Vutrisiran: Uses, Side Effects, Dosage - Drugvu

ℹ️ Compiled from public regulatory records · Last regulator revision: February 20, 2026🚩 Report this page

Vutrisiran

Other Nervous System Drugs

Sold as Amvuttra

GB MHRAEU EMACA Health Canada

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

Overview

Vutrisiran 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	February 20, 2026
EU European Union	EMA	1	August 13, 2025
CA Canada	Health Canada	1	December 31, 2025

GBUnited Kingdom· MHRA

1 product

Uses

GBOfficial regulatory label· revised February 20, 2026[1]

Amvuttra is indicated for the treatment of hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). Amvuttra is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

How to take

EUEuropean Union· EMA

1 product

Uses

EUOfficial regulatory label· revised August 13, 2025[2]

Amvuttra is indicated for the treatment of hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). Amvuttra is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

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)

AMVUTTRAALNYLAM NETHERLANDS B.V.

Sources & citations

[1]MHRA (UK) · PLGB505970006 · revised February 20, 2026
[2]European Medicines Agency · EMEA/H/C/005852 · revised August 13, 2025
[3]Health Canada (DPD) · 02542420 · revised December 31, 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.

Therapy should be initiated under the supervision of a physician knowledgeable in the management of amyloidosis. Treatment should be started as early as possible in the disease course to prevent the accumulation of disability. Posology The recommended dose of Amvuttra is 25 mg administered via subcutaneous injection once every 3 months.
4). 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. There is limited data with vutrisiran in patients with New York Heart Association (NYHA) Class IV and in patients who have both NYHA Class III and National Amyloidosis Centre (NAC) stage III.
However, if patients on vutrisiran progress to these stages, these data suggest that patients can remain on treatment. Missed dose If a dose is missed, Amvuttra should be administered as soon as possible. Dosing should be resumed every 3 months, from the most recently administered dose.
2). 5 to 3 x ULN and any AST) hepatic impairment. 2). 73 m2). 2). Paediatric population The safety and efficacy of Amvuttra in children or adolescents < 18 years of age have not been established. No data are available. Method of administration Amvuttra is for subcutaneous use only.
Amvuttra may be administered by a healthcare professional, the patient, or a caregiver. Patients or caregivers may inject Amvuttra after guidance has been provided by a healthcare professional on proper subcutaneous injection technique.
This medicinal product is ready-to-use and for single-use only. Visually inspect the solution for particulate matter and discolouration. Do not use if discoloured or if particles are present. Prior to administration, if stored cold, the pre-filled syringe should be allowed to warm by leaving carton at room temperature for about 30 minutes.
• The subcutaneous injection should be administered into one of the following sites: the abdomen, thighs, or upper arms. If injected in the upper arm, the injection should be administered by a healthcare professional or a caregiver.
Amvuttra should not be injected into scar tissue or areas that are reddened, inflamed, or swollen. • If injecting into the abdomen, the area around the navel should be avoided.

Side effects & warnings

GBOfficial regulatory label· Adverse reactions· revised February 20, 2026[1]

Tabulated list of adverse reactions The safety profile of Amvuttra was characterised based on the data from randomised-controlled phase 3 clinical studies. Adverse reactions reported in the pooled dataset of HELIOS-A and HELIOS-B studies are presented in Table 1.
The adverse reactions are presented as MedDRA preferred terms and under the MedDRA System Organ Class (SOC). The frequency of the adverse reactions is expressed according to the following category: Common (≥1/100 to <1/10).
Table 1:
Adverse reactions reported for Amvuttra System Organ Class Adverse Reaction Frequency General disorders and administration site conditions Injection site reactiona Common Alanine transaminase increased CommonInvestigations Blood alkaline phosphatase increased Common a Reported symptoms included bruising, erythema, pain, pruritus, and warmth.
Injection site reactions were mild, transient, and did not lead to treatment discontinuation Description of selected adverse reactions Liver function tests In the HELIOS-B study, 97 (30%) of patients treated with Amvuttra and 78 (24%) patients treated with placebo had a mild increased alanine aminotransferase (ALT) greater than the ULN and less than or equal to 3×ULN.
All patients treated with Amvuttra with mild ALT elevations were asymptomatic and the majority had normalization of ALT levels with continued dosing. 3%) Amvuttra- treated patients, respectively, developed anti-drug antibodies (ADA).
In both studies, ADA titres were low and transient with no evidence of an effect on clinical efficacy, safety, or pharmacokinetic or pharmacodynamic profiles of vutrisiran. 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

GBOfficial regulatory label· Warnings and precautions· revised February 20, 2026[1]

1). Serum vitamin A levels below the lower limit of normal should be corrected and any ocular symptoms or signs due to vitamin A deficiency should be evaluated prior to initiation of treatment with Amvuttra. Patients receiving Amvuttra should take oral supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day to reduce the potential risk of ocular symptoms due to vitamin A deficiency.
Ophthalmological assessment is recommended if patients develop ocular symptoms suggestive of 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 or too low vitamin A levels may be associated with an increased risk of foetal malformation. 6). If a woman intends to become pregnant, Amvuttra and vitamin A supplementation should be discontinued and serum vitamin A levels should be monitored and have returned to normal before conception is attempted.
Serum vitamin A levels may remain reduced for more than 12 months after the last dose of Amvuttra. 6). 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 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 Amvuttra. However, increasing vitamin A supplementation to above 3000 IU per day during pregnancy is unlikely to correct plasma retinol levels due to the mechanism of action of Amvuttra and may be harmful to the mother and foetus.
Sodium content This medicinal product contains less than 1 mmol sodium (23 mg) per mL, that is to say essentially ‘sodium-free’.

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

Therapy should be initiated under the supervision of a physician knowledgeable in the management of amyloidosis. Treatment should be started as early as possible in the disease course to prevent the accumulation of disability. Posology The recommended dose of Amvuttra is 25 mg administered via subcutaneous injection once every 3 months.
4). 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. There is limited data with vutrisiran in patients with New York Heart Association (NYHA) Class IV and in patients who have both NYHA Class III and National Amyloidosis Centre (NAC) stage III.
However, if patients on vutrisiran progress to these stages, these data suggest that patients can remain on treatment. 3 Missed dose If a dose is missed, Amvuttra should be administered as soon as possible. Dosing should be resumed every 3 months, from the most recently administered dose.
2). 5 to 3 × ULN and any AST) hepatic impairment. 2). 73 m2). 2). Paediatric population The safety and efficacy of Amvuttra in children or adolescents < 18 years of age have not been established. No data are available. Method of administration Amvuttra is for subcutaneous use only.
Amvuttra may be administered by a healthcare professional, the patient, or a caregiver. Patients or caregivers may inject Amvuttra after guidance has been provided by a healthcare professional on proper subcutaneous injection technique.
This medicinal product is ready-to-use and for single-use only. Visually inspect the solution for particulate matter and discolouration. Do not use if discoloured or if particles are present. Prior to administration, if stored cold, the pre-filled syringe should be allowed to warm by leaving carton at room temperature for about 30 minutes.
• The subcutaneous injection should be administered into one of the following sites: the abdomen, thighs, or upper arms. If injected in the upper arm, the injection should be administered by a healthcare professional or a caregiver.
Amvuttra should not be injected into scar tissue or areas that are reddened, inflamed, or swollen. • If injecting into the abdomen, the area around the navel should be avoided.

Side effects & warnings

EUOfficial regulatory label· Adverse reactions· revised August 13, 2025[2]

Tabulated list of adverse reactions The safety profile of Amvuttra was characterised based on the data from randomised-controlled phase 3 clinical studies. Adverse reactions reported in the pooled dataset of HELIOS-A and HELIOS-B studies are presented in Table 1.
The adverse reactions are presented as MedDRA preferred terms and under the MedDRA System Organ Class (SOC). The frequency of the adverse reactions is expressed according to the following category: Common (≥1/100 to <1/10).
Table 1:
Adverse reactions reported for Amvuttra System Organ Class Adverse Reaction Frequency General disorders and administration site conditions Injection site reactiona Common Investigations Alanine transaminase increased Common Blood alkaline phosphatase increased Common a Reported symptoms included bruising, erythema, pain, pruritus, and warmth.
Injection site reactions were mild, transient, and did not lead to treatment discontinuation 6 Description of selected adverse reactions Liver function tests In the HELIOS-B study, 97 (30%) of patients treated with Amvuttra and 78 (24%) patients treated with placebo had a mild increased alanine aminotransferase (ALT) greater than the ULN and less than or equal to 3×ULN.
All patients treated with Amvuttra with mild ALT elevations were asymptomatic and the majority had normalization of ALT levels with continued dosing. 3%) Amvuttra-treated patients, respectively, developed anti-drug antibodies (ADA).
In both studies, ADA titres were low and transient with no evidence of an effect on clinical efficacy, safety, or pharmacokinetic or pharmacodynamic profiles of vutrisiran. 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 August 13, 2025[2]

1). Serum vitamin A levels below the lower limit of normal should be corrected and any ocular symptoms or signs due to vitamin A deficiency should be evaluated prior to initiation of treatment with Amvuttra. Patients receiving Amvuttra should take oral supplementation of approximately, but not exceeding, 2 500 IU to 3 000 IU vitamin A per day to reduce the potential risk of ocular symptoms due to vitamin A deficiency.
Ophthalmological assessment is recommended if patients develop ocular symptoms suggestive of 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 or too low vitamin A levels may be associated with an increased risk of foetal malformation. 6). If a woman intends to become pregnant, Amvuttra and vitamin A supplementation should be discontinued and serum vitamin A levels should be monitored and have returned to normal before conception is attempted.
Serum vitamin A levels may remain reduced for more than 12 months after the last dose of Amvuttra. 6). 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 Amvuttra. However, increasing vitamin A supplementation to above 3 000 IU per day during pregnancy is unlikely to correct plasma retinol levels due to the mechanism of action of Amvuttra and may be harmful to the mother and foetus.
Sodium content This medicinal product contains less than 1 mmol sodium (23 mg) per 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