N-acetylgalactosamine 4-sulfatase Inhibitors
1 drugsAbout N-acetylgalactosamine 4-sulfatase
N-acetylgalactosamine 4-sulfatase (GALNS) is a lysosomal enzyme that breaks down glycosaminoglycans (GAGs) like chondroitin sulfate and keratan sulfate by removing sulfate groups. This function is crucial for the cell's recycling process within lysosomes.
GALNS deficiency causes mucopolysaccharidosis type VI (MPS VI), a rare lysosomal storage disorder resulting from GAG accumulation. There is currently no genetic evidence readily available linking GALNS mutations directly to common diseases, but its role in MPS VI makes it a therapeutic target.
GALNS is targeted by one approved drug, NAGLAZYME, a biologic (other) enzyme replacement therapy developed by BIOMARIN and approved in 2005 for MPS VI.
Top N-acetylgalactosamine 4-sulfatase Drugs
BIOMARIN is the only company with an approved drug targeting GALNS.
High market concentration indicates potential entry barriers and limited competition.
N-acetylgalactosamine 4-sulfatase Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets N-acetylgalactosamine 4-sulfatase, using enzyme modality.
Consider novel modalities to differentiate from existing enzyme replacement therapy.
N-acetylgalactosamine 4-sulfatase Drug Approval Timeline (2005 - 2005)
NAGLAZYME was first approved in 2005 and remains the only approved drug.
The approval timeline suggests market saturation and limited innovation in this area.
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Approval Timeline
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