Vutrisiran
AMVUTTRA
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)
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
NO BOXED WARNINGS
Principal riskVitamin A falls because TTR carries retinol, so supplementation is required
Classes and tags
Clinical profile
Mechanism of action: Vutrisiran ballicule
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