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MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM › Other drugs for disorders of the musculo-skeletal system
viltolarsen
M09AX12
Forms & Strengths
- Intravenous solution: 250 mg/8 mL (31.25 mg/mL) in single-dose vials
Adult Dosing
- Not applicable (indicated for a pediatric/pediatric-onset genetic disorder)
Pediatric Dosing
- Duchenne muscular dystrophy (DMD) with confirmed mutation amenable to exon 53 skipping: 80 mg/kg intravenously once weekly
- Administer as a constant infusion rate of 15 mg/kg/minute over approximately 60 minutes
Indications
- Treatment of Duchenne muscular dystrophy (DMD) in patients confirmed to have a mutation of the dystrophin gene amenable to exon 53 skipping
Mechanism of Action
- Antisense oligonucleotide designed to bind to exon 53 of the dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations amenable to exon 53 skipping
- Enables production of an internally truncated, functional dystrophin protein in skeletal muscle
Contraindications
- Known hypersensitivity to viltolarsen or any of its excipients
Adverse Reactions
- Upper respiratory tract infection
- Injection site reactions
- Cough
- Pyrexia
- Potential for renal toxicity based on preclinical animal data (monitor renal function)
Drug Interactions
- No formal clinical drug-interaction studies have been conducted
- Renally eliminated drugs may have theoretical competition for renal clearance, though unlikely
Curated Content: Needs Vetting Before Put to Clinical Use