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KEY INSIGHT
Boys aged 4 to <10 years (N=16) with DMD were enrolled in a randomized, double-blind, placebo-controlled, 4-week safety period of once-weekly infusion of 40 or 80 mg/kg VILTEPSO, followed by a 20-week open-label study to assess the efficacy and safety of VILTEPSO. After completion of the 24-week study, patients could enroll in up to a 192-week extension study (216 weeks total). Efficacy assessments were conducted every 12 weeks.
LONG-TERM DATA
Functional tests were compared to Duchenne natural history data as the control group rather than to placebo. Functional data are not in the US Prescribing Information.
KEY INSIGHT
LONG-TERM DATA
NSAA is a validated 34-point scale for assessing motor function in ambulant DMD patients, using 17 physical tests to evaluate disease status and document response to therapy in a clinical trial.
NSAA is used to assess motor function in ambulatory patients with DMD. It includes 17 physical tests, each scored from 0 to 2 based on the patient’s performance, for a validated 34-point scale. The NSAA takes about 10 to 15 minutes and is suitable for both specialist clinics and community settings.
NSAA scores:
0=Unable to perform the task independently
1=Able to perform the task with assistance
2=Able to perform the task without assistance
| * | VILTEPSO Participants | ||
|---|---|---|---|
| Participants with: | 40 mg/kg/wk
n=8 | 80 mg/kg/wk
n=8 | Total N=16 |
| Any TEAE, n (%) | 8 (100) | 8 (100) | 16 (100) |
| Any drug-related TEAE, n (%) | 0 | 1 (13) | 1 (6) |
| Any serious treatment-related AE, n (%) | 0 | 0 | 0 |
| Study drug discontinuation due to TEAE, n (%) | 0 | 0 | 0 |
| Death, n (%) | 0 | 0 | 0 |
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