Bluebird bags breakthrough status for CALD gene therapy
Bluebird Bio has claimed an FDA breakthrough designation for its Lenti-D gene therapy for cerebral adrenoleukodystrophy (CALD) – a rare neurological disease mainly affecting young males.
The new status – which could result in a speedy review by the US regulator and shorten the time to market for the therapy – comes after impressive data from a phase II/III trial suggesting it could stabilise disease for more than two years in boys with the highly progressive and life-threatening disorder.
Data from 17 patients in the STARBEAM study was published in the New England Journal of Medicine last October, and showed that Lenti-D (elivaldogene tavalentivec) stabilised disease in 15 of them, with one boy dying as a result of the disease and another progressing despite therapy.
Read more: http://www.pmlive.com/pharma_news/bluebird_bags_breakthrough_status_for_cald_gene_therapy_1236797
The new status – which could result in a speedy review by the US regulator and shorten the time to market for the therapy – comes after impressive data from a phase II/III trial suggesting it could stabilise disease for more than two years in boys with the highly progressive and life-threatening disorder.
Data from 17 patients in the STARBEAM study was published in the New England Journal of Medicine last October, and showed that Lenti-D (elivaldogene tavalentivec) stabilised disease in 15 of them, with one boy dying as a result of the disease and another progressing despite therapy.
Read more: http://www.pmlive.com/pharma_news/bluebird_bags_breakthrough_status_for_cald_gene_therapy_1236797