elosulfase alfa   Click here for help

GtoPdb Ligand ID: 7392

Synonyms: BMN 110 | rhGALNS | Vimizim®
Approved drug
elosulfase alfa is an approved drug (EMA & FDA (2014))
Comment: Elosulfase alfa is a recombinant enzyme replacement therapy for galactosamine (N-acetyl)-6-sulfatase (GALNS, P34059) deficiency. The peptide is translated from the human cDNA expressed in CHO cells [1].
No information available.
Summary of Clinical Use Click here for help
Approved to treat mucopolysaccharidosis type IVA (Morquio A syndrome), a lysosomal storage disease characterised by an inability to break down keratin sulphate which causes spondylo-epiphyso-metaphyseal dysplasia and results in musculoskeletal abnormalities and growth arrest. Sufferers may also experience nervous complications, respiratory problems, hepatomegaly, valvulopathies, hearing loss and corneal clouding.
Mechanism Of Action and Pharmacodynamic Effects Click here for help
This drug replaces the enzyme deficient in Morquio A syndrome patients, restoring lysosomal catabolism of glycosaminoglycans such as keratin sulphate [1].