Vyondys 53

  • Miscellaneous musculoskeletal disorders

Vyondys 53 Generic Name & Formulations

General Description

Golodirsen 50mg/mL; soln for IV infusion after dilution; preservative-free.

Pharmacological Class

Antisense oligonucleotide.

How Supplied

Single-dose vial (2mL)—1


Generic Availability


Vyondys 53 Indications


Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Vyondys 53 Dosage and Administration

Adults and Children

Give as IV infusion over 35–60mins. 30mg/kg once weekly.

Vyondys 53 Contraindications

Not Applicable

Vyondys 53 Boxed Warnings

Not Applicable

Vyondys 53 Warnings/Precautions


Monitor renal function. Measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio, and consider measuring GFR prior to initiation. During treatment, monitor urine dipstick monthly, and serum cystatin C and urine protein-to-creatinine ratio every 3 months. To test urine protein: may use urine samples obtained prior to infusion or at least 48hrs after the most recent infusion. Refer to a pediatric nephrologist if a persistent increase in serum cystatin C or proteinuria is detected. Interrupt or slow infusion if hypersensitivity reaction occurs. Renal impairment: monitor closely. Pregnancy. Nursing mothers.

Vyondys 53 Pharmacokinetics

See Literature

Vyondys 53 Interactions


Do not mix or infuse with other drugs concomitantly via same IV access line. May cause false (+) results for urine protein with using pyrogallol red reagent.

Vyondys 53 Adverse Reactions

Adverse Reactions

Headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, nausea; renal toxicity, hypersensitivity reactions.

Vyondys 53 Clinical Trials

See Literature

Vyondys 53 Note

Not Applicable

Vyondys 53 Patient Counseling

See Literature