The U.S. Food and Drug Administration granted breakthrough therapy status to Lenti-D, Bluebird Bio Inc.'s gene therapy for treating cerebral adrenoleukodystrophy.
Cerebral adrenoleukodystrophy, or CALD, is a rare genetic progressive neurodegenerative disease characterized by the breakdown of the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control.
The designation is backed by preliminary data from the phase 2/3 Starbeam study examining the gene therapy in male patients 17 years or younger who lack a matched sibling donor.
In the trial, 88%, or 15 out of the 17 patients who received the gene therapy, remained alive and free of major functional disabilities two years after the treatment.
Bluebird Bio's Lenti-D previously received the orphan drug designation from the U.S. FDA and European Medicines Agency. The treatment was also granted the rare pediatric disease designation by the FDA for treating adrenoleukodystrophy.
