Viltolarsen
Generic Name: viltolarsen
Brand Names:
Viltepso
11 DESCRIPTION VILTEPSO (viltolarsen) injection is a sterile, preservative-free, aqueous solution for intravenous administration. VILTEPSO is a clear and colorless solution. VILTEPSO is supplied in single-dose vials containing 250 mg/5 mL viltolarsen (50 mg/mL) in 0.9% sodium chloride. Each milliliter of VILTEPSO contains 50 mg viltolarsen and 9 mg sodium chloride in water for injection.
Overview
11 DESCRIPTION VILTEPSO (viltolarsen) injection is a sterile, preservative-free, aqueous solution for intravenous administration. VILTEPSO is a clear and colorless solution. VILTEPSO is supplied in single-dose vials containing 250 mg/5 mL viltolarsen (50 mg/mL) in 0.9% sodium chloride. Each milliliter of VILTEPSO contains 50 mg viltolarsen and 9 mg sodium chloride in water for injection.
Uses
1 INDICATIONS AND USAGE 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 [see Clinical Studies ( 14 )]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. VILTEPSO is an antisense oligonucleotide 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.
Dosage
2 DOSAGE AND ADMINISTRATION Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. ( 2.1 ) Recommended dosage is 80 milligrams per kilogram of body weight once weekly. ( 2.2 ) Administer as an intravenous infusion over 60 minutes. ( 2.2 , 2.4 ) If the volume of VILTEPSO required is less than 100 mL, dilution in 0.9% Sodium Chloride Injection, USP, is required. ( 2.3 ) 2.1 Monitoring to Assess Safety Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VILTEPSO. Consider measurement of glomerular filtration rate prior to initiation of VILTEPSO. Monitoring for kidney toxicity during treatment is recommended.
Side Effects
6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥15% in patients treated with VILTEPSO) were upper respiratory tract infection, injection site reaction, cough, and pyrexia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact NS Pharma at 1-866 NSPHARM (1-866-677-4276) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
Warnings
5 WARNINGS AND PRECAUTIONS Kidney Toxicity: Based on animal data, may cause kidney toxicity. Kidney function should be monitored; creatinine may not be a reliable measure of renal function in DMD patients. ( 5.1 , 13.2) 5.1 Kidney Toxicity Kidney toxicity was observed in animals who received viltolarsen [see Use in Specific Populations ( 8.4 )]. 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. 4 CONTRAINDICATIONS None. None ( 4 )
Pregnancy
8.1 Pregnancy Risk Summary There are no human or animal data available to assess the use of VILTEPSO during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4%, and miscarriage occurs in 15 to 20% of clinically recognized pregnancies.
Storage
16.2 Storage and Handling Store VILTEPSO at 2°C to 8°C (36°F to 46°F). Do not freeze.
Frequently Asked Questions
What is Viltolarsen used for?▼
1 INDICATIONS AND USAGE 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 [see Clinical Studies ( 14 )]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. VILTEPSO is an antisense oligonucleotide 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.
What are the side effects of Viltolarsen?▼
6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥15% in patients treated with VILTEPSO) were upper respiratory tract infection, injection site reaction, cough, and pyrexia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact NS Pharma at 1-866 NSPHARM (1-866-677-4276) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
Can I take Viltolarsen during pregnancy?▼
8.1 Pregnancy Risk Summary There are no human or animal data available to assess the use of VILTEPSO during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4%, and miscarriage occurs in 15 to 20% of clinically recognized pregnancies.
What are the important warnings for Viltolarsen?▼
5 WARNINGS AND PRECAUTIONS Kidney Toxicity: Based on animal data, may cause kidney toxicity. Kidney function should be monitored; creatinine may not be a reliable measure of renal function in DMD patients. ( 5.1 , 13.2) 5.1 Kidney Toxicity Kidney toxicity was observed in animals who received viltolarsen [see Use in Specific Populations ( 8.4 )]. 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. 4 CONTRAINDICATIONS None. None ( 4 )
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Medical Disclaimer
This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.