Efficacy & Safety

In a clinical study, 100% of patients taking VILTEPSO showed an increase in dystrophin levels

Average dystrophin level increase was nearly 6% at week 25 of treatment.

Impact on Dystrophin Levels
Secondary Endpoints
Safety
Muscle Function Data
Four-Year Safety Data

Impact on Dystrophin Levels

VILTEPSO significantly increased dystrophin production

Graph with orange bars showing dystrophin expression increases for 8 individual patients after 25 weeks on Viltepso. Bar chart optimized for mobile displaying individual baseline and Week 25 dystrophin levels in patients treated with Viltepso.

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 increased dystrophin levels to
nearly 6% of normal

Bar chart illustrating patient-by-patient dystrophin expression levels as percent of normal before and after 25 weeks of Viltepso. Mobile chart displaying dystrophin levels as a percentage of normal at Week 25 compared to baseline, normalized to myosin heavy chain.

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

In the same clinical study, additional results
included evidence of dystrophin production and
timed motor function tests

Dystrophin Production Evidence

Bar chart comparing dystrophin levels at baseline and Week 25 across patients A–H treated with Viltepso; 100% showed increased expression. Mobile-friendly vertical bar chart showing baseline vs. Week 25 dystrophin levels in 8 patients; 88% reached ~3% of normal or higher.
*Exon 53 skipping efficiency assessed by RT-PCR. Mean dystrophin levels assessed by mass spectrometry. Dystrophin localization assessed by immunofluorescence staining. RT-PCR=reverse transcriptase-polymerase chain reaction.
 

Motor Function Tests

Functional tests were compared to Duchenne natural history (DNHS) data as the control group rather than to placebo. Definitive conclusions should not be drawn. Functional data are not in the US Prescribing Information.

Line graph showing motor function progression in two cohorts. Mobile-optimized graph comparing motor function over time.
†Control subjects were matched for age and corticosteroids. Negative time means less time; positive time means more time. Negative distance means less distance traveled; positive distance means greater distance traveled. Negative NSAA means lower score compared to baseline; positive NSAA means higher score compared to baseline.

Safety

Safety profile evaluated in two 24-week clinical studies

Adverse reactions reported in ≥10% of people with DMD treated with VILTEPSO 80 mg/kg once weekly

Bar chart showing comparative safety data across treatments. Mobile chart showing treatment-emergent adverse events comparison.
*Upper respiratory tract infection includes the following terms: upper respiratory tract infection, nasopharyngitis, and rhinorrhea.
†Injection site reaction includes the following terms: injection site bruising, injection site erythema, injection site reaction, and injection site swelling.

No patients in the clinical trial discontinued treatment as a result of treatment-related Serious Adverse Events (SAEs)

MUSCLE FUNCTION DATA

An extended view of patients on VILTEPSO
over 4 years

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.

Time to stand over 4 years*

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.

Seconds†

Chart comparing time to stand over four years between patient cohorts. Chart comparing time to stand over four years between patient cohorts.
 

Time to run/walk 10 meters over 4 years*

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.

Seconds†

Line chart comparing time to run 10 meters over treatment period. Line chart comparing time to run 10 meters over treatment period.
 

Time to climb four stairs over 4 years*

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.

Seconds†

Graph showing average time change in climbing 4 stairs over time by treatment group. Graph showing average time change in climbing 4 stairs over time by treatment group.
*The control subjects for this trial were matched for age, ambulatory status, corticosteroid use, and geographic location from the CINRG DNHS registry.
†Negative time means less time; positive time means more time.
CINRG=Cooperative International Neuromuscular Research Group; DNHS=Duchenne Natural History Study.

FOUR-YEAR SAFETY DATA

Safety assessment for open-label,
four-year extension study data

Detailed desktop chart showing patient safety outcome measures.Mobile-formatted summary chart of patient 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)

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Meet the VILTEPSO Ambassadors

Hear and read what real patients and their families have to say about life and treatment with VILTEPSO.

See Patient Stories
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REMEMBER, A DOCTOR IS ALWAYS YOUR BEST RESOURCE FOR information about DMD and determining if VILTEPSO could be the right treatment option for you.

For more information about DMD, exon skipping, and VILTEPSO, you can download the Caregiver and Patient Guide

DOWNLOAD THE GUIDE

For suggested questions to ask your doctor about DMD, exon skipping, and VILTEPSO, you can download the Doctor Discussion Guide

DOWNLOAD THE GUIDE

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Indication

VILTEPSO 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.

Important Safety Information

  • In clinical studies, no patients experienced kidney toxicity during treatment with VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be possible. Your doctor may monitor the health of your kidneys before starting and during treatment with VILTEPSO.
  • Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever.
  • You are encouraged to report adverse events related to VILTEPSO. To do so, or for general inquiries, please call NS Pharma Medical Information at 1-866-NSPHARM (1-866-677-4276).

For more information about VILTEPSO, see full Prescribing Information.

For more information about VILTEPSO, see full Prescribing Information.