For UAE, Kuwait, Bahrain, Qatar and Oman Healthcare Professionals Only
WAINUA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
FAQs
Frequently asked questions
Hereditary ATTR (hATTR) questions
Hereditary ATTR is a protein-misfolding disorder of the transthyretin (TTR) protein. TTR protein is primarily produced in the liver. In hATTR, mutations in the TTR gene result in unstable TTR protein, causing the tetrameric structure of TTR protein to break down, misfold, and aggregate into amyloid fibrils, ultimately leading to amyloid deposits throughout the body.1,2
ATTR, transthyretin-mediated amyloidosis.
References: 1. Tschöpe C, Elsanhoury A. Treatment of transthyretin amyloid cardiomyopathy: the current options, the future, and the challenges. J Clin Med. 2022;11(8):2148. 2. Ioannou A, Fontana M, Gillmore JD. RNA targeting and gene editing strategies for transthyretin amyloidosis. BioDrugs. 2023;37(2):127-142.
The most common manifestations of hATTR are autonomic neuropathy, cardiac, gastrointestinal, nephropathy, peripheral neuropathy, and musculoskeletal. The most common groupings of manifestations can seem nonspecific and unrelated.1,2
WAINUA is indicated for the treatment of the polyneuropathy of hATTR in adults.3
ATTR, transthyretin-mediated amyloidosis; hATTR, hereditary transthyretin-mediated amyloidosis.
References: 1. Adams D, Algalarrondo V, Polydefkis M, Sarswat N, Slama MS, Nativi-Nicolau J. Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression. Orphanet J Rare Dis. 2021;16(1):411. 2. Gertz M, Adams D, Ando Y, et al. Avoiding misdiagnosis: expert consensus recommendations for the suspicion and diagnosis of transthyretin amyloidosis for the general practitioner. BMC Fam Pract. 2020;21(1):198. 3. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
Some of the most common symptoms of hereditary ATTR are orthostatic hypotension, diarrhea, constipation, tripping, foot drop, muscle weakness, numbness and pain in feet, impaired balance, and falls. Learn more about the symptoms of hATTR.1
WAINUA is indicated for the treatment of the polyneuropathy of hATTR in adults.2
ATTR, transthyretin-mediated amyloidosis; hATTR, hereditary transthyretin-mediated amyloidosis.
References: 1. Kittleson MM, Ruberg FL, Ambardekar AV, et al; Writing Committee. 2023 ACC Expert Consensus Decision Pathway on comprehensive multidisciplinary care for the patient with cardiac amyloidosis: a report of the American College of Cardiology Solution Set Oversight Committee. J Am Coll Cardiol. 2023;81(11):1076-1126. 2. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
It’s important to understand the key hereditary ATTR symptoms that raise clinical suspicion of polyneuropathy and lead to diagnosis. During your neurologic evaluation of amyloidosis, look for autonomic dysfunction, sensory symptoms, and motor loss, and perform a physical examination. A genetic test can confirm if a patient has a transthyretin mutation and diagnose hATTR.1,2
ATTR, transthyretin-mediated amyloidosis; ATTR-PN, polyneuropathy of transthyretin-mediated amyloidosis; hATTR, hereditary transthyretin-mediated amyloidosis.
References: 1. Kittleson MM, Ruberg FL, Ambardekar AV, et al; Writing Committee. 2023 ACC Expert Consensus Decision Pathway on comprehensive multidisciplinary care for the patient with cardiac amyloidosis: a report of the American College of Cardiology Solution Set Oversight Committee. J Am Coll Cardiol. 2023;81(11):1076-1126. 2. Gertz M, Adams D, Ando Y, et al. Avoiding misdiagnosis: expert consensus recommendations for the suspicion and diagnosis of transthyretin amyloidosis for the general practitioner. BMC Fam Pract. 2020;21(1):198.
Genetic testing questions
Yes, a genetic test helps confirm a transthyretin mutation in order to diagnose hATTR. Most payors require a genetic test as proof of presence of hATTR prior to approval of WAINUA for hATTR-PN.
hATTR, hereditary transthyretin-mediated amyloidosis; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis.
Clinical trials questions
Yes, adults with hATTR-PN studied in the NEURO-TTRansform trial were representative of patients in real-world clinical practice. The trial included a broad age range of 18-82 years old (mean: 53), sex (69% male, 31% female), and race.1-4
hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis.
References: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025. 2. Coelho T, Ando Y, Benson MD, et al. Design and rationale of the global phase 3 NEURO-TTRansform study of antisense oligonucleotide AKCEA-TTR-LRX (ION-682884-CS3) in hereditary transthyretin-mediated amyloid polyneuropathy. Neurol Ther. 2021;10(1):375-389. 3. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458. 4. Coelho T, Waddington Cruz M, Chao CC, et al. Characteristics of patients with hereditary transthyretin amyloidosis-polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an open-label phase 3 study of eplontersen. Neurol Ther. 2023;12(1):267-287.
The following variants were included in WAINUA NEURO-TTRansform: V30M (59%), A97S (15%), T60A (3%), F64L (3%), L58H (3%), V122I (3%), S77Y (2%), E89Q (1%), S50R (1%), T49A (1%), and others (10%).*1
*Other TTR variants observed in 1 patient each: E62D, A101V, A39D, p.I127M, E74Q, E54Q, E54G, V32A, F44S, I127V; other TTR variants observed in 2 patients each: I107V, G47A.
TTR, transthyretin.
Reference: 1. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458.
80% of the patients had Coutinho stage 1 polyneuropathy and 20% had Coutinho stage 2 polyneuropathy in the NEURO-TTRansform trial.*1
*Severity of hATTR-PN was defined according to familial amyloid polyneuropathy or Coutinho stage: Stage 1 (ambulatory without assistance) or Stage 2 (ambulatory with assistance).1
hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis.
Reference: 1. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458.
At baseline, 27% of patients in the WAINUA NEURO-TTRansform clinical trial group (n=39, N=144) had a cardiomyopathy diagnosis.1
WAINUA is not indicated to treat cardiomyopathy symptoms.
Reference: 1. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458.
The co-primary endpoints of WAINUA looked at the LSM change from baseline in the mNIS+7 composite score, Norfolk QoL-DN total score, and serum TTR levels.1-3
LSM, least-squares mean; mNIS+7, modified Neuropathy Impairment Score +7; Norfolk QoL-DN, Norfolk Quality of Life-Diabetic Neuropathy.
References: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025. 2. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458. 3. Coelho T, Ando Y, Benson MD, et al. Design and rationale of the global phase 3 NEURO-TTRansform study of antisense oligonucleotide AKCEA-TTR-LRX (ION-682884-CS3) in hereditary transthyretin-mediated amyloid polyneuropathy. Neurol Ther. 2021;10(1):375-389.
ATTR amyloidosis is a protein-misfolding disease of the TTR protein.1,4 Serum TTR levels as an endpoint in the NEURO-TTRansform trial measured the percentage change from baseline between treatment groups in the TTR protein found in blood.2 WAINUA is designed to reduce serum TTR protein and subsequent TTR protein deposits in tissues.3
ATTR, transthyretin-mediated amyloidosis; ATTR-PN, polyneuropathy of transthyretin-mediated amyloidosis; TTR, transthyretin.
References: 1. Ioannou A, Fontana M, Gillmore JD. RNA targeting and gene editing strategies for transthyretin amyloidosis. BioDrugs. 2023;37(2):127-142. 2. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458. 3. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025. 4. Tschöpe C, Elsanhoury A. Treatment of transthyretin amyloid cardiomyopathy: the current options, the future, and the challenges. J Clin Med. 2022;11(8):2148.
The mNIS+7 composite score quantifies neurologic impairment and progression across a variety of measures, including muscle strength/weakness, quantitative sensory testing, sensations, reflexes, nerve conduction, and heart rate response to deep breathing.1,2
ATTR-PN, polyneuropathy of transthyretin-mediated amyloidosis; mNIS+7, modified Neuropathy Impairment Score +7.
References: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025. 2. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458.
The Norfolk QoL-DN composite score is a patient-reported quality-of-life assessment across 5 domains, including physical function/large-fiber neuropathy, activities of daily living, symptoms, small-fiber neuropathy, and autonomic nerve function.1
ATTR-PN, polyneuropathy of transthyretin-mediated amyloidosis; Norfolk QoL-DN, Norfolk Quality of Life-Diabetic Neuropathy.
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
The WAINUA NEURO-TTRansform clinical trial included secondary and prespecified exploratory endpoints that assessed autonomic function and nutritional status.1-3
A secondary endpoint of WAINUA looked at the change in mBMI from baseline.1,2
The study also included a prespecified exploratory endpoint that looked at the mean change from baseline with WAINUA in COMPASS-31 total score.1-3
COMPASS-31, Composite Autonomic Symptom Score-31; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis; mBMI, modified body mass index.
References: 1. Coelho T, Ando Y, Benson MD, et al. Design and rationale of the global phase 3 NEURO-TTRansform study of antisense oligonucleotide AKCEA-TTR-LRX (ION-682884-CS3) in hereditary transthyretin-mediated amyloid polyneuropathy. Neurol Ther. 2021;10(1):375-389. 2. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458. 3. Wixner J, Berk J, Adams D, et al. Eplontersen improves autonomic neuropathy symptoms in hereditary ATTR: an analysis from NEURO-TTRansform. Presented at: 4th International ATTR Amyloidosis Meeting; November 2-3, 2023; Madrid, Spain.
Safety questions
WAINUA has no contraindications.1
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
Yes, treatment with WAINUA leads to a decrease in serum vitamin A levels.1
Because TTR is the primary carrier of retinol, and in order to ensure adequate delivery of vitamin A to tissues in the setting of low serum TTR concentrations, vitamin A supplementation is a requirement in patients who are prescribed any TTR silencer. Patients should supplement with the recommended daily allowance of approximately, but not exceeding, 3,000 IU of vitamin A per day while taking WAINUA. The recommended daily allowance of vitamin A may depend on a number of factors including geography, sex, age, and population.1-5
Higher doses than the recommended daily allowance of vitamin A should not be given to try to achieve normal serum vitamin A levels during treatment with WAINUA, as serum vitamin A levels do not reflect the total vitamin A in the body.1
In the NEURO-TTRansform study, patients were instructed to take the recommended daily allowance of vitamin A. All patients treated with WAINUA had normal vitamin A levels at baseline, 95% of patients developed low vitamin A levels during the study. In some cases, the decreased vitamin A level was reported as an adverse reaction.1
Vitamin A decrease was reported as an adverse reaction in 15% of the WAINUA-treated patients (N=144).1
If ocular symptoms suggestive of vitamin A deficiency occur, refer patient to an ophthalmologist.1
TTR, transthyretin.
References: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025. 2. Vinik EJ, Vinik AI, Paulson JF, et al. Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2014;19(2):104–114. 3. Ioannou A, Fontana M, Gillmore JD. RNA targeting and gene editing strategies for transthyretin amyloidosis. BioDrugs. 2023;37(2):127-142. 4. National Institute for Health and Care Excellence. Highly specialised technology evaluation: inotersen for treating hereditary transthyretin-related amyloidosis [ID1242]. 2018. 5. Tanumihardjo SA, Russell RM, Stephensen CB, et al. Biomarkers of Nutrition for Development (BOND)-vitamin A review. J Nutr. 2016;146(9):1816S-1848S.
There are no laboratory monitoring requirements for WAINUA.1
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
Dosing and administration questions
No premedication is required with WAINUA.1
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
In the case that a patient misses a dose, they should administer their next injection as soon as possible and then resume the standard monthly dosing interval of WAINUA from the date of the last dose.1
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
WAINUA can be administered by a caregiver; the steps are the same. Everyone should be trained by the healthcare provider and read the Instructions for Use that came with the WAINUA autoinjector.1
AstraZeneca Patient Education Managers (PEMs) can assist in injection training and answer any questions for patients and caregivers, both live and virtually. Patients and caregivers can also view the instructional video at WAINUA.com/HowToVideo for more information.
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
Storage questions
WAINUA should be stored in the original carton, protected from light in either the refrigerator or at room temperature, if needed. If storing in the refrigerator, WAINUA should be refrigerated at 2°C to 8°C (36°F to 46°F). Do not freeze.1
WAINUA can be stored at room temperature up to 30°C (86°F) for up to 6 weeks; if not used within the 6 weeks, WAINUA should be discarded. Do not expose to heat.1
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
WAINUA can be stored at room temperature up to 30°C (86°F) for up to 6 weeks; if not used within the 6 weeks, WAINUA should be discarded. Do not expose to heat.1
Reference: 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
Access questions
WAINUA WAY offers a variety of resources for patients, including dedicated educator, educational resources, autoinjector onboarding and support, as well as access and reimbursement assistance, including a commercial co-pay savings program where patients can pay as little as [$0] for WAINUA if eligible.
The AstraZeneca Access 360™ program provides personal support to help streamline access and reimbursement for WAINUA. Access 360 provides:
- Assistance with understanding patient insurance coverage and pharmacy options
- Prior authorization support
- Claims and appeal process support
- Eligibility requirements and enrollment assistance with AstraZeneca’s Co-pay Savings Programs
- Referrals to AZ&Me™ Prescription Savings Program, AstraZeneca’s patient assistance program
- Information about independent charitable patient assistance foundations
To learn more about the Access 360 program, please call 1-844-2-WAINUA (1-844-292-4682) Monday through Friday, 8 AM - 6 PM ET or visit www.MyAccess360.com.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
- Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.
ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).
INDICATION
WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.