Bluebird Bio’s gene therapy for a rare neurological disorder has been approved by the U.S. FDA.
The U.S. Food and Drug Administration (FDA) has approved Bluebird Bio’s gene therapy to treat a rare neurological disorder, the company said late Friday.
The company said in a statement, “SKYSONA is the first FDA-approved therapy that has been shown to slow the progression of neurologic dysfunction in boys with early, active cerebral adrenal leukodystrophy (CALD),” which is a “devastating and fatal neurodegenerative disease.”
Bluebird said that it thinks commercial products will be available by the end of 2022 through a small number of qualified treatment centres in the United States.
In August, the FDA approved the company’s beti-cel therapy to treat a rare blood disorder. It cost a record $2.8 million, making it the most expensive treatment ever.
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CALD is caused by changes in a gene called ABCD1. These changes cause the brain and spinal cord to get full of very long-chain fatty acids. Boys between the ages of 3 and 12 are most likely to get it.
Eli-Cel adds functional copies of the ABCD1 gene to a patient’s stem cells to help them make a protein that is needed to break down long-chain fatty acids.
The approval was mostly expected after a group of outside advisors to the FDA gave the drug their unanimous approval in June.
