biotechnology company, Bluebird Bio, Inc. BLUE announced late Friday that the Food and Drug Administration had approved its gene therapy for a rare neurodegenerative disease.
What happened: The FDA granted Skysona, also known as Eli-Cel, an accelerated approval to slow the progression of neurological dysfunction in boys ages 4 to 17 with early, active cerebral adrenoleukodystrophy (CALD), the Somerville, Massachusetts, office said. resident company in a statement.
The approval was awaited because an adcom meeting in June to review the therapy unanimously recommended approval.
CALD is a rare, progressive neurodegenerative disease caused by mutations in a gene called ABCD1, which leads to the production of very long-chain fatty acids in the brain and spinal cord. It affects young boys and causes irreversible, devastating neurological decline. This often results in loss of communication, cortical blindness, the need for tube feeding, complete incontinence, wheelchair dependence, or complete loss of voluntary movement.
Why it matters: For Bluebird, it’s back-to-back approvals after they’ve received them nod for zynteglo, a one-off gene therapy to treat an inherited bleeding disorder called beta-thalassemia in adult and pediatric patients in August.
About half of patients who don’t receive CALD treatment die within five years of the onset of symptoms, the company says. Before Skysona, the only option available to patients was allogeneic hematopoietic stem cell transplantation, which carries the risk of serious complications, including death.
The European Medicines Agency has already approved Skysona for the same indication.
Wells Fargo Securities said in a note last week that eli-cel’s approval decision could be positive and the stock based on the value of the pediatric…
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