Vutrisiran

AMVUTTRA

Open Vutrisiran in the interactive console ↗

Pronunciation: vyoo-tri-SIR-an

Vutrisiran ballicule: receptor binding, kinetics and half-life diagram — Mechanism of action: Vutrisiran ballicule

Vutrisiran AMVUTTRA mascot mnemonic cartoon — Visual mnemonic: Vutrisiran mascot
**“Amyloid View tries running”**

What it isLiver-targeted siRNA that suppresses transthyretin production; ATTR polyneuropathy and cardiomyopathy.

Why this oneIt removes the amyloid precursor rather than stabilizing the circulating protein, with infrequent subcutaneous treatment.

What limits itVitamin A falls because transthyretin carries retinol, so supplementation and symptom surveillance are required.

FDA-approved for

hATTR polyneuropathy (2022)
ATTR cardiomyopathy (2022)

FDA label

Read the full label on DailyMed ↗

FDA dosing	Population	Start	Target	Max
Transthyretin-mediated amyloidosis (label trials: polyneuropathy of hATTR [HELIOS-A] and ATTR-CM [HELIOS-B])	Adults	25 mg SC once every 3 months	25 mg SC once every 3 months	25 mg SC q3mo

Renal No adjustment for mild-moderate impairment (eGFR 30 to <90 mL/min/1.73 m2); not studied in severe impairment or ESRD

Hepatic No adjustment for mild-moderate impairment; not studied in severe impairment

Instructions

SC use only, administered by a healthcare professional
If refrigerated, warm syringe to room temperature ~30 min before use; inspect — clear, colorless-to-yellow; discard if cloudy/particulates
Inject abdomen, thigh, or upper arm; avoid the 5-cm area around the navel and scarred/reddened/inflamed/swollen skin; pinch skin, insert at 45-90 degrees
Missed dose: administer as soon as possible, then resume q3mo from the most recently administered dose
Supplement vitamin A at the recommended daily allowance (do not exceed RDA); refer to ophthalmologist for ocular symptoms of vitamin A deficiency (e.g., night blindness)

Available strengths

inj 25 mg/0.5 mL

Cautions

Reduces serum vitamin A — supplement at RDA only; serum levels do not reflect total body vitamin A
Refer to ophthalmology if ocular symptoms suggestive of vitamin A deficiency (night blindness) develop

Adverse reactions

Pain in extremity
Arthralgia
Dyspnea
Decreased vitamin A

Pregnancy No human data; lowers serum vitamin A (essential for fetal development; excess also harmful); rat developmental toxicity only at maternally toxic doses

Lactation No data on presence in human milk; weigh breastfeeding benefit vs maternal need

Cost

Vutrisiran (AMVUTTRA)

Strength		#/mo	generic $/unit	CostPlus
25 mg/0.5 mL	inj	per unit		not offered

NADAC is a voluntary CMS survey of what retail community pharmacies pay for the drugs they stock. Vutrisiran is administered under clinical supervision and dispensed outside retail pharmacies, so it rarely appears on surveyed invoices.

NO BOXED WARNINGS

Principal riskVitamin A falls because TTR carries retinol, so supplementation is required

Classes and tags

Clinical profile

Therapeutic safety burden1 / 4

Overdose danger1 / 4

Weight-gain liability0 / 4

Sedation liability0 / 4

QT / Torsades0 / 4

Direct muscarinic antagonism0 / 4

Embryofetal risk2 / 4

Legacy pregnancy categoryN/A (post-2015 labeling)

Defining liabilityVitamin A depletion

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