Exondys 51 (eteplirsen) dosing, indications, interactions, adverse effects, and more

Sections

Sections eteplirsen
Dosing & Uses
Adverse Effects
Warnings
Pregnancy
Pharmacology
Administration
Images
Patient Handout
Formulary

Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

IV solution

50mg/mL (as 100mg/2mL or 500mg/10mL single-dose vials)

Duchenne Muscular Dystrophy

Indicated for Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

30 mg/kg IV once weekly

Also see Administration

Dosing Considerations

Approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients

Clinical benefit has not been established

Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials

Dosage Forms & Strengths

IV solution

50mg/mL (as 100mg/2mL or 500mg/10mL single-dose vials)

Duchenne Muscular Dystrophy

Indicated for Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

30 mg/kg IV once weekly

Also see Administration

Dosing Considerations

Approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients

Clinical benefit has not been established

Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials

>10%

Balance disorder (38%)

Vomiting (38%)

Contact dermatitis (25%)

Confusion (>10%)

Frequency Not Defined

Transient erythema

Facial flushing

Elevated temperature on days of infusion

Contraindications

None

Cautions

Approved under accelerated approval; clinical benefit has not been established

Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria reported; if a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting therapy

Pregnancy

There are no human data available to assess the use during pregnancy

Lactation

Unknown if distributed in human breast milk

Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for the drug, and any potential adverse effects on the breastfed infant from the drug or from the underlying maternal condition

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.

Mechanism of Action

Morpholino antisense oligonucleotide designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping

Exon skipping is intended to allow for production of an internally truncated dystrophin protein

The underlying cause of Duchenne muscular dystrophy (DMD) is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function

Absorption

Peak plasma time: 1.1-1.2 hr (near end of infusion across dose range of 0.5-50 mg/kg/wk)

Distribution

Protein bound: 6-17% (in vitro data)

Vd: 600 mL/kg

Metabolism

Does not appear to be metabolized by hepatic microsomes of any species tested, including humans

Elimination

Half-life: 3-4 hr

Total clearance: 339 mL/hr/kg (at 12 week of therapy with 30 mg/kg/wk)

Excretion: ~66% urine within 24 hr of IV administration

Pharmacogenomics

Indicated processing in patients with genetic mutations that are amenable to exon 51 skipping

IV Preparation

Allow vials to warm to room temperature

Mix vial contents by gently inverting 2-3 times; DO NOT SHAKE

Inspected visually for particulate matter and discoloration prior to administration; should appear as a clear, colorless solution that may have some opalescence

Do not use if solution is discolored or particulate matter is present

With a syringe fitted with a ≤21-ga noncoring needle, withdraw the calculated volume from the appropriate number of vials

Dilute withdrawn dose in 0.9% NaCl to a total volume of 100-150 mL; again, visually inspect for particulates

IV Administration

Consider applying a topical anesthetic cream to infusion site prior to administration

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

Infuse IV over 35-60 min

Do not mix with other medications or infuse other medications concomitantly via the same IV access

Storage

Unopened vials

Refrigerate at 2-8°C (36-46°F)
Do not freeze
Protect from light and store in original carton until ready for use

Diluted drug

Contains no preservatives; should be administered immediately after dilution
Complete infusion of diluted solution within 4 hr of dilution
If unable to administer immediately, diluted solution may be refrigerated for up to 24 hr at 2-8°C (36-46°F)
Do not freeze
Discard unused solution

Images

No images available for this drug.

Patient Handout

A Patient Handout is not currently available for this monograph.

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.

Create Your List of Plans

Adding plans allows you to:

View the formulary and any restrictions for each plan.
Manage and view all your plans together – even plans in different states.
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.

View explanations for tiers and restrictions

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.

Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.

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