Eplontersen: Uses, Side Effects, Dosage - Drugvu

ℹ️ Compiled from public regulatory records · Last regulator revision: May 15, 2026🚩 Report this page

Eplontersen

Other Nervous System Drugs

Sold as Wainzua · WAINUA

GB MHRAEU EMACA Health Canada

Drug class: Other Nervous System Drugs
Availability: See label
Routes: Subcutaneous
Markets covered: 3
Products on record: 3

Overview

Eplontersen is an active pharmaceutical ingredient in the Other Nervous System Drugs group (N07XX). The information below is compiled per regulator from the product labels on record, with direct links to the original documents.

Regulatory status by market

Market	Regulator	Products	Last revision
GB United Kingdom	MHRA	1	May 15, 2026
EU European Union	EMA	1	January 27, 2026
CA Canada	Health Canada	1	September 18, 2025

GBUnited Kingdom· MHRA

1 product

Uses

GBOfficial regulatory label· revised May 15, 2026[1]

Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv amyloidosis) in adult patients with Stage 1 and 2 polyneuropathy.

How to take

EUEuropean Union· EMA

1 product

Uses

EUOfficial regulatory label· revised January 27, 2026[2]

Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.

How to take

CACanada· Health Canada

1 product

1 product on record with this regulator. Detailed label text (uses, dosage, side effects) is being ingested — the original document is linked under Sources [3].

Brands in Canada (1)

WAINUAASTRAZENECA CANADA INC

Sources & citations

[1]MHRA (UK) · PLGB179010377 · revised May 15, 2026
[2]European Medicines Agency · EMEA/H/C/006295 · revised January 27, 2026
[3]Health Canada (DPD) · 02548909 · revised September 18, 2025

Information on this page is compiled from public regulatory records. Drugvu is not affiliated with any regulator or pharmaceutical manufacturer. This is not medical advice. Always consult a qualified healthcare professional.

Treatment should be prescribed and supervised by a treating physician knowledgeable in the management of patients with amyloidosis. Posology The recommended dose of Wainzua is 45 mg administered by subcutaneous injection. Doses should be administered monthly.
4). Missed dose If a dose of Wainzua is missed, then the next dose should be administered as soon as possible. Resume dosing at monthly intervals from the date of the last dose. 2). 73 m2). 2). Hepatic impairment No dose adjustment is necessary in patients with mild hepatic impairment.
2). Patients undergoing liver transplant Wainzua has not been studied in patients undergoing liver transplant. Paediatric population The safety and efficacy of Wainzua in children and adolescents below 18 years of age have not been established.
No data are available. Method of administration Wainzua is for subcutaneous use only. Wainzua is a pre-filled pen for self- administration, ready to use and for single-use only. The first injection administered by the patient or caregiver should be performed under the guidance of an appropriately qualified health care professional.
Patients and/or caregivers should be trained in the subcutaneous administration of Wainzua. The pre-filled pen should be removed from refrigerated storage at least 30 minutes before use and allowed to reach room temperature prior to injection.
Other warming methods should not be used. Inspect solution visually before use. The solution should appear colourless to yellow. Do not use if cloudiness, particulate matter, or discolouration is observed prior to administration. If self-administered, inject Wainzua in the abdomen or upper thigh region.
If a caregiver administers the injection, the back of the upper arm can also be used. Wainzua should not be injected into skin that is bruised, tender, red, or hard, into scars or damaged skin, the area around the navel should be avoided.
Comprehensive instructions for administration are provided in the ‘Instructions for Use’.

Side effects & warnings

GBOfficial regulatory label· Adverse reactions· revised May 15, 2026[1]

Summary of the safety profile The safety data described below reflects exposure to Wainzua in 144 patients with polyneuropathy caused by ATTRv (ATTRv-PN) randomised to eplontersen and who received at least one dose of eplontersen. Of these, 141 patients received at least 6 months of treatment and 137 patients received at least 12 months of treatment.
The mean duration of treatment was 541 days (range: 57 to 582 days). The most frequent adverse reactions during treatment with eplontersen observed in ≥5% of patients were vitamin A decreased, injection site reactions, vomiting and proteinuria.
Tabulated list of adverse reactions Adverse reactions are organised by MedDRA System Organ Class (SOC). Within each SOC, preferred terms are arranged by decreasing frequency and then by decreasing seriousness. Frequencies of occurrence of adverse reactions are defined as: very common (≥1/10); common (≥1/100 to <1/10); uncommon (≥1/1,000 to <1/100); rare (≥1/10,000 to <1/1000); very rare (<1/10,000) and not known (cannot be estimated from available data).
Table 1:
Summary of adverse reactions reported for Wainzua System organ class Adverse reaction Frequency Gastrointestinal disorders Vomiting Common General disorders and administration site conditions Injection site reactions a Common Investigations Vitamin A decreased Very Common Renal and urinary disorders Proteinuria Common Eye disorders Cataracts Common a.
Injection site erythema, injection site pain, injection site pruritus Description of selected adverse reaction Vitamin A decreased In clinical study in patients with ATTRv-PN, all patients were instructed to take the recommended daily allowance of vitamin A.
1). 1% respectively. 3%) developed treatment-emergent anti-drug antibodies (ADAs). The presence of ADAs did not affect eplontersen plasma Cmax or AUC, but increased Ctrough. In the patients who tested positive for anti-eplontersen antibodies, there was no clinically meaningful impact on the efficacy, safety, pharmacokinetics, or pharmacodynamics of eplontersen.
Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store.
By reporting side effects you can help provide more information on the safety of this medicine.

GBOfficial regulatory label· Warnings and precautions· revised May 15, 2026[1]

1). 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 prior to initiation of treatment with Wainzua. Patients receiving Wainzua should take oral supplementation of approximately, but not exceeding, 2 500 IU (female) to 3 000 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. 6). If a woman intends to become pregnant, Wainzua and vitamin A supplementation should be discontinued and serum vitamin A levels should be monitored.
Before conception is attempted, vitamin A should have returned to normal levels. In the event of an unplanned pregnancy, Wainzua should be discontinued. 2), 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 3 000 IU per day, due to the lack of data supporting higher doses. Thereafter, vitamin A supplementation of 2 500 IU to 3 000 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 3 000 IU per day during pregnancy is unlikely to correct serum retinol levels due to the mechanism of action of eplontersen and may be harmful to the mother and foetus.
8 ml, that is to say essentially ‘sodium-free’.

This is not medical advice. Consult a qualified healthcare professional.

Treatment should be prescribed and supervised by a physician experienced in the treatment of patients with hereditary transthyretin-mediated amyloidosis. Posology The recommended dose of eplontersen is 45 mg administered monthly. 4).
1). The decision to continue treatment in those patients whose disease progresses to stage 3 polyneuropathy should be taken at the discretion of the physician based on the overall benefit and risk assessment. Missed dose If a dose of Wainzua is missed, then the next dose should be administered as soon as possible.
Dosing should be resumed at monthly intervals from the date of the last dose; a double dose should not be administered. 2). 73 m2). 2) and should only be used in these patients if the anticipated clinical benefit outweighs the potential risk.
Hepatic impairment No dose adjustment is necessary in patients with mild hepatic impairment. 2). Patients undergoing liver transplant The safety and efficacy of Wainzua have not been evaluated in patients undergoing liver transplant.
No data are available. Paediatric population The safety and efficacy of Wainzua in children and adolescents below 18 years of age have not been established. 1). Method of administration Wainzua is for subcutaneous use. Wainzua is a pre-filled pen for single-use only.
The first injection administered by the patient or caregiver should be performed under the guidance of an appropriately qualified health care professional. Patients and/or caregivers should be trained in the subcutaneous administration of Wainzua.
The pre-filled pen should be removed from refrigerated storage at least 30 minutes before use and allowed to reach room temperature prior to injection. Other warming methods should not be used. Inspect solution visually before use. The solution should appear colourless to yellow.
Do not use if cloudiness, particulate matter, or discolouration is observed prior to administration. If self-administered, Wainzua should be injected in the abdomen or upper thigh region. If a caregiver administers the injection, the back of the upper arm can also be used.
Wainzua should not be injected into skin that is bruised, tender, red, or hard, into scars or damaged skin, the area around the navel should be avoided. Comprehensive instructions for administration using the pre-filled pen are provided in the ‘Instructions for Use’.

Side effects & warnings

EUOfficial regulatory label· Adverse reactions· revised January 27, 2026[2]

Summary of the safety profile The most frequent adverse reactions during treatment with eplontersen were vitamin A decreased (97% of patients) and vomiting (9% of patients). Tabulated list of adverse reactions The safety data reflects exposure to Wainzua in 144 patients with polyneuropathy caused by ATTRv (ATTRv-PN) randomised to eplontersen and who received at least one dose of eplontersen.
130 patients completed treatment with eplontersen through Week 85. The mean duration of treatment was 541 days (range: 57 to 582 days). Adverse reactions are organised by MedDRA System Organ Class (SOC). Within each SOC, preferred terms are arranged by decreasing frequency and then by decreasing seriousness.
Frequencies of occurrence of adverse reactions are defined as: very common (≥ 1/10); common (≥ 1/100 to < 1/10); uncommon (≥ 1/1 000 to < 1/100); rare (≥ 1/10 000 to < 1/1 000); very rare (< 1/10 000) and not known (cannot be estimated from available data).
6 Table 1: Adverse reactions reported for Wainzua System organ class Adverse reaction Frequency Gastrointestinal disorders Vomiting Common General disorders and administration site conditions Injection site erythema Common Injection site pain Common Injection site pruritus Common Investigations Vitamin A decreased Very common* * Based on laboratory findings of vitamin A below the lower limit of normal during the study.
Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.

EUOfficial regulatory label· Warnings and precautions· revised January 27, 2026[2]

1). Serum vitamin A levels below the lower limit of normal should be corrected and any ocular symptoms or signs related to vitamin A deficiency should be evaluated prior to initiation of treatment with Wainzua. Patients receiving Wainzua should take oral supplementation of approximately, but not exceeding, 2 500 IU (female) to 3 000 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. 6). 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. 2), 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 3 000 IU per day, due to the lack of data supporting higher doses. Thereafter, vitamin A supplementation of 2 500 IU to 3 000 IU per day should be resumed in the second and third trimesters 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 3 000 IU per day during pregnancy is unlikely to correct serum retinol levels due to the mechanism of action of eplontersen and may be harmful to the mother and foetus.
8 ml, that is to say essentially ‘sodium-free’.

This is not medical advice. Consult a qualified healthcare professional.

Overview

Regulatory status by market

GBUnited Kingdom· MHRA

EUEuropean Union· EMA

CACanada· Health Canada

Drugs in the same class

Sources & citations