See efficacy data from VILTEPSO’s 24-week primary study, as well as functional data from the four-year, open-label extension study

VILTEPSO improved dystrophin levels in 100% of amenable patients

At 24 weeks, mean increase in dystrophin expression to nearly 6% of normal with VILTEPSO (80 mg/kg/wk) vs 0.6% at baseline (n=8).

SEE THE CLINICAL DATA

Mason (11 years old), a real VILTEPSO patient and compensated spokesperson

Efficacy & Safety
Exon-Skipping Therapy

VILTEPSO is clinically proven to increase dystrophin levels in amenable patients with DMD

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VILTEPSO corrects out-of-frame mutations by skipping exon 53 of the dystrophin pre-mRNA*

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Exon-skipping therapy is designed to produce a shortened dystrophin protein containing essential functional portions.

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During clinical trials, VILTEPSO increased dystrophin levels in 100% of patients†

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Inside each muscle cell, DNA provides the genetic information to create dystrophin. The first step in this process is the formation of pre-mRNA from DNA. The pre-mRNA is further processed into mRNA, which is then translated into a protein.

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Mean increase in dystrophin expression to nearly 6% of normal with VILTEPSO (80 mg/kg/wk) vs 0.6% at baseline. Please see the study detail shown on the Efficacy & Safety page.

Early diagnosis is instrumental to inform the management of progressive muscle weakness and motor function decline in patients with DMD.

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EFFICACY & SAFETY

Review VILTEPSO’s primary and long-term data

Primary efficacy & safety Four-Year data & safety

EXON-SKIPPING THERAPY

VILTEPSO is an exon 53-skipping technology for amenable DMD patients

Exon skipping is a therapeutic technique that “skips over” an exon next to the deleted exon(s) in patients with DMD.
Watch the video to see how exon skipping works.

Did you know nearly 50 DMD mutations are amenable to treatment with VILTEPSO?

Patients with common mutations including 45-52, 47-52, 48-52, 49-52, 50-52, and 52 are eligible for treatment.

Use our tool to check if your patient is a match
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Duchenne muscular dystrophy (DMD) is caused by not having enough dystrophin. VILTEPSO is an exon-skipping therapy that has been granted accelerated approval based on its demonstrated increase in dystrophin in patients with DMD amenable to exon 53 skipping. Preliminary results from a Phase 3 confirmatory study of VILTEPSO have been received and are undergoing analysis and discussion with the FDA.

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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

  • 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 event, 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.