U.S. FDA approves bluebird bio’s gene therapy for a rare neurological disorder

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“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 in a statement, saying CALD is a “devastating and fatal neurodegenerative disease.”

Bluebird 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.

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.