23 May 2018 -  bluebird bio today announced that the U.S. FDA has granted breakthrough therapy designation to Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy, a rare, serious and life-threatening hereditary neurological disorder.

Breakthrough therapy designation is supported by preliminary data from the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D investigational gene therapy in boys with CALD, 17 years of age or less who do not have a matched sibling donor. Findings from 17 patients were published in theNew England Journal of Medicine in October 2017 and showed that 15 of the 17 patients (88%) infused with Lenti-D remained alive and free of major functional disabilities at 2 years post-treatment, the primary efficacy endpoint of the trial. Results also showed that the safety profile of Lenti-D remains consistent with myeloablative chemotherapy. Additionally, no engraftment failure, graft versus host disease or treatment-related mortality occurred, nor was there any evidence of insertional oncogenesis.

bluebird’s Lenti-D investigational gene therapy previously was granted orphan drug designation by the FDA and EMA, as well as rare paediatric disease designation by the FDA for the treatment of adrenoleukodystrophy.

Read bluebird bio press release