Chester Porphyria Medication

Updated: Aug 07, 2020
  • Author: Danielle Nance, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Medication

Blood Components

Hemin (Panhematin)

Limits rate of hepatic and/or marrow porphyria/heme biosynthesis, possibly by inhibiting the enzyme aminolevulinic acid synthetase. Indicated for amelioration of recurrent attacks of acute intermittent porphyria temporally related to the menstrual cycle in susceptible women.

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RNAi Agents

Givosiran (Givlaari)

Double-stranded small-interfering RNA that causes degradation of aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of acute hepatic porphyria.

Indicated for acute hepatic porphyria.

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