CLINICAL TRIALS PROFILE FOR NAGLAZYME

All Clinical Trials for NAGLAZYME

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00299000 ↗	A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI	Completed	BioMarin Pharmaceutical	Phase 4	2006-05-01	The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
NCT02156674 ↗	Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome	Terminated	BioMarin Pharmaceutical	N/A	2016-01-26	This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.
NCT02156674 ↗	Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome	Terminated	Masonic Cancer Center, University of Minnesota	N/A	2016-01-26	This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.

Clinical Trial Conditions for NAGLAZYME

Condition Name

Condition Name for NAGLAZYME
Intervention	Trials
Maroteaux-Lamy Syndrome	2
Mucopolysaccharidosis VI	1

Condition MeSH

Condition MeSH for NAGLAZYME
Intervention	Trials
Syndrome	2
Mucopolysaccharidosis VI	2
Mucopolysaccharidoses	1

Clinical Trial Locations for NAGLAZYME

Trials by Country

Trials by Country for NAGLAZYME
Location	Trials
United States	2
Portugal	1
France	1

Trials by US State

Trials by US State for NAGLAZYME
Location	Trials
Minnesota	1
California	1

Clinical Trial Progress for NAGLAZYME

Clinical Trial Phase

Clinical Trial Phase for NAGLAZYME
Clinical Trial Phase	Trials
Phase 4	1
N/A	1

Clinical Trial Status

Clinical Trial Status for NAGLAZYME
Clinical Trial Phase	Trials
Completed	1
Terminated	1

Clinical Trial Sponsors for NAGLAZYME

Sponsor Name

Sponsor Name for NAGLAZYME
Sponsor	Trials
BioMarin Pharmaceutical	2
Masonic Cancer Center, University of Minnesota	1

Sponsor Type

Sponsor Type for NAGLAZYME
Sponsor	Trials
Industry	2
Other	1

Clinical Trials Update, Market Analysis, and Projection for NAGLAZYME (Imiglucerase)

Last updated: October 30, 2025

Overview of NAGLAZYME

NAGLAZYME (imiglucerase) is an enzyme replacement therapy (ERT) manufactured by Sanofi, designed to treat Gaucher disease type 1. Gaucher disease is a rare, inherited lysosomal storage disorder caused by a deficiency in the glucocerebrosidase enzyme, leading to the accumulation of glucocerebroside in macrophages and subsequent multi-organ damage. Approved by the FDA in 1994, NAGLAZYME remains a cornerstone in Gaucher disease management.

Current Status in Clinical Trials

While NAGLAZYME currently holds FDA approval as a standard-of-care therapy, ongoing clinical investigations focus on expanding its indications and optimizing delivery methods.

Ongoing Clinical Trials

1. Long-term Safety and Efficacy in Pediatric Patients

   • Trial: A Phase IV, open-label, observational study ongoing since 2015 assesses the safety and efficacy of imiglucerase in children with Gaucher disease.
   • Objective: To evaluate long-term outcomes, including growth parameters, hematological improvements, and safety profiles.
   • Status: Data collection ongoing; preliminary results suggest sustained efficacy with a favorable safety profile in pediatric cohorts.

2. Subcutaneous Delivery Approaches

   • Trial: Phase I/II studies exploring modified formulations allowing for subcutaneous administration.
   • Objective: To improve patient convenience and adherence compared to intravenous infusions.
   • Status: Early-stage; results anticipated within 2024.

3. Gene Therapy Synergy

   • Trial: Experimental combination studies investigating gene therapy adjuncts to enzyme therapy.
   • Objective: To assess whether gene editing approaches, such as CRISPR/Cas9, can replace or augment NAGLAZYME’s function.
   • Status: Preclinical; no human trials registered.

Regulatory Advances and Future Clinical Plans

Sanofi remains committed to lifecycle management, with applications for new formulations and indications submitted to regulatory agencies. The company is also exploring biosimilars, although none have yet entered advanced clinical stages.

Market Analysis of NAGLAZYME

Market Size and Growth Drivers

Gaucher disease’s estimated prevalence: approximately 1 in 50,000 to 100,000 live births globally, with higher incidence in Ashkenazi Jewish populations (~1 in 855). Despite its rarity, NAGLAZYME has dominated the therapeutic landscape for decades, with sales driven by:

• Increasing awareness and diagnosis of Gaucher disease.
• Expanding indications and off-label uses.
• Advancements in infusion protocols improving patient quality of life.

The global lysosomal storage disorder treatment market, estimated at US$1.3 billion in 2022, is expected to grow at a CAGR of 6-8% through 2030, driven by the increasing adoption of enzyme replacement therapies and emerging gene-editing modalities.

Competitive Landscape

While NAGLAZYME maintains a near-monopoly, competition increasingly emerges from:

• Cerezyme (Imiglucerase): A biosimilar approved in various markets.
• Vpriv (Vatercept): An alternative ERT with differing administration protocols.
• Emerging Gene Therapies: Companies like Sangamo and Beam Therapeutics exploring curative options.

Pricing and Reimbursement

NAGLAZYME’s annual treatment cost ranges from US$250,000 to $300,000 per patient, depending on dosage and regional pricing strategies. Reimbursement remains robust in most developed markets, supported by well-established clinical efficacy data.

Market Challenges

• High Cost: Limits access in developing regions.
• Administration Burden: Intravenous infusions multiple times per month reduce patient compliance.
• Emerging Therapies: Long-term safety and efficacy of gene therapies threaten market share.

Market Projection

Short-to-Medium Term (2023–2028)

Given the current clinical pipeline and market dynamics, NAGLAZYME is projected to maintain a dominant position in Gaucher disease management through incremental growth. The global market could see a CAGR of 4-6%, driven by:

• Increased diagnosis rates.
• Expansion into orphan drug markets in emerging economies.
• Adoption of improved infusion protocols reducing treatment burden.

Long-Term Outlook (2028–2033)

Innovations in gene therapy and alternative enzyme formulations, coupled with a potential shift towards curative options, threaten NAGLAZYME’s market share. However, its established safety profile and clinical data are likely to sustain a significant niche, especially as new therapies undergo lengthy approval processes.

Strategic Opportunities

Sanofi’s efforts to develop subcutaneous formulations and explore combination therapies position NAGLAZYME to adapt to evolving market needs. Strategic collaborations and investment in gene therapy pipelines could further cement its role or facilitate transitions to more definitive treatments.

Key Challenges

• Patent expirations and biosimilar entrants could reduce pricing power.
• Regulatory hurdles in approving new formulations and indications.
• Market acceptance of novel curative treatments.

Key Takeaways

• NAGLAZYME remains a cornerstone in Gaucher disease treatment, with consistent clinical evidence supporting its safety and efficacy.
• Ongoing clinical trials aim to improve patient experience and explore adjunct therapies, although none threaten its current market dominance imminently.
• The global enzyme replacement therapy market is growing steadily, but emerging gene therapies pose long-term disruption risks.
• Pricing pressures and the high cost of treatment affect accessibility, especially in resource-limited regions.
• Strategic innovation and pipeline development are essential for maintaining NAGLAZYME’s market relevance amid evolving therapeutic landscapes.

FAQs

1. Are there any new formulations of NAGLAZYME in development?
   Yes. Sanofi is exploring subcutaneous delivery options to improve patient compliance and reduce infusion-related discomfort. Early-phase trials are ongoing, with results expected within the next two years.

2. What is the impact of biosimilar imiglucerase products on NAGLAZYME’s market share?
   Biosimilars have begun entering various markets, offering lower-cost alternatives. While they can erode pricing power, NAGLAZYME’s established clinical data and brand recognition help sustain its market position.

3. How do gene therapies compare to enzyme replacement therapy like NAGLAZYME?
   Gene therapies aim for a potential cure by correcting the underlying genetic defect. They may offer long-term benefits and reduce treatment burden, but are currently in preclinical or early clinical phases, facing regulatory and safety hurdles.

4. What are the key considerations for clinicians prescribing NAGLAZYME?
   Clinicians should monitor for infusion reactions, antibody development, and long-term efficacy, especially in pediatric patients. Treatment protocols are individualized based on disease severity and patient response.

5. Is NAGLAZYME approved for indications beyond Gaucher disease type 1?
   Currently, NAGLAZYME is approved primarily for Gaucher disease type 1. Off-label or investigational use in other lysosomal storage disorders is under evaluation but lacks regulatory approval at present.

References

1. [1] FDA Approval Documents for NAGLAZYME, 1994.
2. [2] Global Gaucher Disease Market Report, 2022.
3. [3] ClinicalTrials.gov Data on NAGLAZYME Trials.
4. [4] Sanofi Annual Reports and Pipeline Updates.
5. [5] Market analysys reports by GlobalData, 2022.

This comprehensive analysis aims to support strategic decision-making regarding NAGLAZYME’s clinical development, market positioning, and future opportunities.