Lysodase

Lysodase (PEG-glucocerebrosidase) is a pegylated enzyme replacement therapy (ERT) that was developed for the treatment of Gaucher disease, a lysosomal storage disorder. Gaucher disease is caused by a genetic deficiency of the enzyme glucocerebrosidase, which leads to the pathological accumulation of glucocerebroside in the lysosomes of macrophages. Lysodase consists of the glucocerebrosidase enzyme covalently attached to polyethylene glycol (PEG) molecules. This pegylation was intended to extend the enzyme's circulatory half-life, potentially reducing the required dosage and frequency of administration compared to non-pegylated therapies. The drug was primarily investigated in Phase I/II clinical trials sponsored by the National Institute of Mental Health (NIMH) for patients with Type 1 and Type 3 Gaucher disease, particularly those with neurological involvement. Development of the drug was ultimately discontinued by its developer, Enzon.

Brand names

Lysodase

Other names

PEG-glucocerebrosidasepolyethyleneglycol-modified glucocerebrosidasepegylated glucocerebrosidase