FOR HEALTHCARE PROFESSIONALS
ABOUT VILTEPSO
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*
VILTEPSO was discovered through an innovative triple-screening approach designed to identify antisense oligonucleotides with the highest possible exon-skipping efficiency
mRNA=messenger RNA.
For children with DMD, early diagnosis can be instrumental to inform the management of progressive muscle deterioration and motor function decline
EXON-SKIPPING THERAPY
Exon skipping is a genetic technique that, in essence, “skips over” an exon next to the deleted exon(s) in patients with DMD.
SEE HOW EXON SKIPPING WORKS
EFFICACY
Study 1: Mean increase in dystrophin expression to nearly 6% of normal with VILTEPSO (80 mg/kg/wk) vs 0.6% at baseline
*
P-value for change from baseline at week 25 was statistically significant.
Study design: A 2-period, North American dose-finding study with ambulant males aged 4 to <10 years with a confirmed mutation of the DMD gene amenable to exon 53 skipping who were receiving a stable dose of corticosteroids for ≥3 months (N=16). Primary study endpoints were safety, tolerability, and dystrophin protein production (Western blot). Secondary study endpoints were other dystrophin measures (RT-PCR, MS, and IFS) and timed motor function tests (6MWT, TTCLIMB, TTRW, TTSTAND) and NSAA. Patients were treated for 24 weeks, and then all patients elected to continue in an open-label, up to 192-week extension study for further assessment.
IFS=immunofluorescence staining; MS=mass spectrometry; NSAA=North Star Ambulatory Assessment; QMT=quantitative muscle testing;
RT-PCR=reverse transcriptase-polymerase chain reaction; 6MWT=6-minute walk test; SD=standard deviation; TTCLIMB=time to climb 4 stairs;
TTRW=time to run/walk 10 meters; TTSTAND=time to stand from supine.
DYSTROPHIN WHERE IT MATTERS: Immunofluorescence staining showed VILTEPSO-induced increases in dystrophin levels were correctly localized to the muscle cell membrane, where dystrophin is needed to support muscle health.
Dystrophin expression (% of normal) normalized to myosin heavy chain
†
Mean dystrophin levels as assessed by mass spectrometry (normalized to filamin C) increased from 0.6% (SD 0.2) of normal at baseline to 4.2% (SD 3.7) of normal by week 25, with a mean change in dystrophin of 3.7% (SD 3.8) of normal levels (nominal P=0.03, not adjusted for multiple comparisons); the median change from baseline was 1.9%.
Functional tests were secondary endpoints of study 201 and were compared to Duchenne natural history data, which is not considered an adequate comparator arm. Functional data are not in the US Prescribing Information, and therefore definitive conclusions should not be drawn.
‡
The control subjects for this trial were matched for age and corticosteroids from the CINRG-DNHS registry.
§
Negative velocity means lower rate of velocity; positive velocity means higher rate of velocity.
‖
Negative time means less time; positive time means more time.
¶
Negative number means less distance traveled; positive number means greater distance traveled.
#
Negative number means a lower score relative to baseline; positive number means a higher score relative to baseline.
CINRG=Cooperative International Neuromuscular Research Group; DNHS=Duchenne Natural History Study.
SAFETY
Adverse reactions reported in ≥10% of DMD patients treated with VILTEPSO 80 mg/kg once weekly
*
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.
DOSING
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, either at the patient’s home or at a treatment center
HOW TO DOSE AND ADMINISTER A VILTEPSO INFUSION
VILTEPSO is available as single-dose vials containing 250 mg/5mL (50 mg/mL) solution. The appropriate dose is based on patient’s body weight, with the recommended dosage of VILTEPSO 80 mg/kg administered once weekly as a 60-minute intravenous infusion.
Only odd kg body weight provided for illustrative purposes.
If volume of VILTEPSO required is <100 mL, dilution in 0.9% sodium chloride for injection, USP, is required such that the total volume in the infusion bag is 100 mL. If the volume of VILTEPSO to be infused is ≥100 mL, dilution is not required.
†
The actual number of theoretical vials required is 15.04.
When less than 100 mL of VILTEPSO is required:
1.
Withdraw from the 100‑mL infusion bag a volume of 0.9% sodium chloride for injection, USP, equivalent to the calculated volume of VILTEPSO solution that will be added.
2.
Withdraw the calculated volume of VILTEPSO solution from the appropriate number of vials, and inject into the infusion bag, such that the total volume in the bag is 100 mL.
When 100 mL or more of VILTEPSO is required:
Withdraw the calculated volume of VILTEPSO solution from the appropriate number of vials, and inject into an empty infusion bag. Further dilution is not required if the volume of VILTEPSO is 100 mL or more.
Infusion should begin no more than 5 hours after preparation of VILTEPSO and be completed within 6 hours of preparation (allowing for 1 hour of infusion time) if diluted solution is stored at 20°C to 26°C (68°F to 79°F).
VILTEPSO is administered via intravenous infusion using a peripheral or central venous catheter. Flush the intravenous access line with 0.9% sodium chloride for injection, USP, after infusion. Filtration of VILTEPSO is not required.
Do not mix other medications with VILTEPSO or infuse other medications concomitantly via the same intravenous access line.
This guide serves as a quick reference for information on appropriate VILTEPSO dose and number of corresponding VILTEPSO vials, as well as corresponding volumes of saline, VILTEPSO, and total volume of VILTEPSO solution, for select patient body weights.
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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.