The US Food and Drug Administration (FDA) has approved a gene therapy for Bluebird bio to treat a rare neurological disorder, the company said late Friday.
“Skysona is the first FDA-approved treatment shown to slow the progression of neurological dysfunction in boys” with early active cerebral adrenal adrenal dystrophy (CALD), the company said in a statement.
Bluebird said it expects the commercial product to be available by the end of 2022 through a limited number of qualified treatment centers in the United States.
In August, the company’s beti-cel treatment won FDA approval to treat a rare blood disorder for a record $2.8 million, the most expensive treatment to date.
CALD is caused by mutations in a gene called ABCD1 that lead to a buildup of long-chain fatty acids in the brain and spinal cord. It usually occurs in boys between the ages of 3 and 12.
Eli-cel adds functional copies of the ABCD1 gene into a patient’s stem cells to help produce the protein needed to break down long-chain fatty acids.
The approval was largely expected after the drug won unanimous approval from a panel of the Food and Drug Administration’s external advisors in June.