Eteplirsen

Eteplirsen works by helping the body produce a protein called dystrophin. Dystrophin is a protein that helps stabilize the structure of muscle tissue and is necessary for proper muscle development and function. Without dystrophin, muscle fibers can become weak and easily damaged. This can affect voluntary movement such as walking.

Children with Duchenne muscular dystrophy have a mutation of a gene that would normally cause the body to produce dystrophin.

Eteplirsen is used in children with Duchenne muscular dystrophy (DMD). Eteplirsen is not a cure for DMD, but this medicine may lessen muscle weakness and muscle wasting caused by this disease.

Eteplirsen was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some children responded to this medicine. However, further studies are needed to determine if eteplirsen is generally effective in treating DMD.

Eteplirsen may also be used for purposes not listed in this medication guide.

Tell your child's doctor if any prior eteplirsen injections caused a severe allergic reaction in your child.

Before your child receives eteplirsen, tell the doctor about all your child's medical conditions or allergies, and all the medicines the child uses.

Although eteplirsen has been used mainly in children, it is not known whether this medicine could be harmful if used during pregnancy or while nursing.

Eteplirsen is injected into a vein through an IV. A healthcare provider will give your child this injection.

To make the injection more comfortable, a numbing medicine may be applied to the skin area where the IV needle will be placed.

Eteplirsen is given once per week. This medicine must be injected slowly, and the infusion can take up to 1 hour to complete.

Tell your doctor if your child has any changes in weight. Eteplirsen doses are based on weight, and any changes may affect the dose.

Call your child's doctor for instructions if you miss an appointment for your child's eteplirsen injection.

Specific Drugs

It is essential that the manufacturer’s labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:

Please see product labeling for drug interaction information.

There are no dosage adjustments provided in the manufacturer’s labeling (has not been studied).

Incidences include reactions experienced with approved and unapproved doses.

>10%:

Central nervous system: Equilibrium disturbance (38%)

Dermatologic: Contact dermatitis (25%), excoriation (≥10%), skin rash (≥10%)

Gastrointestinal: Vomiting (38%)

Hematologic & oncologic: Bruise (≥10%)

Local: Catheter pain (≥10%)

Neuromuscular & skeletal: Arthralgia (≥10%)

Respiratory: Upper respiratory tract infection (≥10%)

<1% (Limited to important or life-threatening): Facial flushing (transient; on infusion day), flushing (on infusion day), increased body temperature (transient; on infusion day)

There are no warnings listed in the manufacturer’s labeling.

30 mg/kg IV over 35 to 60 minutes once a week

Comments:
-Administer missed doses as soon as possible after the scheduled time.

Use: For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

Data not available

Administration advice:
-A topical anesthetic may be applied to the infusion site prior to administration.
-Flush the IV line with 0.9% sodium chloride prior to and after the infusion.
-Infuse the solution over 35 to 60 minutes.

Storage requirements:
-The manufacturer product information should be consulted.

Reconstitution/preparation techniques:
-The manufacturer product information should be consulted.

IV compatibility:
-Other medications should not be mixed or infused concomitantly with this drug via the same IV line.

General:
-This drug is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with this drug.
-Clinical benefit of this drug has not been established.
-Continued approval for DMD may be contingent upon verification of clinical benefit in confirmatory trials.

The manufacturer makes no recommendation regarding use during lactation. Excreted into human milk: Unknown Excreted into animal milk: Unknown Comments: -The effects in the nursing infant are unknown.