Inborn errors of metabolism:

Indications for: AMVUTTRA

Polyneuropathy of hereditary transthyretin-mediated amyloidosis.

Adult Dosage:

Give by SC inj in abdomen, thighs, or upper arms. 25mg once every 3 months.

Children Dosage:

Not established.

AMVUTTRA Warnings/Precautions:

Supplement with recommended daily Vit. A allowance. Refer for eye exam if ocular symptoms of Vit. A deficiency occur (eg, night blindness). Moderate or severe hepatic impairment, severe renal impairment, or ESRD: not studied. Elderly. Pregnancy. Nursing mothers.

AMVUTTRA Classification:

Transthyretin-directed small interfering RNA.

Adverse Reactions:

Arthralgia, dyspnea, Vit. A decreased; inj site reactions, AV heart block.


Primary pathway: by endo- and exonucleases to short nucleotide fragments of varying sizes within the liver.

Drug Elimination:

Primary pathway: mean fraction of unchanged vutrisiran eliminated in urine was ~19.4% at the recommended dose of 25 mg. The mean renal clearance of vutrisiran ranged from 4.5 to 5.7 L/hour.

Half-life (Median [range]): 5.2 (2.2, 6.4) hours. Apparent clearance (Median [range]): 21.4 (19.8, 30) L/hour.

Generic Drug Availability:


How Supplied:

Single-dose prefilled syringe—1