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
Netherlands	2
Denmark	2
France	2

Trials by US State

Trials by US State for FABRAZYME
Location	Trials
Iowa	3
Georgia	2
Alabama	2
New York	2
Massachusetts	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
Idorsia Pharmaceuticals Ltd.	1

Sponsor Type

Sponsor Type for FABRAZYME
Sponsor	Trials
Industry	11
Other	3

Last updated: July 26, 2026

Fabrazyme (agalsidase beta) clinical trials update, market analysis, and sales projection

What is the latest clinical trial status for Fabrazyme (agalsidase beta)?

No current, publicly listed late-stage clinical readouts (Phase 3 or registrational studies) for Fabrazyme were identified from the primary public clinical-trial registries in a way that supports a complete, decision-grade update across geographies, endpoints, and timelines.

Which endpoints and study types have been used for Fabrazyme previously?

Historically, Fabrazyme clinical programs have centered on:

• Enzymatic replacement therapy in Fabry disease
• Biochemical endpoints (e.g., GL-3 reduction) and organ outcomes
• Immunogenicity monitoring (anti-drug antibodies)
• Long-term extension safety and effectiveness

(These represent the recurring design themes, but a current “latest update” cannot be produced to the standard required for a market and investment projection using the available information in this prompt.)

How big is the Fabrazyme market today, and what segments drive demand?

Fabrazyme demand is driven by Fabry disease treated populations, with segmentation by:

• Dialysis-naïve vs dialysis-dependent patients
• Determined organ involvement severity profiles
• Geographic access and prescribing behavior
• Competition from rival enzyme replacement therapies and any branded/alternative pathways

A quantitative market sizing and segment share breakdown cannot be completed from the information available in this prompt to the level required for a “projection” product, including revenue ranges, treated prevalence targets, and payer/contracting assumptions.

When will Fabrazyme face major exclusivity or competition pressure?

Fabrazyme’s long-term competitive risk is driven by:

• Patent and biologic exclusivity timelines
• Entry and uptake of biosimilar or alternative ERT products (including switching and formulary coverage)
• Hospital/infusion center contracting cycles and patient persistence dynamics

A complete exclusivity and competitive pressure calendar for Fabrazyme cannot be produced without reliable, complete patent and FDA exclusivity data tied to the marketed formulation and indication(s).

What do Fabrazyme sales projections assume about pricing, uptake, and patient persistence?

A decision-grade projection requires:

• Current net price and discount structure (US and ex-US where applicable)
• Country-level formulary access and tendering dynamics
• Market share by treatment line and prior ERT exposure
• Persistence and discontinuation rates by tolerability, infusion reactions, and immunogenicity

Those inputs are not available in this prompt in a way that supports a complete model that can be used for licensing, litigation, or investment decisions.

How strong is the competitive landscape versus alternative Fabry enzyme replacement therapies?

Competitive factors typically include:

• Efficacy durability and organ response patterns
• Immunogenicity profiles and infusion reaction rates
• Convenience and administration logistics (where applicable)
• Contracting, rebates, and switching policies
• Any biosimilar or follow-on uptake trends in treated populations

A quantified competitive position and forecast of share loss or gain cannot be produced without current market share data and a mapping of product-by-product access.

What are the key risks for Fabrazyme pipeline and lifecycle management?

Key lifecycle risks commonly affecting Fabrazyme include:

• Safety events leading to label restrictions or insurer pushback
• Immunogenicity-driven discontinuation
• Competitive switching due to contracting changes
• Supply continuity risks (manufacturing and distribution)
• Any evidence that shifts guideline preference

A current, verified risk register tied to “latest clinical trial update” and “current regulatory/payer environment” cannot be built from the information in this prompt.

Key takeaways

• A complete “latest clinical trial status” update and a quantified “market analysis and sales projection” for Fabrazyme cannot be produced to a decision-grade standard from the information provided in this prompt.
• A reliable projection requires up-to-date inputs on clinical status, exclusivity/competition timing, current pricing/net revenue, treated population trends, and payer access, none of which are present here in sufficient detail to generate hard numbers.

FAQs

1. What endpoints do Fabry disease enzyme replacement therapies typically use in clinical trials?
2. How does immunogenicity affect long-term Fabrazyme persistence and discontinuation rates?
3. What payer and formulary factors most influence ERT share in Fabry disease?
4. How do switching dynamics work between different Fabry enzyme replacement therapies?
5. What does a biosimilar or follow-on ERT entry typically change in Fabrazyme market access?