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Placebo/rhASB
rhASB (recombinant human arylsulfatase B) is an enzyme replacement therapy that replaces deficient arylsulfatase B to break down glycosaminoglycans accumulated in Maroteaux-Lamy syndrome.
rhASB (recombinant human arylsulfatase B) is an enzyme replacement therapy that replaces deficient arylsulfatase B to break down glycosaminoglycans accumulated in Maroteaux-Lamy syndrome. Used for Maroteaux-Lamy syndrome (mucopolysaccharidosis VI).
At a glance
| Generic name | Placebo/rhASB |
|---|---|
| Sponsor | BioMarin Pharmaceutical |
| Drug class | Enzyme replacement therapy |
| Target | Arylsulfatase B (ARSB) |
| Modality | Small molecule |
| Therapeutic area | Rare genetic disease / Lysosomal storage disorder |
| Phase | Phase 3 |
Mechanism of action
Maroteaux-Lamy syndrome (mucopolysaccharidosis VI) is caused by deficiency of the enzyme arylsulfatase B, leading to accumulation of dermatan sulfate in tissues. rhASB is a recombinant form of this enzyme that catalyzes the breakdown of accumulated glycosaminoglycans, reducing tissue burden and improving organ function. The enzyme is administered intravenously to reach systemic circulation and target affected tissues.
Approved indications
- Maroteaux-Lamy syndrome (mucopolysaccharidosis VI)
Common side effects
- Infusion-related reactions
- Antibody formation
- Pyrexia
- Headache
Key clinical trials
- Study of rhASB in Patients With Mucopolysaccharidosis VI (PHASE3)
- Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI (PHASE3)
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 |