Healthcare professionals
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 exon 53 skipping*
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.
In Study 1 the efficacy of 20-24 weeks of VILTEPSO was evaluated in ambulant males aged 4 to <10 years
*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.
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.
*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 is given as an 80-mg/kg weekly intravenous infusion
The appropriate dose of VILTEPSO is calculated based upon patient weight, at a recommended weekly dose of 80 mg/kg
VILTEPSO is infused for 60 minutes by a healthcare professional, at home or at a treatment center
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.
For more information about VILTEPSO, see full Prescribing Information.