WHY SPINRAZA/EARLY-ONSET STUDY
SPINRAZA delivered powerful survival results and motor function improvement for children with SMA
In a pivotal trial, SPINRAZA achieved significant results in important milestones compared to untreated control group
Pivotal study: ENDEAR
Who: 121 children aged 7 months and younger with Type 1 (early-onset) SMA (80 children were treated with SPINRAZA versus 41 who were given a placebo)
Study time: 13 months
Primary outcomes:
- Time to death or use of permanent assisted ventilation
- The proportion of responders (or number of children) who showed an improvement in motor milestones according to HINE-2
Safety: The most common side effects were lower respiratory infection (55%; 44/80) and constipation (35%; 28/80). Serious adverse reactions of collapsed lung (atelectasis) were more frequent in the SPINRAZA-treated group (18%; 14/80) than in the control group (10%; 4/41).
Those with Type 1 SMA showed improvements in motor milestones that are rarely, if ever, achieved in untreated children
51% (37/73)
of children treated with
SPINRAZA
VS
0% (0/37)
of the children in the
untreated control group
were motor milestone responders
according to HINE-2 at 13 months
Motor milestones included:
There was an overall 47% reduced risk of mortality or permanent ventilation in the SPINRAZA group versus the untreated group and a 63% reduced risk of mortality in the SPINRAZA group versus the untreated group
“Whether she can or can't do
something, she's going to try.”