or other safety updates,” bluebird told Reuters. The risk of blood cancer is known, it said.
Cantor Fitzgerald analyst Eric Schmidt said the FDA probe is “not a surprising development given the recent article in the New England Journal of Medicine that highlighted this risk”.
Patients with this disease have a very poor prognosis, so the FDA will likely view the risk or benefit of treatment with Skysona as favorable, Schmidt said.
Massachusetts-based bluebird, which has been raising going concern doubts for the past couple of years, is also looking to launch its other gene therapies. It had laid off about 25% of its workforce in September, in a second major restructuring in two years to reduce costs.
(Reporting by Christy Santhosh in Bengaluru; Editing by Shilpi Majumdar)
 
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