ZOLGENSMA — evaluated in 4 clinical trials including ongoing and completed studies

STR1VE

An ongoing, open-label, single-arm clinical trial of symptomatic patients with SMA (N=22)1,2,a

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
aOne patient was initially classified as presymptomatic and removed from the intent-to-treat (ITT) data set included in the Prescribing Information. The patient was later confirmed to be symptomatic at baseline and will be included in the final ITT analysis.1
bEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days.

START

A completed, open-label, single-arm, ascending-dose clinical trial of symptomatic patients with SMA that enrolled a low-dose cohort (n=3) and a high-dose cohort (n=12)3,a

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
aThe dosage received by patients in the low-dose cohort was approximately one-third of the dosage received by patients in the high-dose cohort. However, the precise dosages of ZOLGENSMA received by patients in this completed clinical trial are unclear due to a change in the method of measuring ZOLGENSMA concentration, and to decreases in the concentration of stored ZOLGENSMA over time.
bEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days

Long-term follow-up

An ongoing, long-term follow-up study of ZOLGENSMA-treated patients from the START clinical trial (N=13)1,4

SPR1NT

An ongoing (enrolling), open-label, single-arm clinical trial of presymptomatic patients with SMA5

aAs of March 2019 data cut.
bEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days.