Givlaari is a subcutaneously administered ribonucleic acid interference (RNAi) agent that inhibits delta-aminolevulinate synthase 1 (ALAS1) synthesis, a key regulator of the heme biosynthetic pathway in the liver. Givlaari is believed to reduce ALAS1 levels, thereby decreasing delta-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Decreasing ALA and PBG levels to near normal levels is believed to reduce the occurrence of acute attacks in patients with acute hepatic porphyria.
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