Proposed mechanism
of action

MEPSEVII is a recombinant form of human β-glucuronidase (rhGUS)

  • Intended to provide exogenous GUS enzyme for uptake into cellular lysosomes via mannose-6 phosphate residues
  • Helps catabolize the GAGs that accumulate in the tissues of patients with MPS VII

Glycosaminoglycans (also called GAGs) are complex molecules contained within a certain part of the cell called lysosomes.

GAGs accumulate in cells in systems throughout the body, including:

• Bones
• Tissues
• Organs

In all patients evaluated, MEPSEVII treatment resulted in reduction of urinary excretion of GAGs including chondroitin sulfate and dermatan sulfate, which was sustained with continued treatment