Exondys 51 (eteplirsen)
Duchenne Muscular Dystrophy-Exon Skipping Antisense Oligonuc Intravenous Vial (Ml)
Last reviewed · Source: First Databank (FDB) drug data
Overview
Eteplirsen is an antisense oligonucleotide that binds to exon 51 of dystrophin premessenger RNA (pre-mRNA).
Eteplirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass; the drug increases 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. Eteplirsen selectively binds to and promotes exclusion of exon 51 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. Preclinical and clinical studies have demonstrated the ability of eteplirsen to induce dystrophin protein expression in skeletal muscles.
Following single or multiple IV infusions of eteplirsen at doses of 0.5 to 50 mg/kg per week, peak plasma concentrations were observed near the end of the infusion at 1.1-1.2 hours. Pharmacokinetics were approximately dose proportional and linear in multiple-dose studies with no substantial drug accumulation following weekly infusion of the drug across this dose range. In vitro studies have suggested that plasma protein binding of eteplirsen ranges from 6-17%.
Eteplirsen does not appear to be metabolized by hepatic microsomal enzymes. Renal clearance accounts for approximately two-thirds of an administered dose within 24 hours of IV infusion. The elimination half-life of the drug is 3-4 hours.
USES: Eteplirsen 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: eteplirsen
Pronunciation: (e-TEP-lir-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 Exondys 51
Indications
The following indications are listed for Exondys 51:
- Duchenne Muscular Dystrophy Due To Genetic Mutation Amenable To Exon 51 Skipping Therapy
Also known as: Dmd Amenable To Exon Skipping Therapy - Exon 51, Dmd Due To Genetic Mutation Amenable To Exon 51 Skipping Therapy, Duchenne Muscular Dystrophy Amenable To Exon 51 Skipping Therapy.
Dosing
The recommended dosage of eteplirsen 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 |
|---|---|---|
| Exondys-51 100 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 eteplirsen 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
*None.
There are no contraindication contraindications for this drug.
There are no severe contraindications for this drug.
- No disease contraindications
Adverse reactions
Adverse effects reported in at least 35% of patients receiving eteplirsen in clinical studies and more frequently than with placebo include balance disorder and vomiting.
Rare or Very Rare
- Bronchospastic pulmonary disease
- Hypersensitivity drug reaction
- Hypotension
More Frequent
- Ataxia
- Contact dermatitis
- Vomiting
Less Frequent
- Bruising
- Injection site sequelae
- Skin rash
- Upper respiratory infection
Rare or Very Rare
- Chest pain
- Cough
- Dyspnea
- Erythema
- Fever
- Flushing
- Tachycardia
- Urticaria
Precautions
No enhanced messaging available.
Eteplirsen is labeled for use in pediatric patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping.
In juvenile animal studies, renal tubular necrosis associated with clinical pathology changes (e.g., increased serum urea nitrogen and creatinine concentrations, decreased urine creatinine clearance) and changes in bone densitometry parameters (i.e., decreased mineral density, mineral content, and area) were observed following administration of IV eteplirsen. Eteplirsen exposure at the lowest dose tested in animals was similar to exposure at the recommended dose in humans. No effects on the male reproductive system, neurobehavioral development, or immune function were observed.
Eteplirsen has not been studied in female patients. It is not known whether the drug is associated with risk if used in pregnant women.
Eteplirsen has not been studied in female patients. It is not known whether eteplirsen is distributed into human 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 eteplirsen and any potential adverse effects of the drug or disease on the infant.
There is no experience with eteplirsen 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 51 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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