Inborn errors of metabolism:

Indications for TEGSEDI:

Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.


Give as SC inj in abdomen, upper thigh, or outer area of upper arm; rotate inj sites. 284mg once weekly. Monitoring and treatment recommendations: see full labeling.


Not established.


Platelet count <100x109/L. History of acute glomerulonephritis due to Tegsedi.

Boxed Warning:

Thrombocytopenia. Glomerulonephritis.


Risk of thrombocytopenia, glomerulonephritis. Do not initiate if platelets <100x109/L or UPCR ≥1000mg/g; discontinue and/or give corticosteroids (based on platelet count) or immunosuppressants; see full labeling. Avoid Tegsedi therapy if glucocorticoids or immunosuppressants are not advised. Monitor platelets, serum creatinine, eGFR, urine protein to creatinine ratio (UPCR), and urinalysis prior to treatment, every 2 weeks during, and for 8 weeks after discontinuation. Perform repeat platelet count if EDTA-mediated platelet clumping is suspected. Withhold if UPCR ≥1000mg/g or eGFR <45mL/min/1.73m2 develops; may resume treatment once resolved. Permanently discontinue if acute glomerulonephritis is confirmed. Nephrotic syndrome. Monitor ALT/AST, and total bilirubin prior to treatment, every 4 months during, and for 8 weeks after discontinuation. Interrupt or discontinue dose if hepatic dysfunction develops. Risk of stroke and CNS arterial dissection. Discontinue if hypersensitivity reaction occurs. Give recommended Vit. A supplementation; refer for eye exam if symptoms of Vit. A deficiency (eg, night blindness). Moderate or severe hepatic impairment, severe renal impairment or ESRD: not studied. Elderly. Pregnancy. Nursing mothers.

Pharmacologic Class:

Antisense oligonucleotide.


Thrombocytopenia risk with concomitant antiplatelets (eg, adenosine, clopidogrel, prasugrel, ticagrelor, ticlopidine, aspirin, NSAIDs) or anticoagulants (eg, heparin, warfarin); consider discontinuing these agents if platelets <50x109/L. Caution with concomitant nephrotoxic drugs or drugs that may impair renal function.

Adverse Reactions:

Inj site reactions, nausea, headache, fatigue, thrombocytopenia, fever; glomerulonephritis, renal toxicity, inflammatory/immune effects, hepatic effects, hypersensitivity, reduced serum Vit. A.



Generic Availability:


How Supplied:

Single-dose prefilled syringes—1, 4