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N-acetylgalactosamine 4-sulfatase
N-acetylgalactosamine 4-sulfatase is a Enzyme replacement therapy Small molecule drug developed by BioMarin Pharmaceutical. It is currently in Phase 3 development for Morquio A syndrome (mucopolysaccharidosis IVA). Also known as: rhASB.
N-acetylgalactosamine 4-sulfatase is an enzyme replacement therapy that replaces the deficient GALNS enzyme to break down keratan sulfate and chondroitin 6-sulfate in lysosomal storage disease.
N-acetylgalactosamine 4-sulfatase is an enzyme replacement therapy that replaces the deficient GALNS enzyme to break down keratan sulfate and chondroitin 6-sulfate in lysosomal storage disease. Used for Morquio A syndrome (mucopolysaccharidosis IVA).
At a glance
| Generic name | N-acetylgalactosamine 4-sulfatase |
|---|---|
| Also known as | rhASB |
| Sponsor | BioMarin Pharmaceutical |
| Drug class | Enzyme replacement therapy |
| Target | GALNS (N-acetylgalactosamine 4-sulfatase) |
| Modality | Small molecule |
| Therapeutic area | Rare genetic disease / Lysosomal storage disorder |
| Phase | Phase 3 |
Mechanism of action
The drug is a recombinant human arylsulfatase B enzyme that catalyzes the degradation of glycosaminoglycans (keratan sulfate and chondroitin 6-sulfate) that accumulate in Morquio A syndrome due to GALNS enzyme deficiency. By restoring enzymatic activity, it reduces pathological accumulation of these substrates in tissues and organs, slowing disease progression.
Approved indications
- Morquio A syndrome (mucopolysaccharidosis IVA)
Common side effects
- Infusion-related reactions
- Fever
- Chills
- Headache
- Nausea
Key clinical trials
- Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
- A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI (PHASE4)
- Study of rhASB in Patients With Mucopolysaccharidosis VI (PHASE3)
- Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI (PHASE3)
- Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI (PHASE2)
- Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- N-acetylgalactosamine 4-sulfatase CI brief — competitive landscape report
- N-acetylgalactosamine 4-sulfatase updates RSS · CI watch RSS
- BioMarin Pharmaceutical portfolio CI
Frequently asked questions about N-acetylgalactosamine 4-sulfatase
What is N-acetylgalactosamine 4-sulfatase?
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Who makes N-acetylgalactosamine 4-sulfatase?
Is N-acetylgalactosamine 4-sulfatase also known as anything else?
What drug class is N-acetylgalactosamine 4-sulfatase in?
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What does N-acetylgalactosamine 4-sulfatase target?
Related
- Drug class: All Enzyme replacement therapy drugs
- Target: All drugs targeting GALNS (N-acetylgalactosamine 4-sulfatase)
- Manufacturer: BioMarin Pharmaceutical — full pipeline
- Therapeutic area: All drugs in Rare genetic disease / Lysosomal storage disorder
- Indication: Drugs for Morquio A syndrome (mucopolysaccharidosis IVA)
- Also known as: rhASB
- Compare: N-acetylgalactosamine 4-sulfatase vs similar drugs
- Pricing: N-acetylgalactosamine 4-sulfatase cost, discount & access