In an analysis of the Phase 3 ENDEAR end of study results, a greater proportion of infants with SMA on permanent ventilation treated with SPINRAZA demonstrated clinical benefits compared to untreated infants.
End of study data from both the Phase 3 ENDEAR and CHERISH studies further demonstrate that earlier SPINRAZA treatment in individuals with SMA may lead to improved outcomes. In individuals with shorter disease durations (i.e., generally younger at symptom onset), infants in ENDEAR demonstrated a lower risk of death or permanent ventilation and children in CHERISH demonstrated greater motor function improvement from baseline to 15 months compared to untreated individuals.
In addition, further results from the interim analysis of the Phase 2 NURTURE study highlight the clinically meaningful efficacy of SPINRAZA on event-free survival, measures of motor function and achievement of motor milestones when administered to infants with genetically-diagnosed SMA before symptom onset.
“New SPINRAZA data continue to reinforce the positive results seen in clinical studies and in my own practice,” said Thomas Crawford, M.D., co-director, Muscular Dystrophy Association Clinic at Johns Hopkins Medicine. “The SPINRAZA clinical development program demonstrates the impact of early treatment. The additional NURTURE data extends this finding by showing substantial improvements in motor milestones, generally consistent with normal development among infants with SMA who have yet to manifest symptoms before they were treated with SPINRAZA.”
SPINRAZA demonstrated a favorable benefit-risk profile, with commonly reported adverse events consistent with those expected in the general SMA population or related to a lumbar puncture procedure. Safety data involving the intrathecal administration of SPINRAZA showed the incidence and nature of the most common lumbar puncture-related adverse events were similar in children with later-onset SMA with or without scoliosis in the clinical studies.