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 ≤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%)

Better event-free survival compared to untreated patients2

*Permanent 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.


Learn more about the mobility measures used in the SPINRAZA clinical trials.

Those with Type 1 SMA saw improvements in motor milestones that are rarely, if ever, achieved in untreated children2

51% treated vs 0% untreated achieved the motor milestone responder criteria as defined by HINE-2 at 13 months. Motor milestones included2:

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.

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