Why SPINRAZA/Early-Onset Efficacy
Pivotal trial:
ENDEAR2,5
Study: A phase 3, multicenter, double-blind, randomized (2:1), sham-controlled trial
Treatment duration: 13 months
Participants: 121 patients with early-onset SMA aged ≤7months at time of first dose
Primary endpoints: Survival without the need for permanent ventilation and proportion of patients meeting the criteria for motor milestone responder using HINE-2
Safety: The most common side effects were lower repository infection (55%) and constipation (35%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%)
Better event-free survival compared to untreated patients2
aPermanent assisted ventilation was defined as tracheostomy or ventilatory support for ≥16 hours per day for >21 continuous days in the absence of an acute reversible event.
85% SPINRAZA treated vs 61% untreated survived; HR=0.37; P=0.004
Learn more about the mobility measures used in the SPINRAZA clinical trials.
aPermanent assisted ventilation was defined as tracheostomy or ventilatory support for ≥16 hours per day for >21 continuous days in the absence of an acute reversible event.
51% treated vs 0% untreated achieved the motor milestone responder criteria as defined by HINE-2 at 13 months.† Motor milestones included2:
†P<0.0001
Responders=patients achieving an improvement in more categories of motor milestones than worsening according to HINE-2.5
HINE-2=Hammersmith Infant Neurological Examination Section 2, motor milestone portion.
Patients treated with SPINRAZA continued to improve over time (day 394), while untreated patients saw a decline in motor milestones2
SEM=standard error of the mean.
“Whether she can or
can’t do something, she
is going to try.”