WHY SPINRAZA/PRESYMPTOMATIC STUDY
Improvements are possible with SPINRAZA
When started earlier, SPINRAZA may help children progress on WHO motor milestones consistent with normal development.
Supportive study: NURTURE
Who: 25 infants aged 6 weeks and younger who had not yet shown symptoms of SMA
Study time: This supportive study is ongoing
Primary outcomes: Time to death or respiratory intervention†
Secondary outcomes: The effect SPINRAZA has on reaching WHO motor milestones
Limitations: Small number of participants. The study is open-label.
Safety: Consistent with the SPINRAZA prescribing information. After a follow-up of over 14 months to 5.7 years, 25/25 infants experienced any AE (mild, moderate, or severe), and no new safety concerns were identified
After at least 14 months of treatment:
At the initial interim analysis performed after all infants had received SPINRAZA for at least 14 months (median, 25 months; range, 14 to 34 months), 100% (25/25) of infants were alive without the need for permanent ventilation‡, 100% (25/25) were sitting without support, 88% (22/25) were walking with assistance, and 77% (17/22) of infants were walking independently.
After nearly 5 years of follow-up*:
100% (25/25) of presymptomatic infants were alive without permanent ventilation after a median follow-up of 4.9 years in the study. 84% (21/25) never required respiratory intervention
†Respiratory intervention is defined as ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy.
‡Permanent ventilation is defined as ventilation for ≥16 hours/day for >21 days in absence of an acute reversible event, or tracheostomy.
The majority of presymptomatic infants achieved the following WHO motor milestones:
*At least half of patients were followed for at least 4.9 years.
Additional Data
After ~3 years of follow-up§
88%22 out of 25
able to suck/
swallow well
based on maximal HINE-1 score at the
day 778 visit
After ~4 years of follow-up
84%21 out of 25
never required
tube feeds
92%23 out of 25 never needed
to
suction excess saliva or
drool
based on caregiver assessment of swallowing difficulty during mealtime over a period of 7 days
§At least half of patients were followed for at least 3.8 years.
“For Camden to do what he is able
to
do, we are just so thankful.”