Skip to content
PeptidesEncyclopedia
Informational, not medical advice

This is an approved / prescription medicine. Do not use without a prescription and medical supervision.

EMA-approvedaka Vimizim

Elosulfase alfa — Complete Research Guide (2026)

Last updated 2026-06-30

TL;DR

Elosulfase alfa (also known as Vimizim) is a peptide catalogued under Enzymes (MPS IVA (Morquio)). Neutral reference entry; EU status: EU-approved prescription medicine.

What is Elosulfase alfa?

Elosulfase alfa (brand name Vimizim) is a recombinant human N-acetylgalactosamine-6-sulfatase (GALNS) given by intravenous infusion as enzyme replacement therapy for mucopolysaccharidosis type IVA (Morquio A syndrome).

It is an approved orphan drug (FDA 2014, EMA 2014) used under specialist supervision, not a research or wellness peptide.

Its evidence base includes the pivotal MOR-004 randomized, double-blind, placebo-controlled phase 3 trial plus multiple open-label extension studies.

How does Elosulfase alfa work?

Elosulfase alfa provides a functional copy of GALNS, the enzyme deficient in Morquio A, which is needed to break down the glycosaminoglycans keratan sulfate and chondroitin-6-sulfate.

Replacing this activity is intended to reduce lysosomal glycosaminoglycan accumulation that damages bone, cartilage, and connective tissue.

What does the research say about Elosulfase alfa?

  • In the pivotal MOR-004 phase 3 randomized controlled trial, weekly elosulfase alfa significantly improved 6-minute walk distance versus placebo at 24 weeks. [1]
  • Multi-domain analysis of the pivotal trial showed benefits extending across endurance and functional measures rather than a single endpoint. [2]
  • Long-term open-label treatment sustained endurance improvements beyond the placebo-controlled period. [3]

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] Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study — Hendriksz CJ et al., Journal of Inherited Metabolic Disease 2014. (Randomized, double-blind, placebo-controlled phase 3 trial (MOR-004))
  • [2] Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial — Hendriksz CJ et al., Molecular Genetics and Metabolism 2015. (Analysis of pivotal phase 3 trial)
  • [3] Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome — Hendriksz CJ et al., Molecular Genetics and Metabolism 2016. (Long-term open-label extension)
  • [4] Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome — Hendriksz CJ et al., Journal of Inherited Metabolic Disease 2016. (Long-term open-label extension)
  • [5] Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study — Hendriksz CJ et al., Molecular Genetics and Metabolism 2018. (Open-label phase 3 extension)
  • [6] Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study — Harmatz PR et al., American Journal of Medical Genetics Part A 2017. (Open-label phase 2 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 2 mg/kg administered by intravenous infusion once weekly.

This is background context only; actual dosing, infusion duration, and premedication are set by a treating metabolic specialist.

Side effects & safety profile

Infusion-associated reactions are common, including reactions during or after the infusion, and are managed with premedication and adjusting the infusion rate.

Anaphylaxis and other serious hypersensitivity reactions have been reported, so infusions are administered with appropriate medical support available.

Anti-drug antibodies develop in most treated patients and are monitored, and enzyme replacement does not correct all skeletal and systemic features of Morquio A.

Stacking & combinations

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

Finding Elosulfase alfa vendors

Finding Elosulfase alfa vendors

This is an independent, neutral directory — we sell nothing and route no purchases. Use it to read regulatory context and (where available) genuine, moderated vendor information for your country.

Choose your country

We never list placeholder vendors. Pick your country to see neutral regulatory context.

Browse the directory

User reviews

No reviews yet

Reviews appear here once submitted and moderated. An aggregate rating is shown only after at least 5 genuine reviews — we never fabricate ratings.

Sign in to review

Frequently asked questions

It is an approved enzyme replacement therapy for mucopolysaccharidosis type IVA (Morquio A syndrome), a rare lysosomal storage disorder.

References

  1. [1] Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study — Hendriksz CJ et al., Journal of Inherited Metabolic Disease 2014. PMID: 24810369. View sourceStudy: Randomized, double-blind, placebo-controlled phase 3 trial (MOR-004)Weekly elosulfase alfa improved 6-minute walk distance versus placebo at 24 weeks in Morquio A.
  2. [2] Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial — Hendriksz CJ et al., Molecular Genetics and Metabolism 2015. PMID: 25284089. View sourceStudy: Analysis of pivotal phase 3 trialTreatment benefits spanned multiple functional domains beyond the primary endurance endpoint.
  3. [3] Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome — Hendriksz CJ et al., Molecular Genetics and Metabolism 2016. PMID: 27380995. View sourceStudy: Long-term open-label extensionEndurance improvements were sustained with continued weekly treatment over the longer term.
  4. [4] Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome — Hendriksz CJ et al., Journal of Inherited Metabolic Disease 2016. PMID: 27553181. View sourceStudy: Long-term open-label extensionLong-term treatment was associated with stabilization or improvement in respiratory function measures.
  5. [5] Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study — Hendriksz CJ et al., Molecular Genetics and Metabolism 2018. PMID: 29248359. View sourceStudy: Open-label phase 3 extensionContinued treatment was associated with maintained or improved activities of daily living.
  6. [6] Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study — Harmatz PR et al., American Journal of Medical Genetics Part A 2017. PMID: 27774754. View sourceStudy: Open-label phase 2 studyElosulfase alfa showed functional effects in patients with limited ambulation not captured by walk-distance endpoints.
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.