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

[September 17, 2022] 

The U.S. Food and Drug Administration (FDA) has approved Bluebird bio's gene therapy for the treatment of a rare neurological disorder, the company said late on Friday.

"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.

(Reporting by Mrinalika Roy and Jaiveer Shekhawat in Bengaluru; Editing by Sriraj Kalluvila, Devika Syamnath and William Mallard)

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