Which medications in the drug class RNAi Agents are used in the treatment of Acute Intermittent Porphyria?

Updated: Jul 08, 2020
  • Author: Thomas G DeLoughery, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

RNAi Agents

Givosiran (Givlaari)

Small interfering RNA agents. Via RNA interference, they lead to degradation of aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes, which in turn lowers elevated liver ALAS1 mRNA levels. This decreases circulating levels of the neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), both of which are linked to attacks and other manifestations of the acute hepatic porphyrias (AHP).


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