Wainua (eplontersen) is a prescription injection medicine that slows down nerve damage in people with a type of amyloidosis called hereditary transthyretin-mediated amyloidosis (hATTR). The FDA approved Wainua in 2023 for adults with this condition. It is injected under the skin once a month with a ready-to-use auto-injector that can be used at home by the person or their caregiver.
How Does Wainua Work in hATTR?
hATTR happens when changes in the transthyretin (TTR) gene cause clumps of protein called amyloids to form in your body’s organs. When amyloids build up in your nerves, your nerves can become damaged, affecting how they work.
Wainua works by destroying the altered TTR genes. This may result in lowering the amount of abnormal TTR made in your liver, reduce amyloid buildup in your nerves, and help prevent your nerve damage from getting worse.
Is Wainua Right for Me?
Wainua may be the right choice for you if you:
- Are an adult with hATTR
- Have nerve damage caused by hATTR
Your health care provider will review your family history for hATTR, examine your symptoms, order blood tests to confirm the TTR gene change, and run imaging tests (like magnetic resonance imaging, or MRI) to see amyloid buildups in your nerves and other organs.
How Was Wainua Studied for Treating hATTR?
One clinical study, the NEURO-TTRansform trial, was done to look at the benefits and risks of Wainua in people with hATTR.
The main questions this study wanted to answer were: How does Wainua change the amount of TTR in the blood? And how does it affect the symptoms of nerve damage in people with hATTR?
To learn this, the researchers:
- Measured the TTR in the blood
- Used a special rating system, called the neuropathy impairment score (NIS), which is a test that checks for changes in muscle strength, reflexes, and sensations. Lower scores mean better nerve function.
- Measured quality of life, using a test called the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN). This test checks how tired people feel physically and mentally, how well they can do activities, how their daily tasks are affected, and how their mood changes. Lower scores mean a better quality of life.
They compared the results to the results from a group of people with hATTR in another study who received a placebo, a dummy medicine that has no effect. Both the people in the study and the health care providers knew who received Wainua.
What medicines did the people in the study take?
People in the study were randomly selected to get either Wainua or another medicine called inotersen. Inotersen is also a medicine for nerve damage in people with hATTR and was compared to a placebo in an earlier study with a similar population. The researchers wanted to use the placebo information from that study, so they gave inotersen to a group of people in the Wainua study to make sure that people in both studies responded in a similar way.
Who was included in the study?
People in the study met the following requirements:
- Ages 18 to 82 years old
- Have stage 1 (can walk without help) or stage 2 (can walk with help) nerve damage caused by hATTR
- Changes in the TTR gene
- Symptoms of nerve damage from hATTR, with an NIS rating between 10 and 130
Who was excluded from the study?
People could not be in this study if they:
- Had other types of amyloidosis
- Had other causes of nerve damage, like uncontrolled diabetes or autoimmune conditions
- Had a liver transplant or expected to need one within a year after the study
- Previously took or were currently on certain medicines for hATTR, including inotersen
- Had major surgery within three months before the study
- Had medical conditions or test results that made it unsafe for them to join the study
What was the study population?
- A total of 144 people in the study got Wainua.
- The average age of these people was 52 years.
- About 31% of them were female and 69% were male.
- About 78% were White,15% were Asian, 4% were Black, and about 3% were from other races or ethnicities.
- All had changes in their TTR gene.
- About 80% could walk without help (stage 1 nerve damage) and 20% could walk with help (stage 2 nerve damage).
A total of 24 people got inotersen. Researchers compared the results from people who got Wainua in this study to the results from 60 people who got a placebo in the earlier study.
How long was the study?
People got Wainua every four weeks for about 1.5 years (up to 85 weeks).
What Were the Main Benefits of Wainua Seen in the Study?
After 66 weeks of getting Wainua, people in the study had a significantly lower level of TTR in their blood and fewer symptoms of nerve damage, compared with those who got a placebo. They also had a better quality of life, compared to those who got a placebo.
| Wainua | Placebo | |
| TTR levels dropped by | 80% | 11% |
| NIS rating changed by | Less than 1 (0.3) points | 25 points |
| QOL-DN changed by | -5 points | 14 points |
What Are the Possible Side Effects of Wainua, and How Can I Prevent or Manage Them?
The most common side effects of Wainua include:
Low blood vitamin A levels
- Take vitamin A supplements, following your health care provider’s instructions. Do not take more than the amount of vitamin A they have recommended.
- Contact your health care provider if you notice anything unusual with your eyesight, like difficulty seeing in low light or dry eyes. They may refer you to an eye specialist.
Vomiting
- Drink plenty of fluids to keep hydrated.
- Avoid eating heavy or greasy foods.
- Eat and drink slowly.
- Consult with your pharmacist. They may recommend nonprescription medicines to help with your vomiting.
- Contact your health care provider if vomiting does not stop within a few days.
These are not all the possible side effects of Wainua. Contact your health care provider for medical advice about side effects if you are having symptoms that bother you. You can report side effects to the FDA at 800-FDA-1088 (800-332-1088) or www.fda.gov/medwatch. You can also report side effects to the drugmaker, AstraZeneca, at 800-236-9933.
How Do I Know if Wainua Is Working?
Your health care provider will regularly test your blood to measure the levels of TTR and see how much your nerve damage symptoms have improved. You may also notice how Wainua has affected your muscle strength, reflexes, and feelings in your body, as well as your quality of life, including changes in how tired you feel, how well you can do your daily activities, and how your mood has changed.
Do not stop your Wainua shots or change your injection schedule unless your health care provider tells you to do so.
How Do Other Medicines or Foods Interact With Wainua?
No interactions between Wainua and other medicines or foods have been found in clinical studies or lab research so far. But more information may become available in the future.
Tell your pharmacist and other health care providers about all the other prescription and nonprescription medicines, vitamins, supplements, and herbal products you take before starting Wainua and while you are getting it.
How Can I Get Wainua?
You will need a prescription from a health care provider. Wainua is only available at specialty pharmacies. They will mail your auto-injector and sharps container to you.
If you need help paying for Wainua, the drugmaker may be able to assist you. Check out their ‘Wainua WAY” support program at https://www.wainua.com/wainua-way-program to learn more about getting help with costs, your insurance coverage, and how to take your Wainua shots. You can also contact the drugmaker at 844-292-4682.