Wainua (eplontersen) is approved for adults to treat polyneuropathy or nerve damage from a rare genetic problem called hereditary transthyretin-mediated amyloidosis (hATTR). This medication is an antisense therapeutic that's given as an injection under the skin once per month. It's convenient because it comes as an autoinjector that can be used at home. Potential side effects include lower vitamin A levels and vomiting.
Polyneuropathy from hereditary transthyretin-mediated amyloidosis (hATTR)
The transthyretin (TTR) gene tells the body to make the TTR protein to help carry vitamin A and thyroid hormone around.
People with hereditary transthyretin-mediated amyloidosis (hATTR) have a mutated version of the TTR gene that tells the body to make abnormal TTR protein. These abnormal TTR proteins clump together in clusters called amyloid fibrils, which travel throughout your body and cause damage to your organs and nerves.
Wainua (eplontersen) is an antisense therapeutic. It works by breaking down mutated versions of the TTR gene in the body. This lessens the amount of abnormal TTR proteins made, which lessens the amount of amyloid fibrils that can cause damage to the body.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Works well to lower TTR levels and improve nerve pain from hATTR
Injected once per month
Can inject at home (after training from a healthcare professional)
Needs to be given as an injection under the skin
Might be difficult for some people to inject properly
Doesn't cure hATTR
Inject Wainua (eplontersen) under the skin once every month. To help you remember when to inject your medication, you can write your injection dates on your calendar or use a reminder app on your phone.
Ask your prescriber if you should take a vitamin A supplement since Wainua (eplontersen) can lower your vitamin A levels. You can also eat foods high in vitamin A to help maintain your vitamin levels. Check with your prescriber about how much vitamin A you need per day, especially if you're already taking supplements that contain vitamin A or beta-carotene.
Keep the Wainua (eplontersen) autoinjectors in the refrigerator and in the original box to protect it from light (don't freeze). You can store the syringes in the original box at room temperature for up to 6 weeks, but don't let them reach temperatures above 86 degrees Fahrenheit. After 6 weeks at room temperature, throw the autoinjectors away into a sharps container.
Tell your prescriber if you're pregnant, planning to become pregnant, or breastfeeding. Wainua (eplontersen) can potentially harm babies if taken during pregnancy or while breastfeeding.
How to use Wainua (eplontersen) at home:
Make sure a healthcare professional teaches you how to give yourself Wainua (eplontersen) injections properly and safely. Read the instructions on how to inject the medication under your skin. Ask your prescriber or pharmacist if you have any questions about how to use Wainua (eplontersen).
Before each injection, take the Wainua (eplontersen) box out of the refrigerator and place it on a flat surface so it can warm up to room temperature. This will take about 30 minutes. Don't try to warm it up any other way, like with a microwave or hot water, because it can damage your medication.
Always check that the medication is clear and colorless to yellow before you inject Wainua (eplontersen). Don't use the autoinjector if you see that the medication is cloudy, discolored, or has particles in it. Air bubbles are normal.
Wash your hands with soap and water and clean the area you'll inject into with an alcohol swab before injecting Wainua (eplontersen). This will help prevent infection.
Where to inject: Inject Wainua (eplontersen) under the skin into the stomach (at least 2 inches away from the belly button) or the front of the thigh. If someone is helping you to inject the medication, you can also choose to inject it under the skin on back of the upper arm. Don't inject into skin that's damaged, scarred, bruised, tender, red, or hard to prevent more skin irritation.
How to inject: Place the orange needle shield at a 90-degree angle against your skin with the viewing window facing you. Then, push down firmly and hold the autoinjector in place against your skin for 10 seconds to make sure you get the full dose. You'll hear a click when the injection starts. You might hear a second click as the injection continues, but it doesn't mean that the injection is done. The injection is complete when you see the entire viewing window on the autoinjector is orange.
After injecting Wainua (eplontersen), throw away the autoinjector in a sharps container or something made of heavy-duty plastic, such as an empty milk carton or laundry detergent bottle. This lowers the risk of needle-stick injuries. Don't place it into the household trash.
Each Wainua (eplontersen) autoinjector is for a single use only, so you must throw it away after each use. Don't reuse autoinjectors.
Wainua (eplontersen) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Wainua (eplontersen) can lower the vitamin A levels in your blood. Vitamin A is important for your eye health, so having vision problems, like trouble seeing at night or in dim light and dry eye, can be an early sign of very low vitamin A. Let your prescriber know if you notice these eye problems while you're taking Wainua (eplontersen). You'll probably need to get your eyes checked.
During treatment, you might need to take a vitamin A supplement to prevent your levels from dropping too much. It also might be helpful to eat foods that are high in vitamin A. Ask your prescriber about how much vitamin A you need per day. It depends on how old you are, whether you're male or female, and whether you're pregnant or nursing. Don't take more than the maximum recommended daily dose of vitamin A for you to try and raise your vitamin A levels. This can lead to side effects like nausea, dizziness, or headache.
The typical dose is 45 mg injected under the skin once per month. It's injected into the stomach or upper thigh. The medication can also be injected into the back of the upper arm if someone else is helping you.
Polyneuropathy from hereditary transthyretin-mediated amyloidosis (hATTR)
Polyneuropathy from hereditary transthyretin-mediated amyloidosis (hATTR)
Polyneuropathy from hereditary transthyretin-mediated amyloidosis (hATTR)
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Amyloidosis Research Consortium. (n.d.). Hereditary ATTR amyloidosis.
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AstraZeneca Pharmaceuticals LP. (2024). Wainua- eplontersen injection, solution [package insert]. DailyMed.
Coelho, T., et al. (2023). Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy. Journal of the American Medical Association.
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