Elamipretide

Elamipretide is used to help improve muscle strength in adults and children who weigh at least 30 kg and have Barth syndrome.

Elamipretide may also be used for other reasons that are not listed in this medication guide.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Elamipretide is injected under the skin in the stomach or thigh, usually once a day at the same time each day. A healthcare provider may give you this injection or may teach you or your caregiver how to properly use this medicine by yourself.

Read and carefully follow any Instructions for Use provided with your medicine. Ask your doctor or pharmacist if you don't understand how to use an injection.

Prepare an injection only when you are ready to give it. Call your pharmacist if the medicine looks cloudy, has changed colors or has particles in it.

Do not inject within 2 inches of your navel (belly button), or areas where the skin is hard, tender, red, bruised, or has scars or stretch marks. Do not inject into the same place two times in a row.

Do not reuse a needle or syringe. Place them in a puncture-proof "sharps" container and dispose of it following state or local laws. Keep out of the reach of children and pets.

You may be given other medications to treat certain allergic reactions or serious side effects. Keep taking these medicines for as long as your doctor has prescribed.

Store an unopened vial (bottle) in the refrigerator. Do not freeze. After the first dose, you may store the opened vial in the refrigerator or at room temperature.

If the vial is stored in the refrigerator, let it reach room temperature for 10 minutes before using.

Throw away the vial 8 days after you first opened it, even if there is still medicine left inside.

Use only as directed. Inform your doctor if you are taking other medicines or have other medical conditions or allergies.

You should not use elamipretide if you are allergic to it or any of its ingredients.

Tell your doctor if you have or have ever had:

• kidney disease.

Elamipretide may contain an ingredient that can cause serious side effects or death in very young or premature babies. Do not give elamipretide to a child without medical advice.

It is not known if elamipretide will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

Ask a doctor if it is safe to breastfeed while using this medicine.

Get emergency medical help if you notice signs of an allergic reaction: hives, trouble breathing, swelling of your face, lips, tongue, or throat.

Less serious side effects may happen more often, or you may not have any at all.

Common side effects of elamipretide may include:

• pain, bruising, swelling, or irritation at the injection site.

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

Usual Adult Dose for Barth Syndrome:

Patients weighing at least 30 kg:40 mg subcutaneously once a dayComments:-This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Use: To improve muscle strength in adult patients with Barth syndrome weighing at least 30 kg

Usual Pediatric Dose for Barth Syndrome:

Patients weighing at least 30 kg:40 mg subcutaneously once a day Comments:-This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. -This drug should not be used in neonates. Use: To improve muscle strength in pediatric patients with Barth syndrome weighing at least 30 kg

Skip the missed dose and take your next dose at the usual time. Do not take two doses at once.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Follow your doctor's instructions about any restrictions on food, drinks, or activities.

Other drugs may affect elamipretide, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.