Vyondys-53 (golodirsen)
Duchenne Muscular Dystrophy-Exon Skipping Antisense Oligonuc Intravenous Vial (Ml)
Last reviewed · Source: First Databank (FDB) drug data
Overview
Golodirsen is an antisense oligonucleotide that binds to exon 53 of dystrophin premessenger RNA (pre-mRNA).
Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass; the drug increases the production of dystrophin protein in patients with Duchenne muscular dystrophy (DMD) through an exon skipping mechanism. In patients with DMD, frameshift mutations in the dystrophin gene disrupt the translational reading frame, resulting in the production of a nonfunctional dystrophin protein; approximately 63% of these mutations occur between exons 45 and 55. Golodirsen selectively binds to and promotes exclusion of exon 53 in the dystrophin premessenger RNA (pre-mRNA), thus restoring the open reading frame and enabling production of an internally truncated, but partially functional, dystrophin protein.
This truncated form of dystrophin is similar to the dystrophin produced in Becker muscular dystrophy (BMD), a less severe type of muscular dystrophy.
Following IV administration, peak plasma concentrations of golodirsen are observed at the end of the infusion at a median time of approximately 1 hour. Exposure increases in a dose-proportional manner with minimal accumulation following once-weekly dosing. Golodirsen plasma protein binding ranges from 33-39% and is independent of concentration.
Golodirsen is not metabolized by hepatic microsomal enzymes and is principally excreted in the urine as unchanged drug. The elimination half-life of the drug is 3.4 hours.
USES: Golodirsen is used to treat a certain inherited muscle disorder (Duchenne muscular dystrophy-DMD). This disorder is caused by a lack of a certain muscle protein (dystrophin). This medication can increase the amount of dystrophin in your body.
Generic name: golodirsen
Pronunciation: (GOE-loe-DIR-sen)
Drug class: Duchenne Muscular Dystrophy-Exon Skipping Antisense Oligonuc
Therapeutic class: Locomotor System
Reference product & biosimilars
No reference, biosimilar or interchangeable associated with this drug.
Today’s intelligence on Vyondys-53
Indications
The following indications are listed for Vyondys-53:
- Duchenne Muscular Dystrophy Due To Genetic Mutation Amenable To Exon 53 Skipping Therapy
Also known as: Dmd Amenable To Exon Skipping Therapy - Exon 53, Dmd Due To Genetic Mutation Amenable To Exon 53 Skipping, Duchenne Muscular Dystrophy Amenable To Exon 53 Skipping.
Dosing
The recommended adult and pediatric dosage of golodirsen for the treatment of Duchenne muscular dystrophy (DMD) is 30 mg/kg once weekly by IV infusion.
If a dose is missed, the missed dose may be administered as soon as possible.
No dosing information is available for this tab.
| Drug label | Dosing type | Instructions |
|---|---|---|
| Vyondys-53 100 Mg/2 Ml Vial | Maintenance | Infuse 30 mg/kg over 35-60 minute(s) by intravenous route once weekly. |
No dosing information is available for this tab.
Drug interactions
In vitro data indicate that golodirsen has a low potential for drug interactions.
There are no contraindication interactions for this drug.
There are no severe interactions for this drug.
There are no moderate interactions for this drug.
Contraindications
*History of a serious hypersensitivity reaction to golodirsen or any of its inactive ingredients. Anaphylaxis has been reported.
There are no contraindication contraindications for this drug.
There are no severe contraindications for this drug.
- Kidney disease with reduction in glomerular filtration rate (GFr)
Adverse reactions
Adverse effects reported in at least 20% of patients receiving golodirsen in clinical studies and more frequently than placebo include headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea.
Rare or Very Rare
- Anaphylaxis
- Hypersensitivity drug reaction
More Frequent
- Accidental fall
- Acute abdominal pain
- Cough
- Fever
- Headache disorder
- Nausea
- Pharyngitis
- Vomiting
Less Frequent
- Back pain
- Bruising
- Constipation
- Diarrhea
- Dizziness
- Fracture
- Infection of ear
- Influenza
- Injection site sequelae
- Pain
- Pain in oropharynx
- Rhinitis
- Sprains and strains
- Tachycardia
Rare or Very Rare
- Allergic dermatitis
- Exfoliative dermatitis
- Pruritus of skin
- Skin rash
- Urticaria
Precautions
No enhanced messaging available.
Golodirsen is labeled for use in pediatric patients with DMD who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.
In juvenile animal studies, mortality attributed to renal impairment or failure occurred at the highest dosage of golodirsen (900 mg/kg per week) tested. In surviving animals, dose-dependent increases in the incidence and severity of renal tubular effects (e.g., degeneration and/or regeneration, fibrosis, vacuolation, dilatation) associated with clinical pathology changes were observed, even with the lowest dose tested. Golodirsen exposure at the lowest dose tested in animals was about 2.5 times greater than the exposure at the recommended human dose. No evidence of developmental toxicity (e.g., on the male reproductive system, neurobehavioral development, or immune function) was observed.
Golodirsen has not been studied in female patients. It is not known whether the drug is associated with risk if used in pregnant women.
Golodirsen has not been studied in female patients. It is not known whether golodirsen is distributed into milk or if the drug has any effects on nursing infants or on milk production. The known benefits of breast-feeding should be considered along with the mother's clinical need for golodirsen and any potential adverse effects of the drug or disease on the infant.
The manufacturer states that there is no geriatric experience with golodirsen as DMD is largely a disease of children and young adults.
There is no experience with golodirsen in geriatric patients; DMD is generally a disease of children and young adults.
ICD codes
| Code | Type | Description |
|---|---|---|
| Duchenne muscular dystrophy due to genetic mutation amenable to exon 53 skipping therapy | ||
| G71.01 | ICD10CM | Duchenne or becker muscular dystrophy |
ICD code information from First Databank (FDB).
Formulary status
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Last reviewed · Source: First Databank (FDB) drug data
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