Healthcare professionals

VILTEPSO is an exon-skipping therapy studied in children as young as 4 years

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VILTEPSO is designed to bind to and induce skipping of exon 53 of the dystrophin pre-mRNA, resulting in the production of a shortened dystrophin protein that contains essential functional portions

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An estimated 10% of patients with DMD would be amenable to exon 53 skipping*

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VILTEPSO was discovered through an innovative triple-screening approach designed to identify antisense oligonucleotides with the highest possible exon-skipping efficiency

*Common dystrophin gene mutations eligible for exon 53 skipping include deletions of exons 45-52, 47-52, 48-52, 49-52, 50-52, and 52.

mRNA=messenger RNA.

VILTEPSO is for DMD patients with a confirmed mutation that is amenable to exon 53 skipping

Exon skipping is a genetic technique that, in essence, “skips over” an exon next to the deleted exon(s) in patients with DMD. VILTEPSO is designed to bind to and induce skipping of exon 53 of the dystrophin pre-mRNA, resulting in the production of a shortened dystrophin protein that contains essential functional portions. An estimated 10% of patients with DMD would be amenable to treatment with an exon 53-skipping therapy such as VILTEPSO.

Common deletions that can be treated with VILTEPSO include 45-52, 47-52, 48-52, 49-52, 50-52, and 52 of the dystrophin gene. In the United States, about 2% of patients with DMD have an exon 52 deletion which is amenable to an exon 53-skipping therapy such as VILTEPSO.

Deletions amenable to exon 53 skipping

Deletions Table Deletions Table

VILTEPSO provided significant improvements in dystrophin expression

In Study 1 the efficacy of 20-24 weeks of VILTEPSO was evaluated in ambulant males aged 4 to <10 years

Study 1: Mean increase in dystrophin expression to nearly 6% of normal with VILTEPSO (80 mg/kg/wk) vs 0.6% at baseline

Study 1 Graph 1 Study 1 Graph 1

*P-value for change from baseline at week 25 was statistically significant.

Study design: Phase 2 study with an initial 4-week randomized placebo-controlled period for safety followed by a 20-week open-label treatment period with 2 dose cohorts (N=16) that included patients aged 4 to <10 years with DMD amenable to exon 53 skipping.

  • Efficacy was assessed based on the change from baseline in dystrophin protein level, measured as percentage of the dystrophin level in healthy subjects at Week 25
  • In patients who received VILTEPSO 80 mg/kg once weekly, mean dystrophin levels increased from 0.6% (standard deviation [SD] 0.8) of normal at baseline to 5.9% (SD 4.5) of normal by Week 25, with a mean change in dystrophin of 5.3% (SD 4.5) of normal levels (P=0.01) as assessed by validated Western blot (normalized to myosin heavy chain)
    • > Median change from baseline was 3.8%
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DYSTROPHIN WHERE IT MATTERS: Immunofluorescence staining showed VILTEPSO-induced increases in dystrophin levels were correctly localized to the muscle cell membrane.

100% of patients showed an increase in dystrophin levels with VILTEPSO

Study 1: Dystrophin levels at 25 weeks vs baseline

Study 1 Graph 2 Study 1 Graph 2

Exon 53 skipping was observed, on average, in 43.9% of dystrophin mRNA molecules in patients treated with VILTEPSO (80 mg/kg/wk), as measured by RT-PCR.

Safety profile evaluated in two 24-week clinical studies

Adverse reactions reported in ≥10% of DMD patients treated with VILTEPSO 80 mg/kg once weekly (pooled Studies 1 and 2)

Dosing Table Dosing Table

*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.

VILTEPSO offers a choice of treatment location—at home or at a treatment center

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VILTEPSO is given as an 80-mg/kg weekly intravenous infusion

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The appropriate dose of VILTEPSO is calculated based upon patient weight, at a recommended weekly dose of 80 mg/kg

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VILTEPSO is infused for 60 minutes by a healthcare professional, at home or at a treatment center

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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.

For more information about VILTEPSO, see full Prescribing Information.

Important Safety Information

  • Warnings and Precautions: Kidney toxicity was observed in animals who received viltolarsen. Although kidney toxicity was not observed in the clinical studies with VILTEPSO, the clinical experience with VILTEPSO is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VILTEPSO. Serum creatinine may not be a reliable measure of kidney function in DMD patients.
  • Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. Consider also measuring glomerular filtration rate before starting VILTEPSO. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months.
  • Urine should be free of excreted VILTEPSO for monitoring of urine protein. Obtain urine either prior to VILTEPSO infusion, or at least 48 hours after the most recent infusion. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, which has the potential to generate a false positive result due to cross reaction with any VILTEPSO in the urine. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.
  • Adverse Reactions: The most common adverse reactions include upper respiratory tract infection, injection site reaction, cough, and pyrexia.
  • To report an adverse reaction, 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.