FDA Approves Bluebird Bio’s Gene Therapy for a Rare Neurological Disorder

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….