Bluebird wins FDA approval of gene therapy for rare brain disorder
The therapy, called Skysona and cleared to treat cerebral adrenoleukodystrophy, is the product of more than a decade of work by Bluebird. It will cost $3 million.
The Food and Drug Administration on Friday granted accelerated approval to a personalized gene therapy for an ultra-rare childhood brain disease, called cerebral adrenoleukodystrophy or CALD.
Built from a patient’s own stem cells, the therapy is the first medicine to be made available in the U.S. for CALD, which affects young boys and typically results in severe disability or death. It was developed by the biotechnology company Bluebird bio and will be sold as Skysona.
Its approval is Bluebird’s second in four weeks, following an Aug. 17 FDA decision on another gene therapy from the company for the blood disorder beta thalassemia.
The back-to-back clearances come despite FDA concerns over the risk of the treatments causing cancer. Experts convened by the agency in June to vet the two therapies voted unanimously in favor of both, agreeing their benefits outweigh their potential side effects.
Both CALD and severe beta thalassemia are uncommon, although CALD is far rarer. It’s one of a small cluster of diseases that were featured in the 1992 film Lorenzo’s Oil about a boy with adrenoleukodystrophy and his parents’ hunt for a cure. The buildup of harmful fatty acids in the brain and nervous system causes CALD, which leads to learning and behavioral problems before progressing to more severe neurological damage.
In the U.S., an estimated 50 boys are born each year who will go on to develop CALD. Bluebird expects to treat about 10 annually.
Meant to be a one-time infusion, Skysona will cost $3 million. The price tag makes the therapy one of the most expensive ever launched on a single-use basis, exceeding the $2.8 million cost of Bluebird’s other gene therapy.
‘The most expeditious way’
Skysona is for children aged four to 17 years old who are in the early stages of the disease. The accelerated approval granted by the FDA means Bluebird must confirm its therapy’s benefit through further testing. The company expects to use data from long-term follow-up study, as well as from commercially treated patients, it said in a Friday statement.
Bluebird was seeking full approval of Skysona, but agreed to a conditional clearance after talking with the FDA about some of the agency’s concerns. “Through the course of the review after the [June meeting], we discussed this would probably be the most expeditious way to get the therapy to patients,” said Bluebird CEO Andrew Obenshain in an interview Saturday.
Published Original Source Sept. 17, 2022 Ned Pagliarulo, Lead Editor