efficacy & safety

VILTEPSO Clinical Data

Learn about VILTEPSO’s efficacy and safety data across three clinical studies.

Diego, a real VILTEPSO patient and compensated spokesperson, and his mother, Leslie.

VILTEPSO Efficacy & Safety Profile

In a clinical study, VILTEPSO significantly increased dystrophin production in DMD patients.

5.9% of Normal Levels

After 24 weeks, VILTEPSO increased  mean dystrophin levels to nearly 6% of normal vs. 0.6% at baseline (n=8).

100% of Patients

After 20-24 weeks of treatment, 100% of patients taking VILTEPSO showed increased dystrophin levels.

Proven Safety Profile

VILTEPSO was elevated in two 24-week studies, two 48-week studies, and a 4-year open-label extension study.*

* In the pivotal 24-week study, the most common adverse reactions with VILTEPSO included upper respiratory tract infection, injection site reaction, cough, and pyrexia.

VILTEPSO Clinical Studies

Read the VILTEPSO study design details and the results of its clinical studies.

Pivotal 24-Week Study

A placebo-controlled, dose-finding study with ambulant males aged 4 to <10 years with DMD amenable to exon 53 skipping, who received a once-weekly infusion of 40 or 80 mg/
kg VILTEPSO or placebo for four weeks, followed by a 20-week, open-label assessment of efficacy and safety.

key FINDING

VILTEPSO significantly increased dystrophin production

Long-Term Extension Study

After completing the 24-week study, all patients chose to enroll in a 192-week open-label extension study for ongoing safety and efficacy evaluation.

key Insight

Functional efficacy assessments were conducted every 12 weeks

Pulmonary & Upper Limb Function

Galactic53 was a Phase 2, open-label, multicenter study of VILTEPSO (80 mg/kg/wk) in ambulatory and non-ambulatory males aged 8 and older with DMD amenable to exon 53-skipping therapy.

key Insight

First VILTEPSO study to evaluate pulmonary function

*All patients are real VILTEPSO patients and compensated spokespeople.

VILTEPSO Safety Profile

In two 24-week clinical studies, adverse reactions reported in >10% of patients were upper respiratory tract infection, injection site reaction, cough, pyrexia, contusion, arthralgia, diarrhea, vomiting, abdominal pain, ejection fraction decreased, and urticaria.

Key finding

No treatment-related serious adverse events, drug-related treatment-emergent adverse events, discontinuations, or deaths occurred

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Dosing & Administration

Learn about prescribing, dosing, and administering VILTEPSO.

About VILTEPSO

Learn about how VILTEPSO treats
DMD.