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.
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.
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Contact your healthcare provider immediately if you experience any of the following.
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):
How to use Wainua (eplontersen) at home:
In a clinical study looking at how well the medication works, most people who took Wainua (eplontersen) for 2 years had a lower TTR levels in the body than those who took placebo (no medication). People who took the medication also reported having less neuropathy (nerve pain) and better quality of life after 2 years of treatment than people who didn't take medication. Follow up with your prescriber about your treatment progress with Wainua (eplontersen) and ask them if you have any concerns.
Hair loss wasn't a side effect that people who took Wainua (eplontersen) in clinical studies reported having. Contact your healthcare team if you notice hair loss while you're taking this medication so they can help you figure out possible causes.
Weight gain wasn't reported by people who took Wainua (eplontersen) during clinical studies. Talk to your care team if you notice concerning changes in your body weight after you start taking this medication.
It takes almost 4 months for most of Wainua (eplontersen) to leave your body after a single dose. This time frame is estimated based on the half-life of Wainua (eplontersen).
You'll probably need to take Wainua (eplontersen) for the rest of your life, as long as you're not having side effects from the medication and as long as it's helping your condition. Wainua (eplontersen) doesn't cure hATTR. But it helps manage nerve pain from the condition. Speak with your prescriber for more information about managing and treating hATTR.
Yes, you can give Wainua (eplontersen) injections to yourself at home after you get trained by a healthcare professional. You might get the first few injections at your prescriber's office or clinic to see how you react to the medication. Then, your prescriber will discuss with you about making arrangements for you to inject Wainua (eplontersen) at home if you're comfortable and if it's appropriate for you. Discuss with your prescriber about whether injecting Wainua (eplontersen) at home is an option for you.
All three medications are injections that can help treat nerve damage from hereditary transthyretin-mediated amyloidosis (hATTR). How each medication is given and how often they're needed are different. Wainua (eplontersen) is given as an injection under the skin once per month. It's autoinjector that you can use at home. Tegsedi (inotersen) is given as an injection under the skin once per week. You can also inject this medication at home but with a prefilled syringe. And Onpattro (patisiran) is an infusion that's given into the vein once every 3 weeks. It's given by a healthcare professional at a medical office or clinic. In addition, all three medications can possibly lower vitamin A levels. But unlike Wainua (eplontersen), Tegsedi (inotersen) has more serious risks, like bleeding and kidney problems, and Onpattro (patisiran) can cause infusion-related reactions. Speak with your prescriber about which option best fits your needs.
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.