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givosiran (Givlaari)

Indications: - acute hepatic porphyria Dosage: - monthly subcutaneous injections of givosiran 2.5 mg/kg Injection: 1 mL givosiran 189 mg/mL (single dose vial) Adverse effects: - injection site reactions - nausea - hepatoxicity (elevation in serum transaminases) - nephrotoxicity (renal failure) Drug interactions: - avoid concomitant use with CYP1A2 & CYP2D6 substrates Mechanism of action: - aminolevulinate synthase 1-directed siRNA

General

short interfering double-stranded RNA; antisense oligonucleotide (siRNA, microRNA, miRNA) metabolic agent (metabolic modifier)

References

  1. RxNorm
  2. FDA Drig Approvals. Nov 20, 2019 FDA approves givosiran for acute hepatic porphyria. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-givosiran-acute-hepatic-porphyria
  3. Highlights of Prescribing Information. GIVLAARI (givosiran) injection, for subcutaneous use https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/0212194s000lbl.pdf