United States: The U.S. FDA said on Wednesday it is considering whether it needs to take further action on gene therapy from Bluebird Bio Inc. (Bluebird. bio), a treatment for a neurological disorder, after reports of more blood cancer cases, as reported by Reuters.
Bluebird’s Skysona got approval from the FDA in 2022 for cerebral adrenoleukodystrophy (CALD) and initially launched at $3 million.
Rare Disorder and Known Risks
This condition which is very rare affects around 1 in 20,000 to 50,000 people of the world and is most evident in little boys between the ages of three and twelve.
Current prescribing information for Skysona already carries a warning against blood cancers, such as leukemia and myelodysplastic, which is a type of cancer that starts in the blood-forming tissues of the bone marrow and causes it to produce abnormal blood cells instead of healthy ones.
The FDA said patients should consider other treatments, like a stem cell transplant from a compatible donor, before deciding to use Skysona on a child, and the organization is looking into the dangers associated with the gene therapy, including 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.
FDA Investigation Not Triggered by New Cases
Cantor Fitzgerald analyst Eric Schmidt downplayed the FDA investigation as not being a shocking event, especially in the light of a recent article in the New England Journal of Medicine which brought this danger to the public limelight.
The patients with this disease have a very poor quality of life and poor prognosis and so the FDA will most likely see the risk or benefit of using Skysona to treat this disease as favorable, he said, as reported by Reuters.
Bluebird Bio’s Challenges
Another regional player, Bluebird from Massachusetts, which has been raising concern concerns for the past couple of years, is also looking to bring its other gene therapies. It terminated 25% of its employees in September to cut costs, and, sequentially, the second reshuffle in two years.
