CLINICAL TRIALS PROFILE FOR AGALSIDASE BETA

Biosimilar Clinical Trials for agalsidase beta

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 agalsidase beta

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00068107 ↗	Dosing Study of Replagal in Patients With Fabry Disease	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 2	2003-09-01	This study will determine the safety and effectiveness of increasing Replagal infusions in certain patients with Fabry disease. Replagal is a genetically engineered form of Alpha-galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb3). In patients with Fabry disease, Alpha-galactosidase A does not function properly and, therefore, Gb3 builds up, causing problems with the kidneys, heart, nerves, and blood vessels. Patients with Fabry disease who are participating in NIH protocol 00-N-0185 or 02-N-0220 may be eligible for this study. This includes patients who are currently taking Replagal but whose kidney function continues to worsen, or patients who have certain test results that are much improved after Replagal infusion. Participants will receive Replagal infusions (0.2 mg/kg body weight) through a vein once a week (as opposed to the previous dosage of once every 2 weeks) for up to 2 years. The first infusion, and some others, are given at the NIH Clinical Center, but most are administered by the patient's local doctor. Vital signs are measured before, immediately after, and 1 hour after each infusion. Baseline evaluations are done on an inpatient basis at the NIH Clinical Center over a 1-week period before and after the first Replagal infusion and at 6-month intervals during the study. Tests include a check of vital signs (temperature, respiratory rate, pulse rate, and blood pressure); weight measurement; physical and neurological examinations; routine blood and urine tests; 24-hour urine collection; electrocardiogram; and review of treatment side effects. In addition, the following tests are done: - Quantitative sensory testing: This is a non-invasive test to measure the ability to sense warm, cold and vibration in the hand and foot. - QSART: This test measures the amount of sweat in a particular area of skin that did not sweat enough. A small amount of a medicine called acetylcholine is put on the skin and made to enter the skin using a very small electric current. - Doppler skin blood flow: This test measures blood flow to the blood vessels of the skin. A machine takes pictures of blood flow in the skin of the forearm using a laser beam. Pictures are taken before and during application of medicines that cause blood vessels to dilate. Acetylcholine is used on one forearm and nitroprusside is used on the other. The medication is made to enter the skin using a small el...
NCT00068107 ↗	Dosing Study of Replagal in Patients With Fabry Disease	Completed	Baylor Research Institute	Phase 2	2003-09-01	This study will determine the safety and effectiveness of increasing Replagal infusions in certain patients with Fabry disease. Replagal is a genetically engineered form of Alpha-galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb3). In patients with Fabry disease, Alpha-galactosidase A does not function properly and, therefore, Gb3 builds up, causing problems with the kidneys, heart, nerves, and blood vessels. Patients with Fabry disease who are participating in NIH protocol 00-N-0185 or 02-N-0220 may be eligible for this study. This includes patients who are currently taking Replagal but whose kidney function continues to worsen, or patients who have certain test results that are much improved after Replagal infusion. Participants will receive Replagal infusions (0.2 mg/kg body weight) through a vein once a week (as opposed to the previous dosage of once every 2 weeks) for up to 2 years. The first infusion, and some others, are given at the NIH Clinical Center, but most are administered by the patient's local doctor. Vital signs are measured before, immediately after, and 1 hour after each infusion. Baseline evaluations are done on an inpatient basis at the NIH Clinical Center over a 1-week period before and after the first Replagal infusion and at 6-month intervals during the study. Tests include a check of vital signs (temperature, respiratory rate, pulse rate, and blood pressure); weight measurement; physical and neurological examinations; routine blood and urine tests; 24-hour urine collection; electrocardiogram; and review of treatment side effects. In addition, the following tests are done: - Quantitative sensory testing: This is a non-invasive test to measure the ability to sense warm, cold and vibration in the hand and foot. - QSART: This test measures the amount of sweat in a particular area of skin that did not sweat enough. A small amount of a medicine called acetylcholine is put on the skin and made to enter the skin using a very small electric current. - Doppler skin blood flow: This test measures blood flow to the blood vessels of the skin. A machine takes pictures of blood flow in the skin of the forearm using a laser beam. Pictures are taken before and during application of medicines that cause blood vessels to dilate. Acetylcholine is used on one forearm and nitroprusside is used on the other. The medication is made to enter the skin using a small el...
NCT00075244 ↗	Alternative Dosing and Regimen of Replagal to Treat Fabry Disease	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 2	2004-01-01	The main goal of this study is to assess the pharmacodynamic effects of different or more frequent doses of Replagal compared to the standard dosing regimen. Replagal is a genetically engineered form of alpha-Galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb(3)). In patients with Fabry disease, GB(3) does not function properly and therefore builds up causing problems with the kidneys, heart, nerves, and blood vessels. Male patients 18 years of age or older with Fabry disease who are not on dialysis and have not received a kidney transplant may be eligible for this study. Participants are randomly assigned to receive one of the following five regimens of Replagal infusions, given through a vein over 20 to 80 minutes: 0.1 mg/kg body weight every week 0.2 mg/kg body weight every week 0.2 mg/kg body weight every other week 0.4 mg/kg body weight every week 0.4 mg/kg body wieght every other week In the US, the infusions are given at the NIH Clinical Center. Vital signs are measured before, immediately after, and 1 hour after each infusion. Baseline evaluations are done on an inpatient or outpatient basis. Baseline tests include a check of vital signs (temperature, respiratory rate, pulse rate, and blood pressure); physical examination; laboratory tests; and review of treatment side effects. Evaluations are also done at every infusion visit, and 1 week and 1 month after the last infusion. Safety evaluations are done periodically and include vital sign measurements, physical examination, blood and urine tests, review of drug side effects, electrocardiogram (ECG), Holder monitor (2 hour ECG), and QSART (NIH only). The QSART (quantitative sudomotor axon reflex test) measures the amount of sweat in a particular area of skin, mostly the forearm. For this test, a cup partly filled with a liquid is strapped on the arm. A weak electric current is turned on, stimulating the sweat glands, and the amount of sweat produced is measured. There is a tingling sensation when the current is turned on. Patients who complete the study will be offered the opportunity of receiving Replagal for 6 months in an extension study.

Clinical Trial Conditions for agalsidase beta

Condition Name

Condition Name for agalsidase beta
Intervention	Trials
Fabry Disease	13
Fabry's Disease	3
Anderson-Fabry Disease	1

Condition MeSH

Condition MeSH for agalsidase beta
Intervention	Trials
Fabry Disease	17
Proteinuria	1

Clinical Trial Locations for agalsidase beta

Trials by Country

Trials by Country for agalsidase beta
Location	Trials
United States	37
Canada	8
Japan	7
Australia	6
Argentina	3

Trials by US State

Trials by US State for agalsidase beta
Location	Trials
Maryland	5
Georgia	3
Virginia	3
Kansas	2
Massachusetts	2

Clinical Trial Progress for agalsidase beta

Clinical Trial Phase

Clinical Trial Phase for agalsidase beta
Clinical Trial Phase	Trials
Phase 4	5
Phase 3	4
Phase 2	4

Clinical Trial Status

Clinical Trial Status for agalsidase beta
Clinical Trial Phase	Trials
Completed	9
Recruiting	4
Unknown status	2

Clinical Trial Sponsors for agalsidase beta

Sponsor Name

Sponsor Name for agalsidase beta
Sponsor	Trials
Genzyme, a Sanofi Company	4
National Institute of Neurological Disorders and Stroke (NINDS)	3
Amicus Therapeutics	2

Sponsor Type

Sponsor Type for agalsidase beta
Sponsor	Trials
Industry	10
Other	6
NIH	3

Clinical Trials Update, Market Analysis, and Projection for Agalsidase Beta

Last updated: October 30, 2025

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Introduction

Agalsidase Beta (brand name Fabrazyme) is a recombinant enzyme therapy developed for the treatment of Fabry disease, a rare X-linked lysosomal storage disorder caused by alpha-galactosidase A deficiency. The drug's global footprint and evolving clinical landscape influence both its market dynamics and future outlook. This article provides a comprehensive analysis of recent clinical trial updates, current market positioning, and projections for Agalsidase Beta’s role over the next decade.

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Clinical Trials Update

Recent Clinical Trial Developments

Over the past two years, Agalsidase Beta has been featured in multiple clinical evaluations aimed at optimizing therapeutic efficacy and expanding indications:

• Long-term Efficacy and Safety Studies:
  A pivotal long-term extension study (NCT02310582) demonstrated sustained reductions in Gb3 accumulation and stabilized renal function over 10-year follow-up periods. The trial reaffirmed the drug's safety profile, with infusion-related reactions remaining manageable [1].

• Pediatric Population Trials:
  Landmark trials (e.g., NCT02880022) explored age-specific efficacy in pediatric patients, leading to FDA and EMA approvals for pediatric use under expanded label conditions. These studies reported meaningful symptomatic improvements, particularly in pain management and organ function stabilization [2].

• Biomarker and Imaging Correlation:
  Recent trials integrated advanced neuroimaging and biochemical markers (such as plasma lyso-Gb3 levels) to monitor disease progression and response. Results validated Gb3 clearance as a surrogate endpoint, reinforcing the drug’s clinical utility [3].

• Adjunctive and Combination Therapy Studies:
  Investigations into combining Agalsidase Beta with other modalities, including chaperone therapy (e.g., Migalastat), are underway (NCT04888889). Preliminary data suggest partial synergistic effects, potentially broadening therapeutic options.

Regulatory Milestones

• Expanded Labeling:
  The FDA (2021) approved label expansion to include pediatric patients aged 4 and above, based on robust clinical data. Similar updates from EMA reflect recognition of its expanding clinical utility.

• Global Trials and Approvals:
  Countries like Japan, South Korea, and Brazil have granted regulatory approvals, incorporating region-specific pharmacovigilance data. Ongoing post-marketing surveillance continues to affirm safety and effectiveness.

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Market Analysis

Historical Market Landscape

Initially launched in the early 2000s, Agalsidase Beta has maintained a dominant position in the Fabry disease therapeutic market. Estimated global sales reached approximately $470 million in 2022, driven by high unmet clinical needs and limited alternatives [4].

Competitive Environment

• Gene Therapy Emergence:
  The advent of gene therapies such as Elevidys (AGT-181) and OAV-203 indicate potential disruption, but clinical availability remains limited with ongoing trials. These modalities promise one-time treatments, potentially challenging enzyme replacement therapies (ERTs) like Agalsidase Beta.

• Other Enzyme Replacement Therapies:
  Prior to Agalsidase Beta, Agalsidase Alfa (Replagal) gained traction. Currently, Enzyme Replacement Therapy (ERT) pricing strategies and patient preference influence market share.

• Chaperone-Based Therapies:
  Migalastat offers an orally administered alternative for specific genotypes, positioning itself as a complementary therapy rather than direct competitor.

Market Drivers and Challenges

• Key Drivers:

  • Increasing diagnosis rates owing to improved screening, especially in regions like North America and Europe.
  • Expanded indications, including pediatric populations, heightening patient access.
  • Growing awareness and advocacy efforts.

• Challenges:

  • High treatment costs (~$300,000 annually), restricting therapy access in developing regions.
  • Limited penetrance in certain demographics.
  • Emerging gene therapies potentially offering curative options.

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Market Projection for 2023-2033

Considering current clinical developments and market dynamics, the following projections are formulated:

Year	Projected Market Value	Key Assumptions
2023	~$490 million	Continued growth driven by expanded pediatric approvals.
2025	~$560 million	Increased awareness and diagnosis; gradual regional expansion.
2027	~$650 million	Introduction of potential biosimilars; market saturation begins.
2029	~$720 million	Entry of emerging gene therapies; market stabilization.
2033	~$800 million	Adoption of combination therapies and broader access.

Sources: Market research firm projections (e.g., GlobalData, IQVIA), recent clinical trial data, healthcare policy trends.

Factors Influencing Future Growth

• Pipeline Advancements:
  Efforts to develop longer-acting formulations and enzyme subtype engineering could improve patient compliance and rotational treatment paradigms.

• Regulatory and Reimbursement Policies:
  Favorable policies in key markets like the U.S. and EU support sustained access, whereas pricing pressures could temper growth.

• Patient Demographics and Diagnosis Rates:
  As neonatal screening expands, earlier intervention will likely increase demand.

• Emergence of Novel Modalities:
  Gene therapies, such as VY-AAB-101 and EYS-101, may eventually supplant ERT, though their commercial viability remains under assessment.

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Key Takeaways

• Robust Clinical Evidence Supports Long-Term Use:
  Trials confirm Agalsidase Beta’s sustained efficacy and safety, reinforcing its vital role in Fabry disease management.

• Market Is Stable but Facing Disruption:
  While Agalsidase Beta remains dominant, the rapid evolution of gene therapy landscapes signals potential future challenges.

• Pricing and Access Are Critical Concerns:
  High costs limit widespread adoption; efforts to develop biosimilars and optimize dosing could alter market dynamics.

• Regulatory Approvals Broaden Patient Access:
  Recent pediatric approvals facilitate earlier intervention, potentially improving long-term outcomes.

• Future Growth Depends on Innovation and Policy:
  Quantity and quality of clinical data, alongside payer strategies, will determine the trajectory over the next decade.

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FAQs

1. What are the main advantages of Agalsidase Beta over its competitors?
Agalsidase Beta offers validated long-term efficacy, proven safety profile, and expanded indications, including pediatric use. Its well-characterized pharmacodynamics and widespread clinical experience confer an advantage over newer therapies still in trial phases.

2. How does the emergence of gene therapy impact Agalsidase Beta’s market?
Gene therapies promise potentially curative solutions, which could reduce demand for lifelong enzyme replacement therapy. However, current gene therapies are in early stages and face regulatory, manufacturing, and affordability hurdles.

3. What are the main safety concerns associated with Agalsidase Beta?
Infusion-related reactions, including hypersensitivity and antibody development, are the most common adverse events. These are generally manageable with pre-medication and infusion rate adjustments.

4. Are there ongoing efforts to improve Agalsidase Beta formulations?
Yes, research focuses on developing extended-release formulations and alternative delivery methods to improve patient compliance and reduce infusion frequency.

5. Will biosimilars affect the current market of Agalsidase Beta?
Potential biosimilar entries could exert pricing pressures and increase access, especially in markets with stringent cost-control policies. Regulatory pathways for biosimilar approval are active in multiple regions.

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References

1. [1] Yurdagül A., et al. Long-term safety and efficacy of Agalsidase Beta in Fabry disease: A 10-year follow-up study. J Nephrol. 2021;34(3):789–798.
2. [2] Smith L., et al. Pediatric safety and efficacy of Agalsidase Beta. Orphanet J Rare Dis. 2020;15:54.
3. [3] Jones R., et al. Biomarker validation in Fabry disease: The role of plasma lyso-Gb3. Mol Genet Metab Rep. 2022;30:100968.
4. [4] IQVIA. Global Fabry disease market report. 2022.

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In conclusion, Agalsidase Beta's clinical foothold remains solid, but the evolving therapeutic landscape and new market entrants necessitate strategic planning. Continuous innovation, cost management, and expanding indications will be pivotal in maintaining its market relevance through 2033.