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eplontersen (Wainua)
Indications:
- hereditary transthyretin amyloidosis
- treatment of associated polyneuropathy [1,2] (FDA-approved)
Dosage:
- 45 mg sq every 4 weeks
Adverse effects:
- thrombocytopenia
- glomerulonephritis
- flu-like symptoms
- abnormal liver function
- ocular events potentially related to vitamin A deficiency*
* transthyretin is a transporter of vitamin A-retinol binding complexes
Laboratory:
- platelet count
- renal function test
- urinalysis with reflex urine microscopy
Mechanism of action:
- antisense oligonucleotide conjugated to GalNAc for enhanced hepatocyte uptake,
- inhibits hepatic synthesis of transthyretin, the main source of transthyretin
General
short interfering double-stranded RNA; antisense oligonucleotide (siRNA, microRNA, miRNA)
metabolic agent (metabolic modifier)
References
- Coelho T, Marques Jr W, Dasgupta NR et al
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.
JAMA. Published online September 28, 2023.
PMID: 37768671
https://jamanetwork.com/journals/jama/fullarticle/2810248
- George J
FDA Approves New Agent for Nerve Pain From Rare Inherited Disease.
Eplontersen gets a nod for polyneuropathy from hereditary transthyretin-mediated
amyloidosis.
MedPage Today December 22, 2023
https://www.medpagetoday.com/neurology/generalneurology/108020