Version May 2024
Duchenne muscular dystrophy : IV: 30 mg/kg once weekly.
There are no dosage adjustments provided in the manufacturer's labeling; monitor closely.
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
Hypersensitivity reactions: Consider slowing infusion or interrupting therapy.
Refer to adult dosing.
(For additional information see "Golodirsen: Pediatric drug information")
Note: Determine serum cystatin C, urine dipstick (proteinuria), and urine protein-to-creatinine ratio prior to therapy; also consider baseline GFR using an exogenous filtration marker.
Duchenne muscular dystrophy (DMD): Children and Adolescents: IV: 30 mg/kg/dose once weekly. Note: Dosing based on clinical trials in pediatric patients aged 6 to 13 years at study entry.
Dosing adjustment for toxicity:
Hypersensitivity reactions: Consider slowing infusion or interrupting therapy.
There are no dosage adjustments provided in the manufacturer's labeling. Renal clearance is reduced in non-Duchenne muscular dystrophy (DMD) adult patients with renal impairment; patients with known kidney impairment should be closely monitored; however, creatinine is not a reliable measurement of kidney function due to the reduced skeletal muscle mass in patients with DMD. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be monitored; consult pediatric nephrologist if persistent increase in serum cystatin C or proteinuria develops.
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.
>10%:
Central nervous system: Headache (41%), falling (29%)
Gastrointestinal: Abdominal pain (27%), vomiting (27%), nausea (20%)
Respiratory: Cough (27%), nasopharyngitis (27%)
Miscellaneous: Fever (41%)
1% to 10%:
Cardiovascular: Tachycardia (>5%)
Central nervous system: Dizziness (>5%), pain (>5%)
Dermatologic: Excoriation of skin (>5%)
Gastrointestinal: Constipation (>5%), diarrhea (>5%)
Hematologic & oncologic: Bruise (>5%)
Hypersensitivity: Seasonal allergy (>5%)
Infection: Influenza (>5%)
Local: Catheter-site reaction (>5%), infusion-site pain (>5%)
Neuromuscular & skeletal: Back pain (>5%), bone fracture (>5%), sprain (>5%)
Otic: Otic infection (>5%)
Respiratory: Oropharyngeal pain (>5%), rhinitis (>5%)
Frequency not defined:
Dermatologic: Dermatitis, exfoliation of skin, pruritus, skin rash, urticaria
Hypersensitivity: Hypersensitivity reaction
There are no contraindications listed in the manufacturer's labeling.
Concerns related to adverse effects:
• Hypersensitivity: Hypersensitivity reactions (including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation) have been reported with use; manage appropriately and consider slowing the infusion or interrupting therapy.
• Kidney toxicity: Kidney toxicity, including potentially fatal glomerulonephritis, has been observed with some antisense oligonucleotides. Creatinine may not be a reliable measure of kidney function in patients with Duchenne muscular dystrophy. Refer to nephrologist if persistent increases in serum cystatin C or proteinuria occur.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution, Intravenous [preservative free]:
Vyondys 53: 100 mg/2 mL (2 mL)
No
Solution (Vyondys 53 Intravenous)
100 mg/2 mL (per mL): $960.00
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IV: Dilution required prior to administration. Administer by IV infusion over 35 to 60 minutes immediately after dilution through a 0.2-micron, low protein-binding, in-line filter attached to the primary IV tubing; complete infusion within 4 hours (stable for 4 hours at room temperature). Use of a topical anesthetic cream on the infusion site may be considered prior to administration. Flush IV access line with NS before and after infusion. Do not mix with other medications or infuse other medications concomitantly via the same IV access line. Consider slowing the infusion or interrupting therapy for hypersensitivity reactions. Discard unused portion.
IV: Must be diluted prior to administration. Administer by IV infusion over 35 to 60 minutes through a 0.2-micron, low protein binding, in-line filter attached to the primary IV tubing; infusion must be completed within 4 hours of preparation. Flush IV access line with NS prior to and after infusion; may consider application of a topical anesthetic cream to the infusion site prior to administration. Do not mix with other medications or infuse other medications concomitantly via the same IV access line. If hypersensitivity reaction occurs, consider slowing the infusion or interrupting therapy. If a dose is missed, administer as soon as possible after the scheduled time.
Duchenne muscular dystrophy: Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Golodirsen may be confused with Goldenseal, Golden Seal, gold sodium thiomalate.
None known.
There are no known significant interactions.
Animal reproduction studies or studies in females have not been conducted.
It is not known if golodirsen is present in breast milk.
Studies in females have not been conducted. According to the manufacturer, the decision to breastfeed during therapy should consider the risk of infant exposure, the benefits of breastfeeding to the infant, and benefits of treatment to the mother.
Proteinuria by dipstick urinalysis (baseline and monthly); serum cystatin C and urine protein-to-creatinine ratio (baseline and every 3 months); urine samples should be obtained prior to infusion or at least 48 hours after the most recent infusion and use laboratory tests that does not use reagent pyrogallol red (may cross react with golodirsen leading to false positive); GFR using an exogenous filtration marker (baseline); hypersensitivity reactions during infusion; kidney toxicity.
Binds to exon 53 of dystrophin pre-messenger RNA (mRNA), resulting in exclusion of this exon during mRNA processing. Exon 53 skipping allows for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
Distribution: Vdss: 0.67 L/kg.
Protein binding: Plasma: 33% to 39%.
Half-life elimination: 3.4 hours.
Excretion: Urine (mostly unchanged).
Altered kidney function: In non-Duchenne muscular dystrophy adults with chronic kidney disease (CKD), AUC increased ~1.2-fold and 1.9-fold in stage 2 CKD and stage 3 CKD, respectively. Cmax increased 1.2-fold in patients with stage 3 CKD.
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