Why SPINRAZA/Presymptomatic Efficacy

Supportive trial: NURTURE1

Study: Phase 2, open-label, multicenter, multinational, single-arm study

Treatment duration: Ongoing, results from a median of 2.9-year interim analysis

Participants: 25 presymptomatic, genetically diagnosed infants who were likely to develop Type 1 or 2 SMA aged ≤6 weeks at time of first dose

Primary endpoint: Time to death or respiratory intervention

Secondary endpoints: Attainment of WHO motor milestones and change from baseline in the CHOP INTEND motor function scale

Study limitations: Small participant group size and no sham control group; CHOP INTEND score was assessed only until a participant achieved the maximum score of 64. HINE-2 score was assessed until day 778, but not at later study visits

Safety: Consistent with the SPINRAZA prescribing information; no new safety concerns were identified. 80% (20/25) of participants had an adverse event (AE) that was mild or moderate in severity. Eight participants experienced AEs that were possibly or definitely related to the lumbar puncture procedure.

All the following are results after a median follow-up of 2.9 years in the study.1

100% (25/25) of presymptomatic infants were alive without permanent ventilation.1

The majority of infants achieved the following WHO motor milestones:

The natural history of SMA shows infants with Type 1 would not survive
or would require permanent ventilation by 13.5 months of age.1

100% (25/25) of presymptomatic infants treated with SPINRAZA continued to gain motor skills over a median of 2.9 years of follow-up.1

Mean CHOP INTEND scores showed SPINRAZA improved motor function, with scores rising steadily until day 183. The majority of patients achieved a maximum score of 64 and then remained stable over time.

Presymptomatic infants likely to develop Type 1 or type 2 SMA treated with SPINRAZA continued to gain motor skills over time

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders.
SE=standard error.
aIn the original protocol, CHOP INTEND was to be assessed in participants at each visit up to day 778; however, this was amended to be until they had a maximum score of 64. Once a score of 64 was achieved, CHOP INTEND was no longer assessed.

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

Christine, mother of Camden

“We are very, very thankful for the advances in science.”

—Christine, mother of Camden

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