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EMA-approvedaka Naglazyme

Galsulfase — Complete Research Guide (2026)

Last updated 2026-06-30

TL;DR

Galsulfase (also known as Naglazyme) is a peptide catalogued under Enzymes (MPS VI). Neutral reference entry; EU status: EU-approved prescription medicine.

What is Galsulfase?

Galsulfase (brand name Naglazyme) is a recombinant human N-acetylgalactosamine-4-sulfatase (arylsulfatase B) given by intravenous infusion as enzyme replacement therapy for mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome).

It is an approved orphan drug (FDA 2005, EMA 2006) used under medical supervision, not a research chemical or peptide supplement.

It is supported by a pivotal randomized, double-blind, placebo-controlled phase 3 trial and by long-term follow-up and infant-treatment data.

How does Galsulfase work?

Galsulfase supplies a functional copy of arylsulfatase B (N-acetylgalactosamine-4-sulfatase), the enzyme deficient in MPS VI, which is required to degrade the glycosaminoglycan dermatan sulfate.

Restoring this activity reduces lysosomal dermatan sulfate accumulation that causes the progressive multisystem damage of the disease.

What does the research say about Galsulfase?

  • In the pivotal 24-week phase 3 randomized controlled trial, galsulfase improved 12-minute walk distance versus placebo in patients with MPS VI. [1]
  • Early initiation of enzyme replacement therapy in infancy or childhood was associated with better long-term clinical outcomes in a resurvey study. [5]
  • Ten-year follow-up of previously surveyed patients showed sustained functional effects with continued galsulfase treatment. [4]

Clinical research & studies

The references below are the primary sources cited throughout this guide. Each links directly to PubMed or the regulator. Where evidence is preclinical (animal or in-vitro), that is stated rather than implied.

  • [1] Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study — Harmatz P et al., The Journal of Pediatrics 2006. (Randomized, double-blind, placebo-controlled phase 3 trial with open-label extension)
  • [2] Galsulfase (Naglazyme) therapy in infants with mucopolysaccharidosis VI — Harmatz PR et al., Journal of Inherited Metabolic Disease 2014. (Clinical study in infants)
  • [3] Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme) therapy — Braunlin E et al., Journal of Inherited Metabolic Disease 2013. (Long-term observational analysis)
  • [4] Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) - 10-year follow-up of patients who previously participated in an MPS VI Survey Study — Giugliani R et al., American Journal of Medical Genetics Part A 2014. (10-year follow-up / natural history)
  • [5] Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study — Horovitz DDG et al., Molecular Genetics and Metabolism 2021. (Resurvey / long-term observational study)
  • [6] Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy — Garcia P et al., Molecular Genetics and Metabolism 2021. (Long-term observational study)

Dosing context

This is not medical advice or a usage recommendation. Dosing figures are reported research context only, cited from the published literature.

In the trials and labeling, the studied regimen is 1 mg/kg administered by intravenous infusion once weekly.

This is context only; the actual infusion rate, premedication, and dosing are directed by a treating metabolic specialist.

Side effects & safety profile

Infusion-associated reactions are common and are managed with premedication, slowing the infusion, and monitoring.

Serious hypersensitivity reactions including anaphylaxis have been reported, so infusions are given where emergency support is available.

Anti-drug antibodies commonly develop and are monitored, and enzyme replacement does not reverse established skeletal, cardiac, or ocular damage.

Stacking & combinations

It is a lifelong single-enzyme replacement therapy for a specific deficiency and is not intended to be combined or stacked with other peptides.

Finding Galsulfase vendors

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Frequently asked questions

It is an approved enzyme replacement therapy for mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome), a rare lysosomal storage disorder.

References

  1. [1] Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study — Harmatz P et al., The Journal of Pediatrics 2006. PMID: 16647419. View sourceStudy: Randomized, double-blind, placebo-controlled phase 3 trial with open-label extensionGalsulfase improved 12-minute walk distance versus placebo and sustained gains during open-label extension.
  2. [2] Galsulfase (Naglazyme) therapy in infants with mucopolysaccharidosis VI — Harmatz PR et al., Journal of Inherited Metabolic Disease 2014. PMID: 24108527. View sourceStudy: Clinical study in infantsGalsulfase was used in infants with MPS VI with a manageable safety profile, supporting early treatment.
  3. [3] Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme) therapy — Braunlin E et al., Journal of Inherited Metabolic Disease 2013. PMID: 22669363. View sourceStudy: Long-term observational analysisLong-term galsulfase treatment was associated with effects on cardiac disease progression in MPS VI.
  4. [4] Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) - 10-year follow-up of patients who previously participated in an MPS VI Survey Study — Giugliani R et al., American Journal of Medical Genetics Part A 2014. PMID: 24764221. View sourceStudy: 10-year follow-up / natural historyExtended follow-up showed sustained clinical effects of galsulfase relative to untreated natural history.
  5. [5] Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study — Horovitz DDG et al., Molecular Genetics and Metabolism 2021. PMID: 33678523. View sourceStudy: Resurvey / long-term observational studyEarlier initiation of galsulfase was associated with better long-term outcomes in MPS VI patients.
  6. [6] Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy — Garcia P et al., Molecular Genetics and Metabolism 2021. PMID: 33775523. View sourceStudy: Long-term observational studyPatients treated from infancy showed favorable long-term outcomes on continued galsulfase therapy.
This article is for educational and research purposes only. Peptides discussed here are not approved for human consumption by the FDA, EMA, or equivalent regulators outside of specific clinical contexts. Always consult a licensed medical professional before any therapeutic use.