FDA Grants Breakthrough Therapy Designation to Lenti-D™ for the Treatment of Cerebral Adrenoleukodystrophy

bluebird bio (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Lenti-D™ for the treatment of patients with cerebral adrenoleukodystrophy (CALD), a rare, serious and life-threatening hereditary neurological disorder.

As quoted in the press release:

Breakthrough Therapy designation is designed to expedite the development and review of a drug intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

“The founding of bluebird was inspired by the potential to develop a one-time gene therapy for boys suffering from this potentially fatal form of adrenoleukodystrophy,” said David Davidson, M.D., chief medical officer, bluebird bio. “With Lenti-D, we hope the modified, autologous hematopoietic stem cells will keep these boys alive and free from major functional disabilities while avoiding many of the safety risks of the current standard of care, allogeneic hematopoietic stem cell transplant. The FDA’s Breakthrough Therapy designation for Lenti-D brings us one step closer to realizing this mission to bring new hope to the patients and families affected by this devastating disease. We look forward to continuing to work closely with the FDA and EMA to expedite development of Lenti-D as a treatment for CALD.”

Click here to read the full press release.