The US Food and Drug Administration (FDA) approves biotech company bluebird bio's gene therapy for the treatment of a rare neurological disorder
The approval was largely expected after the drug received unanimous endorsement from a panel of outside advisers to the FDA in June. for the treatment of a rare neurological disorder, the company said.
"SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys" with early, active Cerebral Adrenoleukodystrophy , the company said, saying CALD is a "devastating and fatal neurodegenerative disease." 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.