Golodirsen
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Omid Afkhami-Ardakani
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Overview
Golodirsen is an Antisense oligonucleotide that is FDA approved for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.. Common adverse reactions include Common Adverse Reactions (≥20% incidence): Headache, fever, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea..
Adult Indications and Dosage
FDA-Labeled Indications and Dosage (Adult)
DMD primarily affects pediatric patients.
Off-Label Use and Dosage (Adult)
Guideline-Supported Use
There is limited information regarding Off-Label Guideline-Supported Use of Golodirsen in adult patients.
Non–Guideline-Supported Use
There is limited information regarding Off-Label Non–Guideline-Supported Use of Golodirsen in adult patients.
Pediatric Indications and Dosage
FDA-Labeled Indications and Dosage (Pediatric)
for treatment of Duchenne muscular dystrophy in pediatric patients with a confirmed mutation amenable to exon 53 skipping: 30 mg/kg administered once weekly as a 35 to 60-minute intravenous infusion.
Off-Label Use and Dosage (Pediatric)
Guideline-Supported Use
There is limited information regarding Off-Label Guideline-Supported Use of Golodirsen in pediatric patients.
Non–Guideline-Supported Use
There is limited information regarding Off-Label Non–Guideline-Supported Use of Golodirsen in pediatric patients.
Contraindications
Known hypersensitivity to golodirsen or any of its excipients.
Warnings
Hypersensitivity Reactions: Monitor for signs and symptoms; discontinue if severe reactions occur.
Renal Toxicity: Monitor renal function; kidney toxicity observed in animal studies.
Adverse Reactions
Clinical Trials Experience
Headache, fever, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea.
Postmarketing Experience
There is limited information regarding Golodirsen Postmarketing Experience in the drug label.
Drug Interactions
No formal drug interaction studies have been conducted.
Use in Specific Populations
Pregnancy
Pregnancy Category (FDA):
There is no FDA guidance on usage of Golodirsen in women who are pregnant.
Pregnancy Category (AUS):
There is no Australian Drug Evaluation Committee (ADEC) guidance on usage of Golodirsen in women who are pregnant.
Labor and Delivery
There is no FDA guidance on use of Golodirsen during labor and delivery.
Nursing Mothers
There is no FDA guidance on the use of Golodirsen in women who are nursing.
Pediatric Use
Approved for use in patients with DMD amenable to exon 53 skipping.
Geriatic Use
There is no FDA guidance on the use of Golodirsen in geriatric settings.
Gender
There is no FDA guidance on the use of Golodirsen with respect to specific gender populations.
Race
There is no FDA guidance on the use of Golodirsen with respect to specific racial populations.
Renal Impairment
There is no FDA guidance on the use of Golodirsen in patients with renal impairment.
Hepatic Impairment
There is no FDA guidance on the use of Golodirsen in patients with hepatic impairment.
Females of Reproductive Potential and Males
There is no FDA guidance on the use of Golodirsen in women of reproductive potentials and males.
Immunocompromised Patients
There is no FDA guidance one the use of Golodirsen in patients who are immunocompromised.
Administration and Monitoring
Administration
Dilute the calculated dose in 0.9% Sodium Chloride Injection, USP, to a total volume of 100 to 150 mL.
Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter.
Monitoring
Assess glomerular filtration rate prior to initiation.
Monitor for renal toxicity during treatment.
IV Compatibility
There is limited information regarding the compatibility of Golodirsen and IV administrations.
Overdosage
There is limited information regarding Golodirsen overdosage. If you suspect drug poisoning or overdose, please contact the National Poison Help hotline (1-800-222-1222) immediately.
Pharmacology
There is limited information regarding Golodirsen Pharmacology in the drug label.
Mechanism of Action
Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing. This exon skipping restores the reading frame of the DMD gene, allowing for the production of a truncated, yet functional, dystrophin protein.
Structure
There is limited information regarding Golodirsen Structure in the drug label.
Pharmacodynamics
In clinical studies, golodirsen treatment led to increased dystrophin production in skeletal muscle. After 48 weeks of treatment, patients exhibited a mean increase in dystrophin levels from 0.10% to 1.02% of normal, as measured by Western blot analysis.
Pharmacokinetics
Absorption: Administered via intravenous infusion; bioavailability is assumed to be 100%.
Distribution: The mean steady-state volume of distribution is approximately 668 mL/kg.
Protein Binding: Approximately 33% to 39%; not concentration-dependent.
Metabolism: Golodirsen is not metabolized and is excreted unchanged.
Elimination: Primarily excreted unchanged in the urine.
Half-life: Approximately 3.5 hours.
Nonclinical Toxicology
There is limited information regarding Golodirsen Nonclinical Toxicology in the drug label.
Clinical Studies
There is limited information regarding Golodirsen Clinical Studies in the drug label.
How Supplied
Golodirsen is supplied as a concentrated solution for intravenous infusion:
100 mg/2 mL (50 mg/mL) in single-dose vials.
Storage
Storage Conditions: Store at 2°C to 8°C (36°F to 46°F).
Handling: Do not freeze. Protect from light.
Images
Drug Images
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Package and Label Display Panel
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Patient Counseling Information
Administration: Golodirsen is administered via weekly intravenous infusions.
Potential Side Effects: Common side effects include headache, fever, abdominal pain, and nausea.
Renal Monitoring: Regular monitoring of kidney function is recommended during treatment.
Hypersensitivity Reactions: Patients should report any signs of hypersensitivity, such as rash or fever.
Precautions with Alcohol
Alcohol-Golodirsen interaction has not been established. Talk to your doctor about the effects of taking alcohol with this medication.
Brand Names
Vyondys 53®
Look-Alike Drug Names
There is limited information regarding Golodirsen Look-Alike Drug Names in the drug label.
Drug Shortage Status
Price
References
The contents of this FDA label are provided by the National Library of Medicine.