Prescribing guide
Wainua (eplontersen)For hereditary ATTR polyneuropathy: dosing, self-administration, vitamin A, storage and prior authorization criteriaFor prescribers and their offices treating hereditary ATTR amyloidosis with polyneuropathy: what the pharmacy needs before it ships, what the prior authorization wants documented, the titration and the monitoring the label requires.
Dispensing gate
No REMS. The Enrollment Form faxed to Access 360, carrying the prescription when Orsini dispenses; prior authorization; training of the patient or caregiver before the autoinjector is started, or healthcare provider administration of the prefilled syringe.
Example PA threshold
Cigna example: age 18 or older, pathogenic TTR variant on genetic testing and symptomatic polyneuropathy, both documented, no liver transplant, neurologist, geneticist or amyloidosis specialist, Amvuttra tried first unless already on Wainua, no concurrent TTR therapy.
Monitoring
No labs required by the label; vitamin A supplementation at the RDA, not titrated to serum levels; ophthalmology referral for ocular symptoms suggestive of deficiency, such as night blindness or dry eyes.
Support line
AstraZeneca Access 360 and WAINUA WAY, 1-844-292-4682, Monday through Friday, 8 AM to 6 PM ET; benefits investigation, PA and appeal support, a commercial Savings Program from $0, a Free Limited Supply during an insurer delay, AZ&Me referral, and Patient Education Managers for injection training.
Give your patient their plan
A one-page handout for starting Wainua, in plain language: what the first fill waits on, the first dates, who will call and the symptoms to report, with blank lines for the start date and your office's number. The link opens the patient's full first 90 days page, where their dates become calendar entries.
trialfriend.com/p/wainuaBefore the first fill: REMS, labs and enrollment
The steps that the pharmacy, the REMS or the drugmaker's program will wait on, in the order they usually need to happen. A first fill of a specialty drug most often stalls on one of these rather than on the plan.
| Step | What it requires | Source |
|---|---|---|
| Submit the Enrollment Form | Fax pages 1, 3 and 4 to Access 360. The form prints the fax as 1-844-329-2360 (1-844-FAX-A360) on every page, while the Access & Support page prints 1-833-329-2360, and the form that page's own Download link opens also prints 1-844-329-2360, so confirm the number with Access 360 at 1-844-292-4682. Patient name, date of birth and address are required. The ICD-10-CM code preprinted on the form is E85.1. It asks for current treatments and whether Wainua replaces one. | Enrollment Form |
| Or enroll online | The Access & Support page links an online enrollment form in 2 versions, with the patient present and without. A Patient Authorization Form is required for Access 360 support, on page 2 of the Enrollment Form or as the electronic ePAF. | Wainua HCP access |
| Patient authorization | Page 1 carries the Patient Authorization and a separate WAINUA Support Authorization (Savings Program and Additional Support) above a patient signature line; the form says the Support Program box must be checked for co-pay support. The authorization is required for PA follow-up and expires 2 years from signing. A patient unavailable to sign can authorize at www.myaccess360paf.com or 1-844-275-2360. | Enrollment Form |
| Prescription on the form | Section 5A is completed only when Orsini Specialty Pharmacy dispenses. Autoinjector, patient administered: 45mg subcutaneous once monthly, quantity 1, refills 11 preprinted, acquisition through Orsini. Prefilled syringe, healthcare provider administered: choose the administration site (physician office, hospital outpatient, alternate site of care, home health) and acquisition through Orsini or buy and bill. A stamped signature or one signed on the prescriber's behalf is not permitted. | Enrollment Form |
| Pharmacy and distribution | Orsini is the network specialty pharmacy, phone 1-800-986-4975, NPI 1073608998; Alivia Specialty Pharmacy fulfills in Puerto Rico only. A health system's own specialty pharmacy can also dispense. Authorized specialty distributors: ASD Healthcare, Besse Medical, Biocare SD, Cardinal Health Specialty Pharmaceutical Distribution, CuraScript SD, Dakota Drug, DMS Pharmaceutical Group, McKesson Plasma and Biologics, Morris & Dickson Specialty Distribution and Optum SD. | Wainua HCP access |
| Benefit verification and PA support | Access 360 assists with understanding coverage and pharmacy options, prior authorization, and the claims and appeal process; it does not file claims or appeals. The page links a PA and appeal checklist and sample letters for appeal (general, concomitant use, testing documentation, product change) and for medical necessity. AstraZeneca reports that the majority of patients receive Wainua within 10 business days, from Orsini shipments between January 1 and October 28, 2024. | Wainua HCP access |
| Free Limited Supply when the PA stalls | Optional section 5B of the Enrollment Form, for eligible patients who face a delay in approval by their insurer. The patient must have an FDA-approved diagnosis, and the prescriber attests that no reimbursement is sought for the free product. The form calls the program temporary and does not state a quantity or a waiting period. | Enrollment Form |
| Savings Program terms | Commercial insurance that covers Wainua, a valid prescription, US or Puerto Rico residence and enrollment; eligible patients may pay as little as $0. Not valid for Medicare Part D, Medicaid, Medigap, VA, DoD or TRICARE, or Medicare-eligible patients in an employer group waiver or subsidized retiree drug plan, even if processed as cash-paying. A calendar-year Maximum Savings Limit applies; the page does not print its amount. Benefits may vary or end under copay maximizer or accumulator programs. Claims older than 365 days from the date of service are invalid. | Wainua savings |
| Uninsured or Medicare patients who cannot afford it | The Enrollment Form points to AZ&Me, at www.azandmeapp.com or 1-800-292-6363. Access 360 also gives information about independent charitable patient assistance foundations. | Enrollment Form |
| Injection training support | AstraZeneca Patient Education Managers can assist in injection training and answer questions for patients and caregivers, live and virtually. AstraZeneca also states that no premedication is required. | Wainua HCP FAQ |
| Choose the presentation | Carton of one 45mg/0.8mL single-dose autoinjector, NDC 0310-9400-01, or carton of one 45mg/0.8mL single-dose prefilled syringe, NDC 0310-9420-01. The prefilled syringe must be administered by a healthcare provider. | FDA label |
| Train before the first autoinjector dose | The label says to train patients and/or caregivers on preparation and administration of the autoinjector prior to initiation, using the Instructions for Use. | FDA label |
| Counsel on vitamin A | Instruct the patient to take the recommended daily allowance of vitamin A and to contact the office for ocular symptoms such as night blindness or dry eyes. The Patient Information asks patients to report vitamin A and beta-carotene supplements. The label gives no start day for the supplement. | FDA label |
| Pregnancy and lactation | No data in pregnant women. The label asks patients to report pregnancy or plans for it and to be advised of the potential fetal risk, including reduced serum vitamin A. No information on presence in human milk. Vitamin A is essential for normal embryofetal development, excessive levels are associated with adverse developmental effects, and the effect of supplementation on the fetus when maternal serum TTR is reduced is unknown. Weigh the benefits of breastfeeding against the mother's clinical need for Wainua and any potential adverse effects on the breast-fed infant. | FDA label |
| Storage counseling | Refrigerate at 2 to 8 degrees C in the original carton, protected from light. Up to 6 weeks at room temperature up to 30 degrees C in the original carton if needed, then discard. Do not freeze; do not expose to heat. | FDA label |
Prior authorization criteria and what to document
One public policy as the example: Cigna, October 1, 2026. Approval a year at a time. The policy lists no separate renewal criteria. This is Cigna's Drug Coverage Policy IP0628, which took effect on October 1st. Its reference list cites the April 2025 prescribing information, not the April 2026 label, though it already covers the prefilled syringe. Trying Amvuttra first and the ban on combining TTR medicines are the plan's rules, not the label's. For the autoinjector on employer plans, the policy ties the Amvuttra rule to the Value, Advantage and Total Savings drug lists. The policy says a member's own plan document always overrides it. It is here as an example of what a plan asks for, not as a rule.
| Element | What the policy asks | In the chart | Source |
|---|---|---|---|
| Diagnosis | Polyneuropathy of hereditary transthyretin-mediated amyloidosis, age 18 or older | Diagnosis note; Cigna names no ICD-10 codes | Cigna policy |
| Genetic confirmation | A transthyretin pathogenic variant confirmed by genetic testing, documentation required | Genetic test report | Cigna policy |
| Symptomatic polyneuropathy | Documentation required. Examples: reduced motor strength or coordination, impaired sensation (pain, temperature, vibration, touch); assessed by history and clinical exam, electromyography or nerve conduction velocity testing | Neurology note, EMG or nerve conduction report | Cigna policy |
| Liver transplant | No history of liver transplantation | Surgical history in the note | Cigna policy |
| Prescriber | Neurologist, geneticist or a physician who specializes in the treatment of amyloidosis, prescribing or consulting | Specialty on the request | Cigna policy |
| Preferred product | Amvuttra tried with inadequate efficacy or significant intolerance according to the prescriber, or the patient has already been started on Wainua. Applies to the autoinjector (employer plans on the Value, Advantage and Total Savings lists; individual and family plans) and to the prefilled syringe | Prior therapy dates and outcome, or the Wainua start date | Cigna policy |
| Combination | No concurrent Amvuttra, Attruby, Onpattro, Tegsedi or tafamidis product; Cigna treats concurrent use as not medically necessary | Current medication list; a stop date if switching | Cigna policy |
| Dose | 45mg subcutaneously once monthly | Order | Cigna policy |
| Form of documentation | Chart notes, laboratory tests, medical test results, genetic test results or claims records, each with patient-specific identifying information. Approval is for 1 year; no separate renewal criteria are listed | Attach to the request | Cigna policy |
| Drugmaker's note on payers | AstraZeneca states that most payors require a genetic test as proof of hATTR before approving Wainua | Genetic test report | Wainua HCP FAQ |
Wainua dosing and titration
| When | Label | Source |
|---|---|---|
| Day 1 | 45mg (0.8mL) by subcutaneous injection: the entire contents of one single-dose autoinjector or one single-dose prefilled syringe. | FDA label |
| Monthly | 45mg once monthly; no titration. No dose adjustment for age 65 or older, mild to moderate renal impairment (eGFR 30 to under 90), or mild hepatic impairment. Not studied in severe renal impairment, ESRD, moderate or severe hepatic impairment or after liver transplant. Safety and effectiveness in pediatric patients have not been established. In patients 65 or older no overall differences in safety or effectiveness were observed, but greater sensitivity of some older individuals cannot be ruled out. | FDA label |
| Missed dose | Administer as soon as possible, then resume at monthly intervals from the date of the most recently administered dose. | FDA label |
| Preparation | Remove from the refrigerator 30 minutes before injection; no other warming methods. Solution colorless to yellow; do not use if cloudy, discolored or containing particulate matter, or if dropped, damaged, apparently tampered with or expired. | FDA label |
| Injection site | Abdomen or upper thigh; back of the upper arm only if a healthcare provider or caregiver injects. Avoid the 2 inches (5cm) around the navel and skin that is red, warm, tender, bruised, scaly or hard. Rotate sites with each injection. | FDA label |
| Autoinjector | Needle guard flat on the skin at 90 degrees; press and hold for 10 seconds until the orange plunger fills the viewing window. An unfilled window may mean an incomplete dose, and the Instructions for Use tell the patient to contact the healthcare provider. | FDA label |
| Prefilled syringe | Healthcare providers only. Do not remove the needle cover until ready to inject, do not recap, and do not touch the needle guard activation clips, to prevent premature activation of the needle safety guard. Do not expel small air bubbles. Pinch the skin and insert at approximately 45 degrees; the needle safety device triggers once the entire dose is injected and retracts the needle when the plunger is released. | FDA label |
Monitoring the Wainua label requires
| When | What | What the label says to do with it | Source |
|---|---|---|---|
| Baseline and ongoing | No laboratory test required by the label | The label's one warning is reduced serum vitamin A | FDA label |
| Throughout | Vitamin A supplementation at the RDA | Do not dose above the RDA to normalize serum vitamin A; serum levels do not reflect total body vitamin A. Study 1: all normal at baseline, 95% went low; mean steady-state reduction 71% by week 37 | FDA label |
| Any time | Ocular symptoms suggestive of vitamin A deficiency, such as night blindness or dry eyes | Refer to an ophthalmologist | FDA label |
| Any time | Conduction | Study 1 recorded 3 serious adverse reactions of AV block (2%), 1 complete; the label gives no monitoring instruction. At 2.7 times the recommended dose, no clinically significant QTc prolongation | FDA label |
| After each dose | Injection site | Reactions in 7% in Study 1: erythema, pain, pruritus | FDA label |
| Other adverse reactions | Study 1, at least 5% of 144 patients | Vitamin A decreased 15%, vomiting 9%, proteinuria 8%, blurred vision 6%, cataract 6% | FDA label |
| Pharmacodynamics | Serum TTR | Least squares mean reduction of 81% from baseline at week 35, similar across subgroups including Val30Met status, body weight, sex, age and race | FDA label |
Wainua drug interactions and contraindications
| Label category | Agents and what the label says | Source |
|---|---|---|
| Contraindications | None | FDA label |
| CYP enzymes and transporters | No clinical interaction studies. In vitro, not a substrate or inhibitor of transporters, no interaction with highly plasma protein bound drugs, and not an inhibitor or inducer of CYP enzymes. Metabolized by endo- and exonucleases in the liver | FDA label |
| Vitamin A and beta-carotene products | Supplement at the RDA only; the Patient Information asks patients to report vitamin A and beta-carotene supplements | FDA label |
| Immunogenicity | Treatment-emergent anti-drug antibodies in 37% (53 of 144) in Study 1. They raised trough concentration but not Cmax or AUC, with no effect found on pharmacodynamics, safety or efficacy; the label calls the data too limited for definitive conclusions | FDA label |
| Approval history | NDA 217388, sponsor AstraZeneca AB, approved December 21, 2023; supplement S-005, approved April 15, 2026, carries the current label with the prefilled syringe | Drugs@FDA record |
| Payer rule on combinations | Cigna: concurrent Amvuttra, Attruby, Onpattro, Tegsedi or a tafamidis product is not medically necessary, citing a lack of controlled clinical trial data supporting additive efficacy | Cigna policy |
| Billing code | Cigna's coding table lists HCPCS C9311, injection, eplontersen, 1mg, usable for Medicare when given under direct physician supervision and not when self-administered. C9399 and J3490 are listed as effective until September 30, 2026 | Cigna policy |
| REMS | None. Wainua does not appear on the FDA's list of approved REMS, checked November 12, 2026 | FDA REMS list |
| Vitamin A amount, per AstraZeneca | AstraZeneca's prescriber FAQ describes the recommended daily allowance as approximately, but not exceeding, 3,000 IU a day and says it may depend on geography, sex, age and population. The label states only the recommended daily allowance | Wainua HCP FAQ |
| Postmarketing requirements | The December 2023 approval letter, addressed to Ionis Pharmaceuticals, required a pregnancy and lactation outcomes study, a milk-only lactation study, and analyses of glomerulonephritis, thrombocytopenia and atrioventricular block in the placebo-controlled cardiomyopathy trial ION-682884-CST | FDA letter |
Why a first fill stalls, and the fix
- Prior authorization pending or delayedThe enrollment form lets the prescriber request a Free Limited Supply for eligible patients facing an insurance approval delay. The form says the patient must have an FDA-approved diagnosis, and it calls the program temporary.
- The plan wants more recordsCigna asks for documentation of a disease-causing TTR variant found by genetic testing and of symptomatic polyneuropathy. Chart notes, test results and genetic test results are the kinds of records it names.
- The plan wants Amvuttra tried firstCigna's policy asks that Amvuttra was tried and, according to the prescriber, worked inadequately or was not tolerated. The other way to meet that rule is to have already started Wainua.
- The plan will not pay alongside another TTR medicineCigna treats Wainua given with Amvuttra, Attruby, Onpattro, Tegsedi or a tafamidis product as not medically necessary. That is the plan's rule, and the neurologist decides what you take.
- The denial needs an appealAccess 360 offers prior authorization support and support with the claims and appeal process. AstraZeneca notes that Access 360 does not file claims or appeals for patients or offices.
- A cost you cannot payCommercial insurance: the WAINUA Savings Program, with which eligible patients may pay as little as $0. People on Medicare, Medicaid, Medigap, VA, Department of Defense or TRICARE coverage cannot use it. Access 360 can explain other financial assistance programs, including for people without coverage.
- No insurance, or Medicare and the cost is out of reachThe enrollment form says AZ&Me may be able to help patients without coverage, or on Medicare, who cannot afford their medicine. The form lists www.azandmeapp.com and 1-800-292-6363 as the ways to reach that program.
- A damaged autoinjectorAstraZeneca's patient site says not to use a damaged autoinjector and to call 1-844-292-4682 for a replacement.
WAINUA WAY and AstraZeneca Access 360
- AstraZeneca Access 360, which can check your insurance coverage, explain what your plan provides and tell you which financial assistance programs you may be eligible for
- The WAINUA Savings Program, with which eligible people with commercial insurance may pay as little as $0, once the plan has approved Wainua and they have enrolled
- No savings program for Medicare Part D, Medicaid, Medigap, VA, Department of Defense or TRICARE coverage, and a yearly maximum savings limit whose amount the page does not print
- For Medicare Part D, a note that out-of-pocket costs may be paid monthly or in full upfront
- A Patient Education Manager as a dedicated contact, who can meet at home or virtually to share resources on how to take Wainua How to take Wainua
- Patient Education Managers who can assist with injection training for patients and caregivers, live or virtually Wainua HCP FAQ
- A welcome kit after enrolling in WAINUA WAY, with answers to common questions and tools to get started WAINUA WAY
- A demonstration autoinjector with no needle or medicine for practice, for people prescribed Wainua and enrolled in WAINUA WAY How to take Wainua
- A Free Limited Supply, requested by the prescriber, for eligible patients facing a delay in insurance approval Enrollment Form
- AZ&Me, AstraZeneca's patient assistance program, for people without coverage or on Medicare who cannot afford the medicine (1-800-292-6363) Enrollment Form
Questions prescribers' offices ask about Wainua
What is the Wainua dose for hATTR polyneuropathy?
The April 2026 label sets 45mg by subcutaneous injection once monthly, with no titration and no dose adjustment for age 65 or older, mild or moderate renal impairment, or mild hepatic impairment. A missed dose is given as soon as possible, and dosing resumes at monthly intervals from the most recently administered dose.
Who can administer Wainua?
Under the April 2026 label, the 45mg/0.8mL autoinjector is intended for self-administration by patients or administration by caregivers after training, and the 45mg/0.8mL prefilled syringe must be administered by a healthcare provider. Sites are the abdomen or upper thigh; the back of the upper arm only when a healthcare provider or caregiver injects.
What does the label say about vitamin A on Wainua?
The April 2026 label reports that 95% of patients in Study 1, all normal at baseline, developed low serum vitamin A, with a 71% mean steady-state reduction by week 37. It advises supplementation at the recommended daily allowance and says higher doses should not be given to normalize serum levels. Refer to an ophthalmologist for ocular symptoms suggestive of deficiency, such as night blindness or dry eyes.
Does Wainua require a REMS or laboratory monitoring?
Neither: Wainua is not on the FDA's list of approved REMS, checked November 12, 2026, and the April 2026 label requires no laboratory test. The label has no boxed warning, no contraindications and one warning, reduced serum vitamin A. It records 3 serious adverse reactions of AV block (2%) in Study 1, 1 of them complete, without a monitoring instruction.
What do insurers ask for to approve Wainua?
The Cigna policy on this page, IP0628, effective October 1, 2026, approves for 1 year with age 18 or older, a pathogenic TTR variant on genetic testing and symptomatic polyneuropathy, both documented. It also asks for no history of liver transplantation, a neurologist, geneticist or amyloidosis specialist, a trial of Amvuttra unless the patient has already started Wainua, and no concurrent TTR therapy. Its reference list cites the April 2025 prescribing information rather than the April 2026 label.
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Sources
- WAINUA (eplontersen) injection, for subcutaneous use, with Patient Information (Approved: 04/2026) and Instructions for Use, AstraZeneca Pharmaceuticals LP, via DailyMed, 2026-04-15.
- NDA 217388, WAINUA (eplontersen sodium), submissions and approval dates, FDA, via openFDA, 2026-11-12.
- Savings and Affordability | WAINUA® (eplontersen), AstraZeneca, 2026-11-12.
- WAINUA WAY Support Program for Patients | WAINUA® (eplontersen), AstraZeneca, 2026-11-12.
- How to Take 45 mg Injection for Subcutaneous Use | WAINUA® (eplontersen), AstraZeneca, 2026-11-12.
- Frequently Asked Questions | WAINUA® (eplontersen), AstraZeneca, 2026-11-12.
- WAINUA® (eplontersen) Enrollment Form, AstraZeneca Access 360 (US-104756, Last Updated 3/26), AstraZeneca, 2026-11-12.
- Access & Support | WAINUA® (eplontersen) | For HCPs, AstraZeneca, 2026-11-12.
- Frequently Asked Questions | WAINUA® (eplontersen) | For HCPs, AstraZeneca, 2026-11-12.
- Drug Coverage Policy IP0628, Wainua, Cigna, 2026-10-01.
- Approved Risk Evaluation and Mitigation Strategies (REMS), FDA, 2026-11-12.
- NDA 217388/S-005 supplement approval letter, single-dose prefilled syringe, FDA, 2026-04-15.
- NDA 217388/S-006 supplement approval letter, carton quick reference guide and autoinjector Instructions for Use, FDA, 2025-12-17.
- NDA 217388/S-004 supplement approval letter, section 11 description, FDA, 2025-04-18.
- NDA 217388/S-002 supplement approval letter, section 13.1 mouse carcinogenicity, FDA, 2024-09-25.
- NDA 217388 approval letter, FDA, 2023-12-21.
A reference drawn from the April 15, 2026 label, the drugmaker's published program and one public insurer policy shown as an example, checked on November 12, 2026. Labels and policies change; the label on DailyMed and the plan's current policy govern. Trial Friend is not a medical provider. It reports what these documents say and does not judge whether a drug is safe or right for any patient; that is the prescriber's decision. Not a substitute for the prescribing information.