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

Signage outside of the Food and Drug Administration (FDA) headquarters in White Oak, Md., on Aug. 29, 2020. Andrew Kelly/Reuters

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.”