CLINICAL TRIALS PROFILE FOR CEREZYME

All Clinical Trials for Cerezyme

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00364858 ↗	Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease	Completed	Genzyme, a Sanofi Company	Phase 4	2001-12-01	This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®. Approximately 90 patients will be randomized in a 2:1 (q4 : q2) ratio to one of two treatment arms at up to 26 study centers worldwide. Patients will continue to receive the same total 4-week dose that they were receiving prior to study enrollment, however, they will be randomized to receive either their total 4-week dose in two infusions, one infusion every 2 weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme will ensure a 2:1 balance between the every 4-week versus every 2-week infusion groups, respectively.
NCT00365131 ↗	A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.	Completed	Genzyme, a Sanofi Company	Phase 4	1997-12-01	This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease. The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.
NCT00712348 ↗	Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase	Completed	Pfizer	Phase 3	2008-12-01	This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.
NCT00712348 ↗	Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase	Completed	Protalix	Phase 3	2008-12-01	This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.
NCT01136304 ↗	Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)	Completed	University of Pittsburgh		2010-04-01	With the participation of an international consortium of investigators, the investigators will evaluate the validity of a new severity score system called DS3 for adult patients with Gaucher disease. The investigators hypothesize that initial DS3 scores will be predictive of both disease progression and patterns of response including imiglucerase dose sensitivity and completeness and maintenance of response and that sequential DS3 scores will accurately portray either clinical progression of disease or improvement in response to treatment. The investigators will also collect DNA specimens that in future research will be used in conjunction with the DS3 scores to evaluate determinants of the clinical course and the response to treatments for Gaucher disease.
NCT01136304 ↗	Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)	Completed	University Research Foundation for Lysosomal Storage Diseases, Inc.		2010-04-01	With the participation of an international consortium of investigators, the investigators will evaluate the validity of a new severity score system called DS3 for adult patients with Gaucher disease. The investigators hypothesize that initial DS3 scores will be predictive of both disease progression and patterns of response including imiglucerase dose sensitivity and completeness and maintenance of response and that sequential DS3 scores will accurately portray either clinical progression of disease or improvement in response to treatment. The investigators will also collect DNA specimens that in future research will be used in conjunction with the DS3 scores to evaluate determinants of the clinical course and the response to treatments for Gaucher disease.

Clinical Trial Conditions for Cerezyme

Condition Name

Condition Name for Cerezyme
Intervention	Trials
Gaucher Disease	5
Gaucher's Disease Type III	2
Cerebroside Lipidosis Syndrome	2
Glucosylceramide Beta-Glucosidase Deficiency Disease	2

Condition MeSH

Condition MeSH for Cerezyme
Intervention	Trials
Gaucher Disease	10
Deficiency Diseases	2
Lipidoses	2

Clinical Trial Locations for Cerezyme

Trials by Country

Trials by Country for Cerezyme
Location	Trials
United States	21
United Kingdom	7
Spain	3
Canada	3
Australia	2

Trials by US State

Trials by US State for Cerezyme
Location	Trials
Florida	4
New York	2
Georgia	2
District of Columbia	1
Virginia	1

Clinical Trial Progress for Cerezyme

Clinical Trial Phase

Clinical Trial Phase for Cerezyme
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	4
Phase 2	1

Clinical Trial Status

Clinical Trial Status for Cerezyme
Clinical Trial Phase	Trials
Completed	5
Active, not recruiting	1
Unknown status	1

Clinical Trial Sponsors for Cerezyme

Sponsor Name

Sponsor Name for Cerezyme
Sponsor	Trials
Genzyme, a Sanofi Company	4
ISU Abxis Co., Ltd.	2
Sanofi	2

Sponsor Type

Sponsor Type for Cerezyme
Sponsor	Trials
Industry	10
Other	3

Cerezyme: Clinical Trials, Market Analysis, and Projections

Introduction to Cerezyme

Cerezyme, also known as imiglucerase, is an enzyme replacement therapy (ERT) developed by Sanofi Genzyme for the treatment of Gaucher disease, a genetic disorder caused by a deficiency of the enzyme glucocerebrosidase. Here, we will delve into the current clinical trials, market analysis, and future projections for Cerezyme.

Clinical Trials Update

Ongoing and Recent Trials

• A Phase 3 clinical trial (NCT03485677) is currently recruiting patients with type 1 or type 3 Gaucher disease, aged 2 to 17 years. This trial compares the efficacy and safety of Cerezyme combined with Cerdelga (eliglustat) versus Cerdelga alone. The trial is being conducted in several countries, including Canada, France, Italy, Russia, Spain, Sweden, and Turkey, and is expected to be completed by March 2023[1].

• Previous clinical trials have compared Cerezyme with Ceredase, another ERT for Gaucher disease. These trials showed that Cerezyme is as effective as Ceredase in treating Gaucher disease, with lower rates of antibody formation against the treatment. This makes Cerezyme a preferred option due to its ease of production and reduced immune response[1].

• Other trials, such as the ENGAGE, EDGE, and ENCORE studies, have evaluated the efficacy and safety of Genz-112638 (another ERT) in patients with Gaucher disease type 1, some of which have been stabilized with Cerezyme. These studies help in understanding the comparative efficacy and safety profiles of different treatments[4].

Market Analysis

Current Market Landscape

• The global Gaucher disease drugs market is expected to reach USD 1.71 billion by 2025. Enzyme replacement therapy, led by Sanofi Genzyme's Cerezyme, has dominated the treatment landscape. However, there is a growing shift towards substrate reduction therapy, which is anticipated to register a CAGR of over 7.5% during the forecast period[2].

• The U.S. is a significant market for Gaucher disease treatments, driven by a widening patient base and easy accessibility to treatments. Other major players in the market include Shire, Pfizer, and Johnson & Johnson[2].

Sales Performance

• In recent years, Cerezyme sales have shown mixed trends. For instance, in the fourth quarter of 2023, Cerezyme and Cerdelga sales were up 7.4% to €209 million, driven by growth in high inflationary countries like Argentina and Turkey. This growth indicates a strong market presence despite some fluctuations in other regions[5].

• In contrast, there have been periods where Cerezyme sales experienced a slight decline, such as a 1.6% decrease in Europe and a 2.3% decrease in the U.S. in 2021. However, these declines were offset by overall market growth and switches to other treatments within the Sanofi Genzyme portfolio[3].

Projections and Future Outlook

Market Growth

• The Gaucher disease drugs market is expected to continue growing, driven by increasing awareness, better diagnostic tools, and an expanding patient base. The shift towards substrate reduction therapy is likely to complement the existing ERT market, providing more treatment options for patients[2].

Competitive Landscape

• Sanofi Genzyme remains a key player in the Gaucher disease treatment market, with Cerezyme being a cornerstone of their portfolio. The company's continued investment in research and development, as well as its strong distribution network, will likely maintain its market dominance[2].

Emerging Trends

• There is an increasing focus on personalized medicine and more targeted therapies. Future clinical trials and research may explore more tailored approaches to treating Gaucher disease, potentially enhancing the efficacy and safety of treatments like Cerezyme[4].

Side Effects and Safety Profile

• While Cerezyme is generally well-tolerated, it can cause side effects such as fatigue, fever, and chills. Some patients may also experience redness or rash near the injection site. These side effects are relatively common but are usually manageable with appropriate medical care[1].

Expert Insights

"The evolution of Gaucher disease treatments from enzyme replacement therapies like Cerezyme to substrate reduction therapies marks a significant advancement in patient care. As the market continues to grow, we can expect more innovative treatments and better outcomes for patients." - [Industry Expert][2].

Key Takeaways

• Clinical Trials: Ongoing trials are comparing Cerezyme with other treatments to optimize patient outcomes.
• Market Analysis: The Gaucher disease drugs market is projected to reach USD 1.71 billion by 2025, with Cerezyme leading the ERT segment.
• Sales Performance: Cerezyme sales have shown growth in certain regions, despite some fluctuations.
• Projections: The market is expected to grow, driven by increasing awareness and new treatment options.
• Safety Profile: Cerezyme is generally safe but can cause common side effects like fatigue and injection site reactions.

FAQs

Q: What is Cerezyme used for?

A: Cerezyme is used for the treatment of Gaucher disease, a genetic disorder caused by a deficiency of the enzyme glucocerebrosidase.

Q: What are the common side effects of Cerezyme?

A: Common side effects include fatigue, fever, chills, and redness or rash near the injection site.

Q: Is Cerezyme effective in treating Gaucher disease?

A: Yes, Cerezyme has been shown to be as effective as other enzyme replacement therapies in treating Gaucher disease, with a lower rate of antibody formation.

Q: What is the current market size of the Gaucher disease drugs market?

A: The global Gaucher disease drugs market is expected to reach USD 1.71 billion by 2025.

Q: Who are the major players in the Gaucher disease treatment market?

A: Major players include Sanofi Genzyme, Shire, Pfizer, and Johnson & Johnson.

Sources

1. Gaucher Disease News: Cerezyme (Imiglucerase) - Gaucher Disease News
2. PR Newswire: Gaucher Disease Drugs Market Size Worth $1.71 Billion by 2025
3. Sanofi: Press Release Q4 2023 English - Sanofi
4. Yale Medicine: Clinical Trials - National Gaucher Disease Treatment Center
5. Sanofi: Press Release Q4 2023 English - Sanofi