Drug intelligence / Profile preview

alglucerase

Development stage
Phase 2
Lead developer
Sanofi
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration
Intravenous
01

Overview

Alglucerase is a mannose-terminated form of human placental beta-glucocerebrosidase used as an enzyme replacement therapy for Type I Gaucher disease. It was the first approved treatment for this condition and works by catalyzing the hydrolysis of glucocerebroside into glucose and ceramide, thereby reducing the accumulation of lipid-laden macrophages (Gaucher cells) in tissues such as liver, spleen, and bone marrow. Alglucerase is derived from human placenta tissue collected after birth and modified to expose mannose residues on its oligosaccharide chains to enhance uptake by macrophages. The drug does not cure Gaucher disease but helps control symptoms such as hepatosplenomegaly, anemia, thrombocytopenia, and bone disease. Alglucerase was approved in 1991 but has since been discontinued in favor of recombinant alternatives[1][2][3][4][5][7].

Brand names
Ceredase
Other names
beta-glucocerebrosidasehuman placental glucocerebrosidase
02

Targets

Sphingolipid (Sphingolipid metabolism)MRC1 (Mannose Receptor C-type 1)

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