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The biotechnology company’s Skysona gene therapy will help slow the progression of neurologic dysfunction in boys with active Cerebral Adrenoleukodystrophy and will be available by the end of 2022.
The US Food and Drug Administration (FDA) has approved bluebird bio’s gene therapy for the treatment of a rare neurological disorder, the company said.
In a statement late Friday, bluebird bio said it anticipates commercial product will be available by the end of 2022 through a limited number of qualified treatment centers in the United States.
“SKYSONA is the first FDA approved therapy shown to slow the
progression of neurologic dysfunction in boys” with early,
active Cerebral Adrenoleukodystrophy (CALD), the company said, saying CALD is a “devastating and fatal
neurodegenerative disease.”
In August, the company’s beti-cel therapy secured FDA
approval to treat a rare blood disorder that was priced at a
record $2.8 million, the most expensive treatment to date.
CALD is caused by mutations in a gene called ABCD1 that
leads to the 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 adds functional copies of the ABCD1 gene in a
patient’s stem cells to help produce a protein required to break
down the long-chain fatty acids.
The approval was largely expected after the drug received unanimous endorsement from a panel of outside advisers to the FDA in June.
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We’re delighted @USFDA has approved our #genetherapy for the treatment of early, active cerebral #adrenoleukodystrophy (CALD) in boys 4-17 years of age. https://t.co/vI9uHVjsB1 [1/2] pic.twitter.com/3Kk2OcWgJo
— bluebird bio (@bluebirdbio) September 17, 2022
Source: Reuters
US FDA approves bluebird bio’s gene therapy for rare neurological disorder
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