Name: Givosiran

Dosing & Uses


Pending FDA approval for treatment of acute hepatic porphyria (AHP)


Mechanism of Action

Ribonucleic acid interference (RNAi) therapeutic agent that targets the liver-expressed enzyme aminolevulinic acid synthase 1 (ALAS1)

In clinical trials, monthly SC injections have been shown to lower induced liver ALAS1 levels in a sustained manner, thereby decreasing neurotoxic heme intermediates, aminolevulinic acid (ALA), and porphobilinogen (PBG) towards normal levels

Patient Handout

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