eplontersen (Wainua)
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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
Mechanism of action
- antisense oligonucleotide conjugated to GalNAc for enhanced hepatocyte uptake,
- inhibits hepatic synthesis of transthyretin, the main source of transthyretin
More general terms
- short interfering double-stranded RNA; antisense oligonucleotide (siRNA, microRNA, miRNA)
- metabolic agent (metabolic modifier)
References
- ↑ Jump up to: 1.0 1.1 Coelho T, Marques Jr W, Dasgupta NR et al Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy. JAMA. Published online September 28, 2023. PMID: https://www.ncbi.nlm.nih.gov/pubmed/37768671 https://jamanetwork.com/journals/jama/fullarticle/2810248
- ↑ Jump up to: 2.0 2.1 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