viltolarsen

Drug name: ビルトラルセン

Trade name: Viltepso

English name: viltolarsen

Indications

VILTPSO is an antisense oligonucleotide suitable for the treatment of DMD gene mutations in patients with Duchenne muscular dystrophy, which is suitable for exon 53 skipping. This indication was approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILEPSO. Continued approval for this indication may be contingent on verification and description of clinical benefit in confirmatory trials.

Specification dosage

250mg/5mL per bottle

Usage and Dosage

The recommended dose of VILEPSO is to calculate the total dose of VILEPSO based on the patient's weight and the recommended dose of 80 mg/kg, once a week, and infuse intravenously for 60 minutes.

If a dose of VILEPSO is missed, give it as soon as possible after the scheduled dosing time.

Serum cystatin C, urine dipstick and urine protein/creatinine ratio should be measured before starting VILEPSO. Consider measuring glomerular filtration rate before starting VLTEPSO. Monitoring for nephrotoxicity during treatment is recommended. Obtain a urine sample before infusing VILEPSO or at least 48 hours after the most recent infusion.

Common side effects

The most common adverse reactions (occurring in ≥15% of patients treated with VILEPSO) are upper respiratory tract infection, injection site reaction, cough, and pyrexia.

Notes

Nephrotoxicity has been observed in animals receiving vitolasen. Although nephrotoxicity was not observed in clinical studies using VLTEPSO, clinical experience with VLTEPSO is limited, and nephrotoxicity, including potentially fatal glomerulonephritis, has been observed following administration of certain antisense oligonucleotides. Renal function should be monitored in patients taking VILEPSO. Serum creatinine may not be a reliable measure of renal function in patients with DMD due to the effect of reduced skeletal muscle mass on creatinine measurements. Serum cystatin C, urine dipstick and urine protein/creatinine ratio should be measured before starting VILEPSO. Before initiating VILEPSO, also consider measuring glomerular filtration rate using exogenous filtration markers. During treatment, urine dipsticks were monitored monthly, and serum cystatin C and urine protein/creatinine ratio were measured every three months. Only urine expected to be VILEPSO-free should be used to monitor urine protein. Urine obtained on the day of VILEPSO infusion prior to infusion or urine obtained at least 48 hours after the most recent infusion may be used. Alternatively, use a laboratory test that does not use pyrogallol red reagent as this reagent has the potential to cross-react with any VILEPSO excreted in the urine, resulting in a false-positive result for urine protein.