N-acetylgalactosamine-6-sulfatase Inhibitors
1 drugsAbout N-acetylgalactosamine-6-sulfatase
N-acetylgalactosamine-6-sulfatase (GALNS) is a lysosomal enzyme that breaks down glycosaminoglycans (GAGs) by removing sulfate groups from N-acetylgalactosamine-6-sulfate residues in keratan sulfate and chondroitin-6-sulfate.
GALNS deficiency leads to GAG accumulation and health problems, motivating therapeutic development. Currently, there is no genetic evidence data available for this target.
GALNS is targeted by one approved biologic drug, VIMIZIM (BIOMARIN PHARM), indicated for two conditions. BIOMARIN PHARM is the only company with an approved drug.
Top N-acetylgalactosamine-6-sulfatase Drugs
BIOMARIN PHARM is the only company with an approved GALNS-targeting drug.
High market concentration suggests significant entry barriers or limited commercial opportunity.
N-acetylgalactosamine-6-sulfatase Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets N-acetylgalactosamine-6-sulfatase, using enzyme modality.
Explore small molecule or gene therapy approaches to diversify the therapeutic options.
N-acetylgalactosamine-6-sulfatase Drug Approval Timeline (2014 - 2014)
The first and only drug, VIMIZIM, was approved in 2014.
The approval timeline indicates a potentially unsaturated market with room for innovation.
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Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 1 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 1-drug timeline
- • First-of-modality markers
Full summary • All drugs • Genetic evidence • Trials • Timeline