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Therapeutic Classification: none assigned

Pharmacologic Classification: antisense oligonucleotides

Indications ⬆ ⬇

REMS

Polyneuropathy of hereditary transthyretin-mediated amyloidosis.

Action ⬆ ⬇

Acts as an antisense oligonucleotide that binds to and causes degradation of mutant and wild-type human transthyretin (TTR) mRNA, which subsequently results in a reduction of serum TTR protein and TTR protein deposits in tissues.

Therapeutic effects:

Reduction in impairment from neuropathy and improved quality of life.

Pharmacokinetics ⬆ ⬇

Absorption: Rapidly absorbed following SUBQ administration.

Distribution: Extensively distributed to tissues.

Protein Binding: >98%.

Metabolism/Excretion: Metabolized by nucleases to nucleotides of various lengths; primarily excreted in urine (<1% as unchanged drug).

Half-Life: 21 days.

Time/Action Profile ⬆ ⬇

(plasma concentrations)

ROUTE	ONSET	PEAK	DURATION
SUBQ	unknown	2 hr	unknown

Contraind./Precautions ⬆ ⬇

Contraindicated in:

None.

Use Cautiously in:

Severe renal impairment or end-stage renal disease;
Moderate or severe hepatic impairment;
OB: Safety not established in pregnancy;
Lactation: Use while breastfeeding only if potential maternal benefit justifies potential risk to infant;
Pedi: Safety and effectiveness not established in children.

Adv. Reactions/Side Effects ⬆ ⬇

CV: heart block

EENT: blurred vision, cataracts

GI: vomiting

GU: proteinuria

Local: injection site reactions

Misc: ↓vitamin A concentrations

Interactions ⬆ ⬇

Drug-drug:

None reported.

Route/Dosage ⬆ ⬇

SC (Adults ): 45 mg once monthly.

Availability ⬆ ⬇

Solution for injection (autoinjectors): 45 mg/0.8 mL

Assessment ⬆ ⬇

Monitor impairment from neuropathy at baseline and periodically during therapy.

Lab Test Considerations:

Causes ↓ serum vitamin A. Supplement no higher than with recommended daily allowance of vitamin A. If ocular symptoms of vitamin A deficiency occur, refer to ophthalmologist.

Implementation ⬆ ⬇

SC: Bring single-dose autoinjector to room temperature for 30 min prior to injection. Solution is colorless to yellow; do not use if cloudy, discolored, or contains particulates. Inject SUBQ into abdomen or upper thigh (or upper arm if administered by health care professional).
- Protect from light and refrigerate. May keep at room temperature ≤6 wk.

Patient/Family Teaching ⬆ ⬇

Explain purpose and side effects of medication. Advise patient to read Patient Information before starting therapy.
- Instruct patient on appropriate administration technique and disposal of equipment if self-administration is appropriate.
- If a dose is missed, administer as soon as possible and resume at monthly intervals from that date.
Advise patient to notify health care professional if vomiting, blurred vision, dry eyes, night blindness, or injection site reaction (erythema, pain, itching) occurs.
Advise patient to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult health care professional before taking other medications.
Rep: Advise women of reproductive potential to notify health care professional if pregnancy is planned or suspected or if breastfeeding.

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Pronunciation ⬇

Classifications ⬆ ⬇