Impact on Dystrophin Levels
88% (7 out of 8 people) showed increases of ~3% or higher, measured at week 25 of treatment.
These significant increases in dystrophin production with VILTEPSO were identified by a method called western blot and verified by a highly sensitive measuring technique known as mass spectrometry.
VILTEPSO was studied in 16 ambulatory (walking) boys ages 4 to less than 10 years who were receiving a stable dose of corticosteroids for at least 3 months.
In this graph, their average dystrophin levels at week 25 of VILTEPSO treatment are compared with their average dystrophin levels before treatment.
Secondary Endpoints
Safety
No patients in the clinical trial discontinued treatment as a result of treatment-related Serious Adverse Events (SAEs)
Functional tests were compared to Duchenne natural history data as the control group rather than to placebo.
Definitive conclusions should not be drawn. Functional data are not in the US Prescribing Information.
With VILTEPSO, the mean change from baseline at week 205 was 2.7 seconds and in the CINRG group the mean change from baseline at week 205 was 8.3 seconds.
Time to stand measures the amount of time it takes for a DMD patient to go from lying on their back to standing.
With VILTEPSO, the mean change from baseline at week 205 was 2.0 seconds and in the CINRG group the mean change from baseline at week 205 was 6.0 seconds.
Time to run/walk 10 meters measures the amount of time it takes for a DMD patient to run or walk 10 meters.
With VILTEPSO, the mean change from baseline at week 205 was 3.1 seconds and in the CINRG group the mean change from baseline at week 205 was 6.1 seconds.
Time to climb four stairs measures the amount of time it takes for a DMD patient to climb four stairs.
FOUR-YEAR SAFETY DATA
AE=adverse event; TEAE=treatment-emergent AE; wk=week.
No patients discontinued the study as a result of
treatment-related Serious Adverse Events (SAEs)
Hear and read what real patients and their families have to say about life and treatment with VILTEPSO.
See Patient StoriesREMEMBER, A DOCTOR IS ALWAYS YOUR BEST RESOURCE FOR information about DMD and determining if VILTEPSO could be the right treatment option for you.
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Get SupportVILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
For more information about VILTEPSO, see full Prescribing Information.
For more information about VILTEPSO, see full Prescribing Information.