Eplontersen injection

WHY is this medicine prescribed?

Eplontersen is used for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (an inherited condition in which abnormal proteins build up and deposit in tissues, resulting in nerve damage) in adults. Eplontersen is in a class of medications called antisense oligonucleotides. It works by decreasing the amount of abnormal proteins and the amount of abnormal protein deposited in the body's tissues, which decreases nerve damage.

Are there OTHER USES for this medicine?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

HOW should this medicine be used?

Eplontersen comes as a solution (liquid) in a prefilled autoinjector to be given subcutaneously (under the skin). It is usually given once a month by the patient or caregiver at home.

Ask your doctor or pharmacist to show you or the person who will be injecting the medication how to inject and prepare it.

Remove the prefilled autoinjector from the refrigerator 30 minutes before planned administration time and allow to warm to room temperature in its original carton. Do not try to warm the medication by heating it in a microwave, placing it in hot water, leaving it in sunlight, or through any other method.

Always look at eplontersen solution before injecting it. Check that the expiration date has not passed and that the liquid is clear or slightly yellow. The liquid should not contain visible particles. Do not use a syringe or autoinjector if it is cracked or broken, if it is expired or frozen, or if the liquid is cloudy or contains small particles.

You can inject eplontersen injection anywhere on the front of your thighs (upper leg) or abdomen (stomach) except your navel and the area 2 inches (5 centimeters) around it. If you have a caregiver to inject the medication, the back of the upper arm may also be used. To reduce the chances of soreness or redness, use a different site for each injection. Do not inject into an area where the skin is tender, bruised, red, or hard or where you have scars or stretch marks.

Wash hands thoroughly and clean injection site with alcohol swab. Remove the clear cap by pulling it off while holding the autoinjector. Place the orange top of the autoinjector at a 90 degree angle to the skin with the viewing window visible to you. Push firmly and hold the autoinjector directly against the skin. You will hear a click when the injection starts. Continue to hold the autoinjector still against the skin for 10 seconds until the entire dose has been dispensed. Check to make sure the orange plunger is completely filling the viewing window to assure that the full dose has been given. Remove the autoinjector by lifting straight up and the orange needle shield will lock back into place and cover the needle.

Place used autoinjector in a sharps container or household container that is made of heavy duty plastic such as a milk jug or a laundry detergent bottle. When your sharps container is mostly full, you will need to consult with your community guidelines on how to dispose of it. Do not place in your regular trash.

What SPECIAL PRECAUTIONS should I follow?

Before using eplontersen,

• tell your doctor and pharmacist if you are allergic to eplontersen, any other medications, or any of the ingredients in eplontersen injection. Ask your pharmacist or check the Medication Guide for a list of the ingredients.

• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take.

• tell your doctor if you have or have ever had kidney or liver disease or vitamin A deficiency.

• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while receiving eplontersen, call your doctor.

What SPECIAL DIETARY instructions should I follow?

Unless your doctor tells you otherwise, continue your normal diet.

What should I do IF I FORGET to take a dose?

If you forget to inject a dose, inject as soon as you remember it and then inject the next dose 1 month after the last injection to establish a new monthly dosing schedule.

What SIDE EFFECTS can this medicine cause?

Eplontersen may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• bruising, pain or redness at the site of injection
• vomiting
• blurred vision

Some side effects can be serious. If you experience any of these symptoms, call your doctor immediately:

• difficulty seeing at night or in low lit areas, dry eyes

Eplontersen may cause low vitamin A levels which can be serious. Vitamin A deficiency can cause damage to the eyes. Your doctor will ask you to take a vitamin A supplement during treatment. It is important to continue taking this as long as your doctor recommends it and no to take more than is recommended. Talk to your doctor about the risks of taking this eplontersen.

Eplontersen may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What should I know about STORAGE and DISPOSAL of this medication?

Store eplontersen autoinjectors in their original carton in the refrigerator until ready for use. You may store them at room temperature in the original container for up to 6 weeks. If storing at room temperature and the autoinjector is not used within 6 weeks, do not use and instead, discard eplontersen autoinjector. Store your medication only as directed. Make sure you understand how to store your medication properly.

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. http://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

What should I do in case of OVERDOSE?

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

What OTHER INFORMATION should I know?

Keep all appointments with your doctor and the laboratory. Your doctor may order certain lab tests to check your body's response to eplontersen.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.