US FDA approves Bluebird Bio's gene therapy
The US Food and Drug Administration (FDA) has approved Bluebird bio's gene therapy for the treatment of a rare neurological disorder, the company announced late Friday.
"SKYSONA is the first FDA-approved treatment shown to slow the progression of neurological dysfunction in boys with early, active Cerebral Adrenoleukodystrophy (CALD)," the company said in a statement, adding that CALD is a "devastating and fatal neurodegenerative disease."
Bluebird said it expects commercial product
to be available through a limited number of qualified treatment centers in the United States by the end of 2022.
In August, the company's beti-cel therapy received FDA approval to treat a rare blood disorder at a record price of $2.8 million, the most expensive treatment to date.
CALD is caused by mutations in a gene called ABCD1, which leads to a buildup of very long-chain fatty acids in the brain and spinal cord. It typically occurs in boys between the ages of 3 and 12.
Eli-cel inserts functional copies of the ABCD1 gene into the patient's stem cells to help produce a protein needed to break down long-chain fatty acids.
Approval was widely expected after the drug won unanimous approval from a panel of the FDA's outside advisors in June.
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