pegtarviliase

Pegtarviliase is a novel recombinant human enzyme engineered to reduce elevated levels of total homocysteine in plasma by degrading the amino acid homocysteine and its dimer. It is a PEGylated, engineered form of the human cystathionine gamma-lyase (CGL) enzyme, designed with high substrate specificity for both monomeric and dimeric forms of homocysteine but not for its native substrate, cystathionine. Pegtarviliase is being developed as an enzyme replacement therapy primarily for classical homocystinuria, a rare inherited disorder of methionine metabolism that leads to toxic accumulation of homocysteine and associated complications such as lens dislocation, skeletal abnormalities, vascular issues, neurologic effects, and increased risk of early death. The drug has received Orphan Drug Designation in both the US and EU as well as Rare Pediatric Disease Designation in the US. Clinical trials have shown that pegtarviliase can lower total homocysteine levels; however, immunogenicity (anti-drug antibodies) may impact efficacy at higher doses[1][3][4][5][6].

Other names

pegtarviliase