Bluebird bio’s stem cell-gene therapy receives FDA’s breakthrough therapy designation

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The FDA grants breakthrough therapy designation to bluebird bio’s Lenti-DTM for treating patients with cerebral adrenoleukodystrophy (CALD).

X-linked adrenoleukodystrophy, also known as Lorenzo’s Oil disease, is caused by mutations in the ABCD1 gene that encodes a protein of the peroxisomal membrane named ALDP. It affects one in every 21,000 male births worldwide. The cerebral form of the disease, CALD is characterized by demyelination and neurodegeneration and is fatal.

The breakthrough therapy designation is based on preliminary results obtained from the company’s ongoing Phase 2/3 STARBEAM trial. The study published in New England Journal of Medicine in October 2017 reported the safety and efficacy of transplanting autologous CD34+ hematopoietic stem cells transduced with Lenti-D (lentiviral vector that encoded ABCD1 gene) in treating young boys with cerebral adrenoleukodystrophy. The primary end point of the study was being alive and having no major functional disability at 24 months after infusion. Interim analysis performed at the median follow-up of 29.4 months showed that 15 out of 17 treated patients were alive at the end of 24 months, with no graft failure and no major functional disabilities.

Lenti-D has also previously received Orphan Drug designation from the FDA and EMA, as well as Rare Pediatric Disease designation by the FDA for the treatment of adrenoleukodystrophy.

Dr David Davidson, bluebird bio’s CMO commented: “With Lenti-D, we hope the modified, autologous hematopoietic stem cells will keep these boys with adrenoleukodystrophy 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.”

Source: FDA Grants Breakthrough Therapy Designation to Lenti-D™ for the Treatment of Cerebral Adrenoleukodystrophy. Press Release