Exondys 51

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

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.

Eteplirsen has the following uses:

Eteplirsen is an antisense oligonucleotide 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 eteplirsen. A clinical benefit of eteplirsen has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.1

AHFS First Release. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.

In the U.S.

• Exondys 51

Available Dosage Forms:

• Solution

Therapeutic Class: Musculoskeletal Agent

• It is used to treat Duchenne muscular dystrophy (DMD).

Dosing Information

The recommended dose of EXONDYS 51 is 30 milligrams per kilogram administered once weekly as a 35 to 60 minute intravenous infusion.

If a dose of EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time.

Preparation Instructions

EXONDYS 51 is supplied in single-dose vials as a preservative-free concentrated solution that requires dilution prior to administration. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Use aseptic technique.

1. Calculate the total dose of EXONDYS 51 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of EXONDYS 51 needed and the correct number of vials to supply the full calculated dose.
2. Allow vials to warm to room temperature. Mix the contents of each vial by gently inverting 2 or 3 times. Do not shake.
3. Visually inspect each vial of EXONDYS 51. EXONDYS 51 is a clear, colorless solution that may have some opalescence. Do not use if the solution in the vials is discolored or particulate matter is present.
4. With a syringe fitted with a 21-gauge or smaller non-coring needle, withdraw the calculated volume of EXONDYS 51 from the appropriate number of vials.
5. Dilute the withdrawn EXONDYS 51 in 0.9% Sodium Chloride Injection, USP, to make a total volume of 100-150 mL. Visually inspect the diluted solution for particulates.
6. EXONDYS 51 contains no preservatives and should be administered immediately after dilution. Complete infusion of diluted EXONDYS 51 solution within 4 hours of dilution. If immediate use is not possible, the diluted solution may be stored for up to 24 hours at 2ºC to 8ºC (36ºF to 46ºF). Do not freeze. Discard unused EXONDYS 51.

Administration Instructions

Application of a topical anesthetic cream to the infusion site prior to administration of EXONDYS 51 may be considered.

EXONDYS 51 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion.

Infuse the diluted EXONDYS 51 solution over 35 to 60 minutes. Do not mix other medications with EXONDYS 51 or infuse other medications concomitantly via the same intravenous access line.

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.

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

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.

Get emergency medical help if your child has signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Common side effects may include:

• pain, bruising, itching, skin rash, or other irritation where the medicine was injected;
• vomiting;
• joint pain;
• problems with balance; or
• cold symptoms such as stuffy nose, sneezing, sore throat.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Other drugs may interact with eteplirsen, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell each of your child's healthcare providers about all medicines the child uses now, and any medicine the child starts or stops using.