Galsulfase is a recombinant human enzyme used as replacement enzyme therapy for the treatment of of adults and children with Mucopolysaccharidosis VI, a rare genetic disorder caused by a deficiency of a lysosomal enzyme. Galsufase is a variant form of the polymorphic human enzyme N-acetylgalactosamine 4-sulfatase of recombinant DNA origin.
- Mechanism
- Curator reviewed
Galsulfase supplies recombinant-engineered galsulfase, a normal variant form of the polymorphic human enzyme, N-acetylgalactosamine 4-sulfatase. It is a lysosomal hydrolase that catalyzes the cleavage of the sulfate ester from terminal N-acetylgalactosamine 4-sulfate residues of GAG chondroitin 4-sulfate and dermatan sulfate. Increased catabolism of GAG in turn reduces systemic dermatan sulfate accumulation, thereby reducing the primary symptoms of MPS VI.
- Primary indication
- For the treatment of adults and children with Mucopolysaccharidosis VI.Curator reviewed · 1 structured indication
- Formula / weight
- C2534H3851N691O719S16 · 56012.6 Da
- First approval
- Canada, 2013 · United States, 2005 · European Union, 2016
- Also known as
- Arylsulfatase B galsulfase · Galsulfase (genetical recombination) · N-acetylgalactosamine-4-sulfatase, recombinant human · rhASB
- Brand names
Resolves to
What you can answer from here — as of July 17, 2026
Which drugs share a target with Galsulfase, and which of those have an active Phase 3 trial?
Create a free account or log in to explore the full data card.
Create Account