Why SPINRAZA/Early-Onset Efficacy

Pivotal trial: ENDEAR2,5

Study: Phase 3, multicenter, double-blind, randomized (2:1), sham-controlled

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: 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

61% of those with Type 1 early-onset SMA treated with SPINRAZA survived without need for permanent ventilation

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.

Measures of Mobilty

Those with Type 1 SMA saw improvements in motor milestones that are rarely, if ever, achieved in untreated children.2

51% treated vs 0% untreated achieved the motor milestone responder criteria as defined by HINE-2 at 9 months.b Motor milestones included2:

graphic featuring major milestones children on treatment with SPINRAZA achieved
graphic featuring major milestones children on treatment with SPINRAZA achieved

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 milestones.2

SEM=standard error of the mean.

Ian Quote

“Whether she can or can’t do something, she is going to try.”

—Mother of Sofia

Review the warnings and precautions including thrombocytopenia, coagulation abnormalities, and renal toxicity.