Acute Intermittent Porphyria Medication

Updated: Jan 13, 2023
  • Author: Thomas G DeLoughery, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Medication Summary

The goals of pharmacotherapy are to reduce morbidity and to prevent complications. Intravenous hematin is the treatment of choice, both for treatment of severe acute attacks and for prevention of recurrent attacks. [14]  Givosiran, which is given monthly by subcutaneous injection, is a newer treatment option. [21]


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).


Blood Components

Hemin (Panhematin)

Provides negative feedback to the heme synthetic pathway and shuts down productions of porphyrins and porphyrin precursors.