Infants with spinal muscular atrophy who receive nusinersen experience greater survival and motor function improvement compared with controls, according to findings from a randomized, double-blind, phase 3 study published in the New England Journal of Medicine.
Investigators randomly assigned infants with spinal muscular atrophy to receive either nusinersen (n=80) or a sham treatment (n=41). The primary end point was comprised of event-free survival and motor-milestone response.
At the prespecified interim analysis, the researchers observed a significantly greater proportion of infants in the nusinersen group vs the control group who achieved a motor-milestone response (41% vs 0%; P <.001). The findings of this interim analysis resulted in early trial termination.
Results of the final analysis demonstrated superiority of nusinersen vs sham treatment for achieving motor-milestone response (51% vs 0%). Event-free survival was significantly higher among patients taking nusinersen compared with controls (hazard ratio for death or the use of permanent-assisted ventilation 0.53; P =.005).