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
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
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	Withdrawn	Genzyme, a Sanofi Company	Phase 4	2006-04-01	People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the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.
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.
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.
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."
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.

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

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−

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

Trials by US State

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−

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
Rigshospitalet, Denmark	1

Sponsor Type

Sponsor Type for FABRAZYME
Sponsor	Trials
Industry	11
Other	3

Fabrazyme: Clinical Trials, Market Analysis, and Projections

Introduction to Fabrazyme

Fabrazyme, also known as agalsidase beta, is an enzyme replacement therapy (ERT) developed by Genzyme (now part of Sanofi) for the treatment of Fabry disease. This rare genetic disorder is characterized by the deficiency of the enzyme alpha-Gal A, leading to the accumulation of globotriaosylceramide (Gb3) in various tissues, which can result in severe health complications.

Clinical Trials Overview

Phase 3 Clinical Trial

The Phase 3 clinical trial for Fabrazyme, initiated in 1999, enrolled 58 patients with Fabry disease, aged 16-61, who had not previously received ERT. Participants were randomly assigned to receive either Fabrazyme or a placebo every two weeks for 20 weeks. The primary objective was to compare the number of patients with no or nearly no Gb3 accumulation in kidney capillaries and to assess the safety and efficacy of Fabrazyme. The results showed significant clearance of Gb3 from kidney capillaries in the Fabrazyme-treated group compared to the placebo group[1][4].

Phase 4 Clinical Trial

The Phase 4 trial involved 82 adults with advanced Fabry disease, aged 20-72, who had never received ERT. Patients were randomly assigned to Fabrazyme or a placebo, administered every two weeks for up to 35 months. The study aimed to determine if Fabrazyme could delay the time to clinically significant events such as heart attacks, kidney failure, strokes, or death. The results indicated a 53% delay in these events among patients treated with Fabrazyme compared to those on the placebo, with a median treatment duration of 18.5 months[1][4].

Phase 2 Trial in Children

The pediatric Phase 2 study enrolled 16 children and adolescents with Fabry disease, aged 8-16. All participants were treated with Fabrazyme for about a year. The study found that Gb3 accumulation in skin capillaries was no longer detectable in any of the 12 patients who had detectable levels at the start of the trial after six months. Additionally, Gb3 levels in the blood decreased significantly in male patients within the first four weeks of treatment and remained within normal limits throughout the study[1].

Clinical Outcomes and Safety

Fabrazyme has demonstrated significant clinical benefits in reducing the accumulation of Gb3 in various tissues, including the kidneys, heart, and skin. The treatment has been shown to substantially lower the rate of renal, cardiac, and cerebrovascular clinical events, with a relative risk reduction of 61% compared to placebo-treated patients[4].

However, long-term follow-up data indicate that patients on Fabrazyme may still experience a decline in their estimated glomerular filtration rate (eGFR) scores, albeit at a slower rate than those not receiving the treatment. For instance, patients on Fabrazyme saw a decline in their eGFR scores of 1.5 mL per minute per 1.73 m² each year over up to five years of follow-up[1].

Market Analysis

Current Market Size and Growth

The global treatment market for Fabry disease is projected to grow significantly. In 2014, the market was valued at $683 million, and it is expected to reach approximately $1.25 billion by 2024, driven by increasing physician awareness and higher diagnosis rates. This growth represents a moderate Compound Annual Growth Rate (CAGR) of 6.3% across the seven major markets (7MM) of the US, France, Germany, Italy, Spain, UK, and Japan[2].

Market Leaders and Competition

Fabrazyme is the market leader in the ERT segment for Fabry disease, particularly in the US where it is the only approved therapy. Other key players in the market include Shire’s Replagal and Amicus Therapeutics’ migalastat, a pharmacological chaperone. Despite the entry of new therapies, ERT is expected to remain the standard of care due to its positive reimbursement status and coverage by insurance plans[2][3].

Regional Market Dynamics

North America is currently the largest market for Fabry disease treatments, driven by high healthcare expenditure and increasing awareness of the condition. The Asia-Pacific region is expected to be the fastest-growing market over the forecast period, driven by incremental healthcare spending and the introduction of new therapies[5].

Market Projections

Forecasted Sales and Growth

The market for Fabry disease treatments is expected to continue growing, with a projected CAGR of 6.8% from 2024 to 2029. Fabrazyme is forecasted to maintain its market dominance, although it will face competition from emerging therapies. The report by ResearchAndMarkets predicts that the market scenario for Fabry disease will change due to extensive research and incremental healthcare spending, expanding the market size and enabling drug manufacturers to penetrate more deeply into the market[3][5].

Emerging Therapies and Challenges

The launch of late-stage emerging therapies is expected to significantly impact the market. For instance, Genzyme has initiated clinical trials to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms in Fabry disease patients. These new therapies will focus on novel approaches to treat and improve the disease condition, posing both challenges and opportunities for existing treatments like Fabrazyme[5].

Key Takeaways

• Clinical Efficacy: Fabrazyme has shown significant efficacy in reducing Gb3 accumulation and delaying clinically significant events in patients with Fabry disease.
• Market Growth: The global Fabry disease treatment market is expected to grow from $683 million in 2014 to $1.25 billion by 2024, driven by increasing awareness and diagnosis rates.
• Market Leadership: Fabrazyme remains the market leader in the ERT segment, particularly in the US.
• Regional Dynamics: North America is the largest market, while the Asia-Pacific region is expected to be the fastest-growing.
• Emerging Therapies: New therapies, including pharmacological chaperones and other novel approaches, are expected to enter the market, posing competition to Fabrazyme.

FAQs

What are the key clinical trials that supported the approval of Fabrazyme?

The approval of Fabrazyme was supported by a Phase 3 clinical trial, a Phase 4 trial, and a Phase 2 trial in children and adolescents[1].

How does Fabrazyme impact the accumulation of Gb3 in patients with Fabry disease?

Fabrazyme significantly reduces the accumulation of Gb3 in the kidneys, heart, and skin, as demonstrated in various clinical trials[1][4].

What is the current market size and growth rate of the Fabry disease treatment market?

The global treatment market for Fabry disease is expected to reach $1.25 billion by 2024, growing at a CAGR of 6.3% from 2014[2].

Which region is expected to be the fastest-growing market for Fabry disease treatments?

The Asia-Pacific region is expected to be the fastest-growing market over the forecast period[5].

What are the main competitors to Fabrazyme in the Fabry disease treatment market?

The main competitors include Shire’s Replagal and Amicus Therapeutics’ migalastat, along with emerging therapies[2][3].

Sources

1. Fabry Disease News: "Fabrazyme (agalsidase beta) for Fabry disease"
2. Pharm Exec: "Fabry Disease Treatment Market Value to Reach $1.25 Billion by 2024"
3. ResearchAndMarkets: "FABRAZYME, Drug Insight and Market Forecast - 2032"
4. Sanofi Campus: "Fabrazyme (agalsidase beta) Evidence"
5. Mordor Intelligence: "Fabry Disease Treatment Market Size & Share Analysis"