WHY SPINRAZA/PRESYMPTOMATIC STUDY
Considering treatment?
The Low Dose Regimen of SPINRAZA®
(nusinersen) has been studied for over
a decade in the longest SMA clinical
development program to date
Presymptomatic infants with spinal muscular atrophy (SMA) progressed with sitting and walking milestones, supporting early treatment 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: A supportive study with results up to 5 years
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, which means all infants received SPINRAZA and there is not an untreated group for comparisons
Safety: Consistent with the SPINRAZA prescribing information
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†
*Median, 4.9 years; range, 3.8 to 5.5 years.
†Respiratory intervention was defined as ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy. Permanent ventilation is defined as equal or greater than 16 hours/day continuously for greater than 21 days in absence of an acute reversible event or tracheostomy.
The majority of presymptomatic infants achieved the following WHO motor milestones:
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.
