Generic name: eteplirsen
Brand name: Exondys 51
Dosage form: intravenous solution (50 mg/mL)
Drug class: Miscellaneous uncategorized agents
Medically reviewed by A Ras MD.
What is eteplirsen used for?
Eteplirsen is a prescription medicine that is used to treat Duchenne muscular dystrophy (DMD).
EXONDYS 51 (eteplirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. EXONDYS 51 is clear and colorless, and may have some opalescence, and may contain trace amounts of small, white to off-white amorphous particles. EXONDYS 51 is supplied in single dose vials containing 100 mg or 500 mg eteplirsen (50 mg/mL). EXONDYS 51 is formulated as an isotonic, phosphate buffered saline solution with an osmolality of 260 to 320 mOsm and a pH of 7.5. Each milliliter of EXONDYS 51 contains 50 mg eteplirsen; 0.2 mg potassium chloride, 0.2 mg potassium phosphate monobasic, 8 mg sodium chloride, and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. The product may contain hydrochloric acid or sodium hydroxide to adjust pH.
Eteplirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMOs are synthetic molecules in which the five-membered ribofuranosyl rings found in natural DNA and RNA are replaced by a six-membered morpholino ring. Each morpholino ring is linked through an uncharged phosphorodiamidate moiety rather than the negatively charged phosphate linkage that is present in natural DNA and RNA. Each phosphorodiamidate morpholino subunit contains one of the heterocyclic bases found in DNA (adenine, cytosine, guanine, or thymine). Eteplirsen contains 30 linked subunits. The molecular formula of eteplirsen is C364H569N177O122P30 and the molecular weight is 10305.7 daltons.
The structure and base sequence of eteplirsen are:
Mechanism of Action
Eteplirsen is 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, which was evaluated in Study 2 and Study 3
Before taking eteplirsen, tell your doctor:
- If you are allergic to eteplirsen; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.
This medicine may interact with other drugs or health problems.
Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take eteplirsen with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.
What are some things I need to know or do while I take eteplirsen?
- Tell all of your health care providers that you take eteplirsen. This includes your doctors, nurses, pharmacists, and dentists.
- Tell your doctor if you are pregnant or plan on getting pregnant. You will need to talk about the benefits and risks of using eteplirsen while you are pregnant.
- Tell your doctor if you are breast-feeding. You will need to talk about any risks to your baby.
How is eteplirsen best taken?
Use eteplirsen as ordered by your doctor. Read all information given to you. Follow all instructions closely.
- It is given as an infusion into a vein over a period of time.
What do I do if I miss a dose?
- Call your doctor to find out what to do.
What are the side effects of eteplirsen that I need to call my doctor about immediately?
WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:
- Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
- Shortness of breath.
- Very bad dizziness or passing out.
- Change in balance.
What are some other side effects of eteplirsen?
All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:
- Throwing up.
- Joint pain.
- Pain and irritation where eteplirsen goes into the body.
- Signs of a common cold.
These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.
You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.
If overdose is suspected:
If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.
How do I store and/or throw out eteplirsen?
- If you need to store eteplirsen at home, talk with your doctor, nurse, or pharmacist about how to store it.