US FDA Approves Gene Therapy of Bluebird Bio for Rare Neurological Disorder


The US Food and Drug Administration (FDA) of the United States has approved gene therapy of Bluebird Bio for the treatment of a rare neurological disorder. SKYSONA is the first therapy approved by US FDA shown to slow the progression of neurologic dysfunction in boys with active and early Cerebral Adrenoleukodystrophy (CALD).

Bluebird Bio said, the CALD is a dangerous and fatal neurodegenerative disease. The company anticipates that commercial product will be available by the end of 2022 through a limited number of the qualified treatment centers in the United States of America.

In August 2022, beti-cell therapy of the company secured US FDA approval for the treatment of a rare blood disorder that was priced at a record 2.8 million dollars, which is the most expensive treatment till date.

Cerebral Adrenoleukodystrophy (CALD) is caused by the mutation in a gene called as ABCD1, which leads to the development of very long chain fatty acids in the spinal cord and brain. It typically occurs in boys between the age group of 3 to 12 years.

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Eli-cel adds functional copies of ABCD1 gene in the stem cells of a patients to help decrease a protein that is required to break down the long chain fatty acids. This approval was expected after the drug received endorsement from a panel of advisors to US FDA in June 2022.