Eplontersen

Generic name: Eplontersen
Brand name: Wainua
Dosage form: subcutaneous injection (45 mg/0.8 mL)
Drug class: Miscellaneous metabolic agents

Medically reviewed by Melisa Puckey, BPharm. Last updated on Dec 22, 2023.

What is eplontersen?

Eplontersen (Wainua) is used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN), which is a rare, progressive disease that can be fatal if not treated. Amyloidosis is a disease caused by a buildup of abnormal proteins called amyloid. 

Eplontersen is from a class of medications called LICA (ligand-conjugated antisense oligonucleotide). Eplontersen works by decreasing the amount of TTR protein that is made, which slows disease progression and improves neuropathy and the patient's quality of life.

Eplontersen is given as a monthly self-administered subcutaneous injection.

Eplontersen received FDA approval on December 21, 2023, after positive results from the phase III clinical trial NEURO-TTRansform (NCT04136184).

What is ATTR amyloidosis?

ATTR amyloidosis is caused by a protein in the body called transthyretin (TTR) that changes from its normal shape and then forms fibrous clumps. These clumps of misshapen protein are then deposited into various organs and nerves, which can stop the organs and nerves from working properly.

ATTR amyloidosis polyneuropathy (ATTR-PN) is the build-up of amyloid protein in nerves and can cause symptoms such as a loss of sensation, tingling, numbness, or pain in the hands and feet.

ATTR cardiomyopathy (ATTR-CM) is when the clumps of amyloid build up in the heart tissue, which affects the heart’s ability to work properly, leading to symptoms similar to heart failure and enlarged heart.

Eplontersen side effects

Common eplontersen side effects were low vitamin A serum levels and vomiting, which occurred in at least 9% of patients. Low vitamin A level is a serious, but common side effect of treatment with this medicine. Your healthcare provider should tell you to take vitamin A supplements while using eplontersen. Do not take more than the amount of vitamin A your healthcare provider has recommended. Call your healthcare provider if you develop eye problems such as difficulty seeing at night, or in low-lit areas (night blindness), or dry eyes. If you develop eye problems while receiving treatment with this medicine, your healthcare provider should send you to see an eye doctor.

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 the FDA at 1-800-FDA-1088.

Before taking this medicine

Pregnancy

Tell your healthcare provider if you are pregnant, plan to become pregnant or think you may be pregnant, as it is not known if eplontersen can harm your unborn baby. Changes in vitamin A levels and vitamin A supplementation related to use of eplontersen may harm your unborn baby. 

Breastfeeding

Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if eplontersen can pass into your breast milk or harm your baby. Talk with your healthcare provider about the best way to feed your baby while you are using this medicine. 

How should I use eplontersen?

Read the detailed Instructions for Use that come with your eplontersen single-dose autoinjector. Your healthcare provider will show you or your caregiver how to inject eplontersen.

If you or your caregiver have any questions, ask your healthcare provider.

Instructions for use

Remove the single-dose autoinjector from the refrigerator 30 minutes prior to the injection and allow it to warm to room temperature. Do not use other warming methods. 

Inspect the autoinjector visually for particulate matter and discoloration prior to administration. The solution should appear colorless to yellow. Do not use if cloudiness, particulate matter, or discoloration is observed prior to administration. 

Eplontersen is injected under your skin (subcutaneously) in your stomach area (abdomen), or the front of your upper legs (thighs) by you or a caregiver. A caregiver may also give you an injection in the outer area of your upper arm. 

Eplontersen should be injected 1 time on the same day of each month

Follow your healthcare provider’s instructions on when to inject eplontersen.

For more detailed instructions with diagrams, click here Wainua Patient information

If you miss a dose, take the missed dose as soon as possible. Then inject WAINUA 1 month from the date of your last dose to get back on a monthly dosing schedule. If you have questions about your schedule, ask your healthcare provider. 

Dosing information

Recommended eplontersen dose: 45 mg administered by subcutaneous injection once a month.

Eplontersen is available as a 45 mg/0.8 mL single-dose autoinjector.

How effective is eplontersen?

In the global, open-label, randomized Phase 3 NEURO-TTRansform trial for eplontersen in patients with ATTR amyloidosis polyneuropathy (ATTRv-PN), there was an 81.2% reduction in serum transthyretin (TTR) concentration when compared to the baseline, showing that eplontersen reduced TTR protein production. 

The study showed that eplontersen had a statistically significant effect on the modified Neuropathy Impairment Score +7 (mNIS+7), which is a measure of neuropathic disease progression when compared to the placebo group.

Treatment also significantly improved patient-reported quality of life compared to the external placebo group (p<0.0001) as measured by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).

Eplontersen is also being studied in the treatment of ATTR cardiomyopathy (ATTR-CM).

Interactions

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. 

Tell your healthcare provider if you take: 

• Vitamin A or beta-carotene supplements.

Ask your healthcare provider or pharmacist if you are not sure if you take any of these medicines. Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine. Not all possible interactions are listed here.

Ingredients

Active ingredient: eplontersen sodium. 

Wainua Inactive ingredients: dibasic sodium phosphate, anhydrous; monobasic sodium phosphate, dihydrate; sodium chloride; water for injection, and may include hydrochloric acid and sodium hydroxide for pH adjustment. 

Storage

• Store in the refrigerator between 36°F to 46°F (2°C to 8°C) in the original carton. 
• Wainua can also be kept at room temperature that is no higher than 86°F (30°C) in the original carton for up to 6 weeks. 
• Do not let Wainua reach temperatures above 86°F (30°C).
• If you do not use an autoinjector kept at room temperature within 6 weeks, throw it away.
• Do not freeze.
• Do not expose WAINUA to heat or light.

Company

Distributed by: AstraZeneca Pharmaceuticals LP, Wilmington, DE 19850 AstraZeneca.

Ionis Pharmaceuticals, Inc.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

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