Studies in patients with spinal muscular
atrophy (SMA) show SPINRAZA
Achieved meaningful results across
age
groups—from infants to adults1-3
Backed by the longest clinical trial
program to date in infants and
children.4 Supported by extensive
real-world evidence in
adults, teens,
and older children2,3,5-7
Click the tabs to see the data in the selected population
Pivotal trial: ENDEAR1,8
Study: A phase 3, multicenter, double-blind, randomized (2:1), sham-controlled trial
Treatment duration: 13 months; SPINRAZA (12 mg)
Participants: 121 patients with early-onset SMA aged ≤7 months 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 respiratory infection (55%) and constipation (35%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%)
HINE-2, Hammersmith Infant Neurological Exam Part 2; SMA, spinal muscular atrophy.
Better event-free survival compared to untreated patients. Event-free survival was the primary endpoint, defined as no death or use of permanent assisted ventilation8
Primary endpoint: No death or use of permanent assisted ventilation*
Learn more about the mobility measures used in the SPINRAZA clinical trials.
*Permanent assisted ventilation was defined as tracheostomy or ventilatory support for ≥16 hour per day for >21 continuous days in the absence of an acute reversible event.
CI, confidence interval; HR, hazard ratio.
51% treated vs 0% untreated achieved the motor milestone responder criteria as defined by HINE-2 at 13 months.† Motor milestones included8:
†P<0.0001.8
A treatment responder was defined as any patient achieving an improvement in more categories of motor milestones than worsening according to HINE-2.1
Patients treated with SPINRAZA continued to improve over time, as measured at Day 394, while untreated patients saw a decline in motor milestones8
The ENDEAR trial concluded early because of the results of the interim analysis, hence why not all patients were assessed at Day 394.
Mean change in HINE-2 total motor milestone score over time
HINE-2, Hammersmith Infant Neurological Examination Section 2, motor milestone portion; SEM, standard error of the mean; SMA, spinal muscular atrophy.