In sur­prise, FDA ad­comm votes unan­i­mous­ly in fa­vor of blue­bird's gene ther­a­py for rare dis­ease even with safe­ty con­cerns

In the first day of a two-day meet­ing, the FDA’s Cel­lu­lar, Tis­sue and Gene Ther­a­pies ad­vi­so­ry com­mit­tee on Thurs­day gave a big thumbs up to blue­bird bio’s po­ten­tial gene ther­a­py for the rare but fa­tal con­di­tion known as cere­bral adrenoleukody­s­tro­phy (CALD) by a vote of 15-0, de­spite FDA safe­ty con­cerns.

Fol­low­ing crit­i­cal com­ments and safe­ty ques­tions from FDA in its brief­ing doc­u­ments, in­clud­ing a link be­tween three can­cer cas­es known as myelodys­plas­tic syn­drome (MDS) in those re­ceiv­ing the treat­ment, dubbed eli-cel, ad­comm mem­bers dug through what tran­spired in each of the cas­es and called for con­tin­ued mon­i­tor­ing.

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