eplontersen (Wainua)

Indications

• hereditary transthyretin amyloidosis
  • treatment of associated polyneuropathy (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

More general terms

• short interfering double-stranded RNA (siRNA, microRNA, miRNA)
• metabolic agent (metabolic modifier)