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.
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:
NO
How Supplied:
Single-dose prefilled syringe—1
