golodirsen (Rx)

Brand and Other Names:Vyondys 53
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Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

injection, concentrated solution

  • 100mg/2mL (50mg/mL) single-dose vial

Duchenne Muscular Dystrophy

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

30 mg/kg IV qWeek

Dosage Modifications

Renal impairment

  • Renal clearance is reduced in non-DMD adults with renal impairment, based on estimated glomerular filtration rate (eGFR) calculated using the Modification of Diet and Renal Disease (MDRD) equation
  • No specific dosage adjustment can be recommended for patients with DMD who have renal impairment based on eGFR
  • Closely monitor patients with known renal function impairment during treatment

Hepatic impairment

  • Not studied

Dosing Considerations

Measure GFR before initiating

Monitoring for renal toxicity during treatment is recommended

Dosage Forms & Strengths

injection, concentrated solution

  • 100mg/2mL (50mg/mL) single-dose vial

Duchenne Muscular Dystrophy

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

30 mg/kg IV qWeek

Dosage Modifications

Renal impairment

  • Renal clearance is reduced in non-DMD adults with renal impairment, based on estimated glomerular filtration rate (eGFR) calculated using the Modification of Diet and Renal Disease (MDRD) equation
  • No specific dosage adjustment can be recommended for patients with DMD who have renal impairment based on eGFR
  • Closely monitor patients with known renal function impairment during treatment

Hepatic impairment

  • Not studied

Dosing Considerations

Measure GFR before initiating

Monitoring for renal toxicity during treatment is recommended

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Adverse Effects

>10%

Headache (41%)

Pyrexia (41%)

Fall (29%)

Abdominal pain (27%)

Nasopharyngitis (27%)

Cough (27%)

Vomiting (27%)

Nausea (20%)

1-10%

>5%

  • Administration site pain
  • Back pain
  • Pain
  • Diarrhea
  • Dizziness
  • Ligament sprain
  • Contusion
  • Influenza
  • Oropharyngeal pain
  • Rhinitis
  • Skin abrasion
  • Ear infection
  • Seasonal allergy
  • Tachycardia
  • Catheter site-related reaction
  • Constipation
  • Fracture

Frequency Not Defined

Hypersensitivity

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Warnings

Contraindications

None

Cautions

Hypersensitivity

  • Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation, reported, some requiring treatment
  • If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting therapy

Renal toxicity

  • Renal toxicity was observed in animals
  • Although renal toxicity was not observed in the clinical studies, renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides
  • Monitor renal function during therapy
  • Because of the effect of reduced skeletal muscle mass on creatinine measurements, serum creatinine may not be a reliable measure of renal function in patients with DMD
  • Measure GFR by 24-hr urine collection before initiating therapy
  • Monthly monitoring for proteinuria by dipstick urinalysis and monitoring of serum cystatin C q3Months is recommended
  • If confirmed dipstick proteinuria of 2+ or greater or elevated serum cystatin C, a 24-hr urine collection to quantify proteinuria and assess GFR should be performed
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Pregnancy & Lactation

Pregnancy

No human or animal data are available to assess use during pregnancy

Lactation

No human or animal data are available to assess effects on milk production, presence in milk, or effects on the breastfed infant

Pregnancy Categories

A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

NA: Information not available.

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Pharmacology

Mechanism of Action

Binds to exon 53 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping

Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping

Absorption

Systemic exposure increases proportionally with dose, with minimal accumulation

Distribution

Protein bound:33-39%

Vd: 668 mL/kg

Metabolism

Metabolically stable; no metabolites detected in plasma or urine

Elimination

Half-life: 3.4 hr

Plasma clearance: 346 mL/hr/kg

Mostly excreted unchanged in urine

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Administration

IV Preparation

Preservative-free concentrated solution that requires dilution before administration

Inspect vials visually for particulate matter and discoloration; solution should appear as a clear to slightly opalescent, colorless liquid

Use aseptic technique

Allow vials to warm to room temperature

Gently invert vials to mix contents; do not shake

Withdraw calculated dose from vial(s) and dilute in 0.9% NaCl for a total volume of 100-150 mL; gently invert 2-3 times to mix; do not shake

Visually inspect diluted solution for particulates

Administer immediately after dilution; complete infusion within 4 hr of dilution

IV Administration

Consider topical anesthetic cream application to the infusion site before administration

Flush IV access line with 0.9% NaCl before and after infusion

Infuse IV over 35-60 minutes

Do not mix with other medication or infuse other medication concomitantly vial same IV access line

Missed dose: May be administered as soon as possible after the scheduled dose

Storage

Unopened vials

  • Refrigerate at 2-8ºC (36-46ºF)
  • Do not freeze
  • Store in original carton until ready for use to protect from light

Diluted solution

  • If immediate use is not possible, may store for up to 24 hr at 2-8ºC (36-46ºF)
  • Do not freeze
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Formulary

FormularyPatient Discounts

Adding plans allows you to compare formulary status to other drugs in the same class.

To view formulary information first create a list of plans. Your list will be saved and can be edited at any time.

Adding plans allows you to:

  • View the formulary and any restrictions for each plan.
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  • Compare formulary status to other drugs in the same class.
  • Access your plan list on any device – mobile or desktop.

The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

Tier Description
1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
NC NOT COVERED – Drugs that are not covered by the plan.
Code Definition
PA Prior Authorization
Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
QL Quantity Limits
Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
ST Step Therapy
Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
OR Other Restrictions
Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.