Section 16 · Monograph
Golodirsen
Reviewed Nov 2026FDA label
Vyondys 53Antisense oligonucleotide (PMO)
- Forms
- IV: 50mg/mL single-dose vials
- Dose
- 30mg/kg IV once weekly (exon 53 amenable DMD; accelerated approval)
- MOA
- Skips dystrophin exon 53
Indications
- Duchenne muscular dystrophy with a confirmed DMD mutation amenable to exon 53 skipping (accelerated approval based on dystrophin increase; clinical benefit not yet confirmed)FDA-labeled
Mechanism of Action
- Phosphorodiamidate morpholino oligomer that binds exon 53 of dystrophin pre-mRNA, causing exon skipping and production of a truncated functional dystrophin
Full Dosing
Adult / usual
- 30mg/kg IV infusion once weekly (over about 35-60 minutes)
- Obtain baseline kidney function before starting
Contraindications
- None listed in labeling
Drug Interactions
- No significant drug interactions identified
Adverse Effects
- Headache, fever, cough, vomiting, abdominal pain
- Hypersensitivity reactions
- Kidney toxicity (including glomerulonephritis)
- Infusion-related reactions
Monitoring Parameters
- Kidney function (urine dipstick, serum cystatin C, urine protein-to-creatinine ratio) at baseline and periodically
- Hypersensitivity during infusion
Pregnancy & Lactation
- Limited data; weigh risks and benefits