US FDA approves Bluebird Bio’s gene therapy for 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
Bluebird said it anticipates commercial product will be available by the end of 2022 through a limited number of qualified treatment centres in the US.
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 three and 12 years.
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.
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