recombinant human porphobilinogen deaminase

Recombinant human porphobilinogen deaminase (rhPBGD) is a biologic enzyme replacement therapy designed to treat acute intermittent porphyria (AIP), a rare metabolic disorder caused by deficiency of the enzyme porphobilinogen deaminase in the heme biosynthesis pathway. The drug acts by supplementing or replacing deficient endogenous PBGD activity in hepatocytes, thereby reducing toxic accumulation of heme precursors such as aminolevulinic acid and porphobilinogen. Preclinical studies and early clinical trials have shown that intravenous administration of rhPBGD can increase hepatic enzymatic activity and lower plasma concentrations of harmful metabolites in both animal models and humans with AIP. The drug has been developed primarily for acute intermittent porphyria but may have potential for other hepatic enzymopenic disorders[2][3][5][8].

Brand names

PorphogenPorphozym

Other names

porphobilinogen deaminase, human recombinantPBGD, HemeBiotechPBGD, Zymenexrecombinant porphobilinogen deaminase, HemeBiotechrecombinant porphobilinogen deaminase, Zymenex