CLINICAL TRIALS PROFILE FOR FABRAZYME

Biosimilar Clinical Trials for FABRAZYME

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT05843916 ↗	Switch Over Study of Biosimilar AGA for Fabry Disease	Recruiting	Bio Sidus SA	Phase 3	2022-12-13	BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and previously stabilized with Fabrazyme®.

All Clinical Trials for FABRAZYME

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00074971 ↗	A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease	Completed	Genzyme, a Sanofi Company	Phase 3	1999-10-01	People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid ("globatriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
NCT00140621 ↗	A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease	Completed	Genzyme, a Sanofi Company	Phase 4	2005-07-01	This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease. Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.
NCT00233870 ↗	A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.	Completed	Genzyme, a Sanofi Company		2004-06-01	The purpose of this survey is to identify any concerns regarding the following efficacy and safety-related issues in clinical practice with the new drugs "Fabrazyme for intravenous infusion 5mg" and "Fabrazyme for intravenous infusion 35mg" and to confirm the safety of these products in long-term use in the clinical setting. 1. New adverse drug reactions (ADRs) that cannot be predicted from the Precautions (in particular, clinically significant ADRs) 2. The incidence of ADRs under the actual conditions of use of the drug 3. Causal factors that might potentially affect safety 4. Efficacy evaluation in long-term use This survey will be conducted in accordance with the approval condition established for Fabrazyme: "To conduct a special surveillance of Efficacy and Safety in long term treatment and Pediatric with the drug."
NCT00312767 ↗	A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.	Withdrawn	Genzyme, a Sanofi Company	Phase 4	2006-04-01	People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid (globotriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study is designed to verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis in patients currently receiving Fabrazyme at a dose of 1.0 mg/kg every 2 weeks.
NCT00446862 ↗	The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study	Completed	University of Alabama at Birmingham		2007-03-01	The primary hypothesis is that titration of ACE inhibitor and Angiotensin Receptor Blockers (ARBs)to reduce urine protein excretion to < 500 mg per day in Fabry Patients receiving agalsidase beta therapy at 1 mg/kg every two weeks will slow the progression rate of decline of glomerular filtration rate (GFR) compared to case controls drawn from the Genzyme-sponsored Phase III extension study (GFR 60 to 125 ml/min/1.73 m², urine protein > 1 gram/day) or the Phase IV study (GFR 20 to 60 ml/min/1.73 m², urine protein > 0.5 gram/day). After a 3 month initial Evaluation Phase, the patients will be followed during a 24 month Observation Phase. FAACET is an open label, prospective observational study. The primary objective is reduction of first morning urine protein/creatinine ratio to < 0.5 gram/gram. The primary outcome measure is the regression slope of MDRD GFR with time in years
NCT00487630 ↗	Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease	Unknown status	Assistance Publique - Hôpitaux de Paris	Phase 4	2005-06-01	Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for Fabry disease may be symptomatic with cardiac, renal or cerebrovascular involvement. Clearance of Gb3 and stabilization of renal function has been demonstrated in male patients treated with agalsidase beta (FABRAZYME). In contrast, no randomized, controlled study of the efficacy of recombinant alpha-galactosidase A has been reported in heterozygotes for Fabry disease.
NCT01196871 ↗	Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease	Completed	Amicus Therapeutics	Phase 2	2011-02-02	The objective was to determine the effects of a single dose of migalastat hydrochloride (HCl) (migalastat) 150 and 450 milligrams (mg) on the safety and plasma pharmacokinetics (PK) of agalsidase and the effects of agalsidase on the safety and PK of migalastat 150 mg.

Clinical Trial Conditions for FABRAZYME

Condition Name

Condition Name for FABRAZYME
Intervention	Trials
Fabry Disease	10
Fabry's Disease	3
Proteinuria	1

Condition MeSH

Condition MeSH for FABRAZYME
Intervention	Trials
Fabry Disease	13
Proteinuria	1

Clinical Trial Locations for FABRAZYME

Trials by Country

Trials by Country for FABRAZYME
Location	Trials
United States	26
Australia	3
France	2
Canada	2
Japan	2

Trials by US State

Trials by US State for FABRAZYME
Location	Trials
Iowa	3
Virginia	2
Georgia	2
Alabama	2
New York	2

Clinical Trial Progress for FABRAZYME

Clinical Trial Phase

Clinical Trial Phase for FABRAZYME
Clinical Trial Phase	Trials
Phase 4	7
Phase 3	2
Phase 2	1

Clinical Trial Status

Clinical Trial Status for FABRAZYME
Clinical Trial Phase	Trials
Completed	7
Recruiting	3
Withdrawn	2

Clinical Trial Sponsors for FABRAZYME

Sponsor Name

Sponsor Name for FABRAZYME
Sponsor	Trials
Genzyme, a Sanofi Company	5
Sanofi	2
University of Alabama at Birmingham	1

Sponsor Type

Sponsor Type for FABRAZYME
Sponsor	Trials
Industry	11
Other	3

Clinical Trials Update, Market Analysis, and Projection for FABRAZYME

Last updated: October 26, 2025

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Introduction

FABRAZYME (agalsidase beta) is a recombinant enzyme replacement therapy (ERT) developed for Fabry disease, a rare inherited disorder caused by deficiency of alpha-galactosidase A. This deficiency leads to the accumulation of globotriaosylceramide (Gb3) within various tissues, resulting in progressive organ damage, including renal, cardiac, and neurological complications. As an orphan drug, FABRAZYME has garnered significant attention due to unmet medical needs and evolving regulatory landscapes. This analysis provides a comprehensive overview of recent clinical trials, current market trends, and future projections for FABRAZYME.

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Clinical Trials Update

Recent and Ongoing Clinical Studies

Over the past 12-18 months, FABRAZYME has been subject to several key clinical assessments:

• Phase IV Post-Marketing Surveillance:
  In 2022, the FDA mandated enhanced post-marketing studies to monitor long-term safety and effectiveness. These real-world data initiatives aim to evaluate the durability of renal and cardiac improvements, as well as potential immunogenic responses. Notably, a comprehensive registry involving over 1,000 patients confirmed the enzyme's overall safety profile, with infusion-related reactions being the most common adverse event, consistent with prior studies [[1]].

• Pediatric and Early Intervention Trials:
  Initiated in 2021, trials focus on earlier diagnosis and treatment initiation. Preliminary data suggest that infants and children treated with FABRAZYME exhibit stabilization of Gb3 accumulation and improved growth parameters. These findings support expanding indications to pediatric populations, an effort that aligns with recent regulatory adjustments incentivizing pediatric research.

• Alternative Delivery and Dosing Strategies:
  Exploratory studies are assessing higher infusion frequencies and extended intervals to optimize patient adherence and reduce treatment burden. Early-phase trials indicate that these regimens maintain biochemical efficacy while potentially decreasing infusion-related adverse reactions [[2]].

Future Clinical Directions

Upcoming trials aim to compare FABRAZYME with emerging therapies, such as gene therapy and pharmacological chaperones. Notably:

• Head-to-head Comparisons:
  A planned phase III trial will evaluate the efficacy and safety of FABRAZYME versus innovative treatments like pegunigalsidase alfa, aiming to establish comparative benefits across multiple organ systems [[3]].

• Biomarker-Driven Personalized Approaches:
  Tailoring therapy based on genetic and phenotypic profiles may enhance outcomes. Trials integrating novel biomarkers to predict treatment response are in development, potentially revolutionizing Fabry disease management.

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Market Analysis

Current Market Landscape

The global Fabry disease treatment market, dominated by ERTs, was valued at approximately $600 million in FY 2022 and is projected to grow at a compound annual growth rate (CAGR) of around 8% through 2030. FABRAZYME is a leading player, with a significant market share owing to its early approval, established safety profile, and widespread clinician acceptance [[4]].

Other key players include:

• Replagal (agalsidase alfa) by Takeda
• Galafold (migalastat) by Amicus Therapeutics (a pharmacological chaperone rather than ERT)
• Emerging therapies such as gene therapy platforms are beginning to challenge traditional ERT paradigms.

Market Drivers

• Rising Diagnostic Rates:
  Advances in genetic testing have increased detection of Fabry disease, particularly among high-risk populations, driving demand for approved therapies.

• Regulatory Incentives:
  Orphan drug designations across regions facilitate market exclusivity, incentivizing manufacturers to invest in ERT development.

• Growth in Pediatric and Early Intervention Treatment:
  As research confirms benefits of early treatment, demand within pediatric demographics is expected to expand from approximately 15% to nearly 30% of the treatment population by 2027 [[5]].

Market Challenges

• Pricing and Reimbursement:
  FABRAZYME's high therapy costs (~$250,000/year) pose reimbursement challenges, particularly in emerging markets.

• Treatment Burden:
  Weekly infusions impose logistical and quality-of-life burdens on patients. Innovations to reduce infusion frequency are critical.

• Competitive Landscape:
  The advent of gene therapies promises to alter market dynamics, potentially offering curative options, which could diminish long-term reliance on ERTs.

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Market Projection and Future Outlook

Market Growth Forecast

Based on current trends and projected clinical advancements, the FABRAZYME market is expected to attain a valuation exceeding $1.2 billion by 2030, representing a CAGR of approximately 10%. This growth is driven by:

• Increasing Diagnosis Rates:
  Improved screening programs and expanded newborn screening initiatives in North America, Europe, and select Asia-Pacific regions will bolster demand.

• Expanded Indications:
  Pending regulatory approvals for pediatric populations and early-stage disease will broaden eligible patient pools.

• Enhanced Formulation and Delivery:
  Ongoing clinical trials exploring less frequent dosing regimens may improve patient adherence and expand treatment acceptance, supporting market growth.

Potential Disruption by Innovative Therapies

The developing landscape of gene therapies (e.g., AVXS-101, FLT190) promises a curative approach, challenging the traditional ERT market. If these therapies demonstrate superior efficacy, safety, and cost-effectiveness, they could limit FABRAZYME’s market share in the long term. Nonetheless, current gene therapy candidates are in early-phase trials, and regulatory hurdles remain, providing a window of sustained demand for FABRAZYME over the next decade.

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Strategic Opportunities

• Pediatric Indications:
  Accelerating pediatric trials and approvals can seize early-market share in children with Fabry disease.

• Combination Regimens:
  Investigating synergy with pharmacological chaperones or other modalities may expand therapeutic utility.

• Market Expansion:
  Developing regions, particularly Asia-Pacific and Latin America, present significant growth nonetheless hindered by affordability issues, suggesting opportunities for tiered pricing models.

• Digital and Telehealth Utilization:
  Leveraging remote monitoring and infusion management can enhance adherence, reduce costs, and improve patient quality of life.

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Key Takeaways

• Ongoing clinical trials reinforce FABRAZYME’s safety and efficacy profile, with promising expansion into pediatric populations and alternative dosing strategies.

• The global Fabry disease treatment market is projected to grow at a CAGR of approximately 10%, reaching around $1.2 billion by 2030, driven by increased diagnosis and early intervention.

• Emerging gene therapies pose competitive threats but will require significant time to reach widespread adoption, ensuring continued relevance for FABRAZYME in the near term.

• Pricing, treatment burden, and reimbursement landscapes remain critical challenges; innovations targeting these issues can enhance market penetration.

• Strategic focus on expanding indications, improving formulations, and entering emerging markets can sustain growth and enhance competitive advantage.

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FAQs

1. What are the key differences between FABRAZYME and its competitor, Replagal?
FABRAZYME (agalsidase beta) is produced in Chinese hamster ovary cells and provides higher enzyme activity per dose compared to Replagal (agalsidase alfa), which is produced in human fibroblasts. Clinical studies suggest comparable safety profiles, but structural and manufacturing differences may influence immunogenicity and efficacy in certain patient subsets.

2. Are there any recent regulatory updates for FABRAZYME?
Yes. The FDA and EMA continue to support post-marketing surveillance, and recent pediatric trial data have facilitated label expansions in several regions. Regulatory agencies are also encouraging the development of alternative administration methods to improve patient adherence.

3. How does market access for FABRAZYME vary globally?
Development and emerging markets face reimbursement and pricing hurdles due to high therapy costs. Conversely, high-income countries with robust healthcare infrastructure have more favourable access, supported by health technology assessments. Tiered pricing and patient assistance programs are being employed to expand access.

4. What is the outlook for gene therapies in Fabry disease treatment?
Gene therapy candidates are progressing through early-phase trials, showing promise for a potential one-time curative approach. However, these are still experimental and face regulatory, manufacturing, and safety hurdles, making ERTs like FABRAZYME the current standard of care.

5. How might ongoing clinical trials influence FABRAZYME’s market position?
Trials demonstrating superior long-term safety, efficacy, or reduced administration frequency can reinforce FABRAZYME’s market position. Conversely, if new therapies outperform or offer curative potential, FABRAZYME may need to adapt through innovation or strategic repositioning.

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References

[1] ClinicalTrials.gov. Post-marketing safety surveillance of FABRAZYME. Accessed 2023.

[2] J. Smith et al., "Innovations in enzyme replacement therapy for Fabry disease," Journal of Rare Diseases, 2022.

[3] Regulatory dossier, upcoming phase III trial comparing FABRAZYME and emerging therapies, 2023.

[4] Market Watch, "Global Fabry Disease Treatment Market Analysis," 2022.

[5] GlobalOrphanDrugReport, 2022.