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WAINZUA has been shown to be generally well tolerated with a manageable safety profile for patients with ATTRv-PN.1–3 In the NEURO-TTRansform study, WAINZUA demonstrated low treatment-related discontinuation rates: 4% (n=6/144) vs. 3% (n=2/60) in the placebo group after 66 weeks.2
The most frequent adverse reactions during treatment with WAINZUA observed in ≥5% of patients were vitamin A decreased, injection site reactions, vomiting and proteinuria.1
System organ class | Adverse reaction | Frequency* |
| Gastrointestinal disorders | Vomiting | Common |
| General disorders and administration site conditions | Injection site reactions† | Common |
| Investigations | Vitamin A decreased | Very common |
| Renal and urinary disorders | Proteinuria | Common |
| Eye disorders | Cataracts | Common |
*Frequencies of occurrence of adverse reactions are defined (by the Medical Disctionary for Regulatory Archives [MedDRA] System Organ Class) as very common (≥1/10); common (≥1/100 to <1/10); uncommon (≥1/1000 to <1/100); rare (≥1/10,000 to <1/1000); very rare (<1/10,000); and not known.
†Injection site erythema, injection site pain, injection site pruritus.1
All patients treated with WAINZUA had normal vitamin A levels at baseline and 96.5% (n=139/144) of those developed vitamin A levels below the lower limit of normal during the study.
Injection site erythema, injection site pain and injection site pruritus were reported in 3.5% (n=5/144), 3.5% (n=5/144), and 2.1% (n=3/144)
of patients, respectively.
After an 84-week treatment period (median treatment duration of 561 days [80 weeks], range: 57 to 582 days), 58 patients (40.3%) developed treatment-emergent anti-drug antibodies (ADA) between antibodies. However, there was no clinically meaningful impact on the efficacy, safety, pharmacokinetics, or pharmacodynamics of WAINZUA in these patients.
Based on the mechanism of action, WAINZUA is expected to reduce serum vitamin A below normal levels. Serum vitamin A levels below the lower limit of normal should be corrected and any symptoms or signs related to vitamin A deficiency should be evaluated before initiation of treatment with WAINZUA.
Patients receiving WAINZUA should take oral supplementation of approximately, but not exceeding, 2500 IU (female) to 3000 IU (male) of vitamin A per day to reduce the potential risk of ocular symptoms due to vitamin A deficiency. Referral for ophthalmological assessment is recommended if patients develop ocular symptoms consistent with vitamin A deficiency, including reduced night vision or night blindness, persistent dry eyes, eye inflammation, corneal inflammation or ulceration, corneal thickening or corneal perforation.
During the first 60 days of pregnancy, both too high and too low vitamin A levels may be associated with an increased risk of foetal malformation. Therefore, pregnancy should be excluded before initiation of WAINZUA therapy and women of childbearing potential should practise effective contraception. If a woman intends to become pregnant, WAINZUA and vitamin A supplementation should be discontinued and serum vitamin A levels should be monitored and have returned to normal before conception is attempted.
In the event of an unplanned pregnancy, WAINZUA should be discontinued. Due to the long half-life of WAINZUA, a vitamin A deficit may even develop after cessation of treatment. No recommendation can be given whether to continue or discontinue vitamin A supplementation during the first trimester of an unplanned pregnancy. If vitamin A supplementation is continued, the daily dose should not exceed 3000 IU per day, due to the lack of data supporting higher doses. Thereafter, vitamin A supplementation of 2500 IU to 3000 IU per day should be resumed in the second and third trimester if serum vitamin A levels have not yet returned to normal, because of the increased risk of vitamin A deficiency in the third trimester.
It is not known whether vitamin A supplementation in pregnancy will be sufficient to prevent vitamin A deficiency if the pregnant female continues to receive WAINZUA. However, increasing vitamin A supplementation to above 3000 IU per day during pregnancy is unlikely to correct serum vitamin A levels due to the mechanism of action of WAINZUA and may be harmful to the mother and foetus.
WAINZUA contains less than 1 mmol sodium (23 mg) per dose of 0.8 ml, that is to say essentially ‘sodium-free’.
Hypersensitivity to the active substance or to any of the excipients listed is contraindicated: sodium dihydrogen phosphate dihydrate, disodium hydrogen phosphate anhydrous, sodium chloride, hydrochloric acid, sodium hydroxide, and water for injection.
No clinical drug-drug interaction studies exist for WAINZUA. In-vitro data show that WAINZUA is not a substrate or inhibitor of transporters, does not interact with highly plasma protein bound drugs, and is not an inhibitor or inducer of cytochrome P450 (CYP) enzymes. Therefore, WAINZUA is not expected to cause or be affected by drug-drug interactions mediated through drug transporters, plasma protein binding or CYP enzymes.
Due to the potential teratogenic risk arising from unbalanced vitamin A levels, WAINZUA should not be used during pregnancy and in women of childbearing potential not using contraception. In case of pregnancy, close monitoring of the foetus and vitamin A status should be carried out, especially during the first trimester.
Human or animal lactation studies have not been conducted to assess the presence of WAINZUA or its metabolites in breast milk, the effects on the breastfed infant, or the effects on milk production for the mother. A risk to the breastfed child cannot be excluded. A decision must be made whether to discontinue breast feeding or to discontinue/abstain from WAINZUA therapy, taking into account the benefit of breast feeding for the child and the benefit of therapy for the woman.
The safety and efficacy of WAINZUA in patients under 18 years of age have not been established. No overall differences in pharmacokinetics were observed between adult and elderly (≥65 years of age) patients and no dose adjustment is required.
A population pharmacokinetic and pharmacodynamic analysis showed no clinically meaningful differences in the pharmacokinetics or pharmacodynamics of WAINZUA based on mild and moderate renal impairment (estimated glomerular filtration rate (eGFR) ≥45 to <90 ml/min) and on mild hepatic impairment (total bilirubin ≤ x Upper Limit of Normal (ULN) and aspartate aminotransferase (AST) >1 x ULN, or total bilirubin >1.0 to 1.5 x ULN and any AST). WAINZUA has not been studied in patients with severe renal impairment or end-stage renal disease, nor in patients with moderate or severe hepatic impairment or prior liver transplant.
There is no specific treatment for an overdose with WAINZUA. In the event of an overdose, supportive medical care should be provided, including consulting with a healthcare professional.
ADA=anti-drug antibody; AST=aspartate aminotransferase; ATTRv-PN=hereditary transthyretin amyloidosis with polyneuropathy; CYP=cytochromes P450; eGFR=estimated glomerular filtration rate; IU=international unit; MedDRA=The Medical Dictionary for Regulatory Archives; ULN=upper limit of normal.
GB-62846 | November 2025
Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. Adverse events should also be reported to AstraZeneca by visiting https://contactazmedical.astrazeneca.com/ or by calling 0800 783 0033.
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