CLINICAL TRIALS PROFILE FOR VELAGLUCERASE ALFA

All Clinical Trials for Velaglucerase Alfa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00391625 ↗	Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)	Completed	Shire	Phase 1/Phase 2	2004-09-13	Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the long term safety of enzyme replacement therapy with DRX008A (VPRIV®, GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher disease.
NCT00954460 ↗	Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease	Approved for marketing	Shire		1969-12-31	Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this treatment protocol is to observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase.
NCT01842841 ↗	Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease	Completed	Quintiles, Inc.	Phase 3	2013-03-13	Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with Type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression. The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa. Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.
NCT01842841 ↗	Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease	Completed	Shire	Phase 3	2013-03-13	Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with Type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression. The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa. Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.
NCT02528617 ↗	The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease	Withdrawn	Texas Scottish Rite Hospital for Children	Phase 4	2015-07-01	The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.

Clinical Trial Conditions for Velaglucerase Alfa

Condition Name

Condition Name for Velaglucerase Alfa
Intervention	Trials
Gaucher Disease	4
Gaucher Disease, Type 1	2
Gaucher Disease Type 1	1
Gaucher Disease Type 3	1

Condition MeSH

Condition MeSH for Velaglucerase Alfa
Intervention	Trials
Gaucher Disease	9

Clinical Trial Locations for Velaglucerase Alfa

Trials by Country

Trials by Country for Velaglucerase Alfa
Location	Trials
United States	26
Japan	3
Israel	3
Romania	1
United Kingdom	1

Trials by US State

Trials by US State for Velaglucerase Alfa
Location	Trials
Virginia	2
Pennsylvania	2
North Carolina	2
New York	2
Illinois	2

Clinical Trial Progress for Velaglucerase Alfa

Clinical Trial Phase

Clinical Trial Phase for Velaglucerase Alfa
Clinical Trial Phase	Trials
Phase 4	4
Phase 3	2
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Velaglucerase Alfa
Clinical Trial Phase	Trials
Completed	4
Not yet recruiting	2
Approved for marketing	1

Clinical Trial Sponsors for Velaglucerase Alfa

Sponsor Name

Sponsor Name for Velaglucerase Alfa
Sponsor	Trials
Shire	5
Shaare Zedek Medical Center	2
Takeda	1

Sponsor Type

Sponsor Type for Velaglucerase Alfa
Sponsor	Trials
Industry	8
Other	4

Velaglucerase Alfa: Clinical Trials, Market Analysis, and Projections

Introduction to Velaglucerase Alfa

Velaglucerase alfa, marketed as VPRIV, is an enzyme replacement therapy (ERT) specifically designed for the treatment of Type 1 Gaucher disease (GD1), a rare genetic disorder characterized by the deficiency of the enzyme glucocerebrosidase. This enzyme is crucial for breaking down a certain type of fat called glucocerebroside.

Clinical Trials Overview

Gaucher Outcomes Survey (GOS)

The Gaucher Outcomes Survey (GOS) is a significant ongoing observational registry that includes patients with Gaucher disease, regardless of their treatment status. This registry, which started in July 2010 and is set to continue until January 2025, aims to evaluate the safety and long-term effectiveness of velaglucerase alfa, among other objectives. It involves 1257 participants and focuses on clinical assessments and routine patient care as determined by the patients' treating physicians[1].

VPRIV Clinical Trials Program

The VPRIV clinical trials program is one of the largest for an ERT in Type 1 Gaucher disease. Here are some key points from the trials:

• Initial Studies: The safety and efficacy of VPRIV were evaluated in 99 patients across three initial clinical trials. These trials included patients aged 4 years and older, with some patients participating in a 5-year long-term extension study. The primary objectives included changes in red blood cell concentration, platelet count, and spleen and liver volumes[3][4].

• 12-Month Study: This study involved 25 adults and children, with patients receiving either 45 units/kg or 60 units/kg of VPRIV once every other week. The study showed improvements in hemoglobin levels and stability in other clinical parameters[3].

• 9-Month Comparison Study: This study compared VPRIV with imiglucerase, another ERT for Gaucher disease. It involved 34 patients and showed that VPRIV was as effective as imiglucerase in improving red blood cell count, platelet count, and reducing spleen and liver volumes[3].

• 5-Year Long-Term Extension Study: This open-label study continued the treatment with VPRIV for up to 5 years, maintaining stability in all clinical measurements such as hemoglobin and platelet counts, and spleen and liver volumes[3].

• Switch Study: The 12-month switch study evaluated the safety of switching patients from imiglucerase to VPRIV. The study found that patients maintained stability in all clinical parameters, indicating that switching to VPRIV did not compromise the efficacy or safety of the treatment[5].

Safety and Efficacy

Primary Outcomes

The clinical trials have consistently shown that VPRIV is safe and effective in treating Type 1 Gaucher disease. Key outcomes include:

• Hemoglobin Concentration: Improvements and stability in hemoglobin levels were observed across various studies[3][5].
• Platelet Count: Significant increases or stability in platelet counts were noted[3][5].
• Liver and Spleen Volumes: Reductions in liver and spleen volumes were observed, indicating effective management of the disease[3][5].

Adverse Events

The trials also monitored adverse events, including infusion-related reactions and serious adverse events. The data indicate that VPRIV has a favorable safety profile, with no significant increase in adverse events compared to other ERTs[1][3].

Market Analysis and Projections

Global Enzyme Replacement Therapy Market

The global enzyme replacement therapy market is expected to grow significantly, driven by the increasing prevalence of lysosomal storage diseases (LSDs) and the rapid regulatory approval of orphan drugs.

• Market Size: The market is projected to grow from $9.67 billion in 2021 to $15.18 billion by 2028, with a CAGR of 6.8% from 2022 to 2028[2].
• Segmentation: The market is segmented by enzyme type, therapeutic conditions, route of administration, and end-users. Velaglucerase alfa is one of the key enzymes in this market, particularly for the treatment of Gaucher's disease[2].

Competitive Landscape

The market is competitive, with several players offering enzyme replacement therapies for various LSDs. Takeda Pharmaceutical Company, the manufacturer of VPRIV, has expanded its presence in regions like India, introducing velaglucerase alfa for Gaucher disease, idursulfase for Hunter syndrome, and agalsidase alfa for Fabry disease[2].

Regional Growth

The market growth is driven by regions such as North America, Europe, and Asia Pacific, with increasing investments in rare disease therapies. The expansion of these therapies into emerging markets is also a significant factor in the market's growth[2].

Key Takeaways

• Clinical Efficacy: Velaglucerase alfa has demonstrated safety and efficacy in treating Type 1 Gaucher disease through extensive clinical trials.
• Market Growth: The global enzyme replacement therapy market is expected to grow significantly, driven by the increasing prevalence of LSDs and regulatory approvals.
• Competitive Position: VPRIV is a key player in the market, with Takeda Pharmaceutical Company expanding its reach in various regions.
• Long-Term Benefits: The long-term extension studies have shown that VPRIV maintains stability in clinical parameters, making it a reliable long-term treatment option.

FAQs

What is Velaglucerase Alfa Used For?

Velaglucerase alfa, marketed as VPRIV, is used for the long-term enzyme replacement therapy (ERT) for patients with Type 1 Gaucher disease.

How Effective is Velaglucerase Alfa in Clinical Trials?

Clinical trials have shown that velaglucerase alfa is effective in improving hemoglobin levels, increasing platelet counts, and reducing liver and spleen volumes in patients with Type 1 Gaucher disease.

What is the Expected Market Growth for Enzyme Replacement Therapy?

The global enzyme replacement therapy market is expected to grow from $9.67 billion in 2021 to $15.18 billion by 2028, with a CAGR of 6.8% from 2022 to 2028.

Who are the Key Players in the Enzyme Replacement Therapy Market?

Takeda Pharmaceutical Company, among others, is a key player in the enzyme replacement therapy market, particularly with its product VPRIV for Gaucher disease.

What are the Common Adverse Events Associated with Velaglucerase Alfa?

Common adverse events include infusion-related reactions and other treatment-emergent adverse events, but the overall safety profile of VPRIV is favorable.

Can Patients Switch from Imiglucerase to Velaglucerase Alfa?

Yes, clinical studies have shown that patients can safely switch from imiglucerase to velaglucerase alfa without compromising the efficacy or safety of the treatment.

Sources

1. Gaucher Disease Outcome Survey (GOS) - Takeda Clinical Trials

   • https://clinicaltrials.takeda.com/study-detail/5f6b5fcd4db2bf003ab4662b

2. Enzyme Replacement Therapy Market Worth $15.18 Billion by 2028 at 6.8% CAGR - Global Analysis by The Insight Partners

   • https://www.globenewswire.com/news-release/2022/07/22/2484361/0/en/Enzyme-Replacement-Therapy-Market-Worth-15-18-Billion-by-2028-at-6-8-CAGR-Led-by-Hospitals-Segment-49-Market-Share-in-2021-Global-Analysis-by-The-Insight-Partners.html

3. VPRIV® Clinical Studies | VPRIV® (velaglucerase alfa) for injection

   • https://www.vpriv.com/clinical-studies

4. VPRIV Clinical Trials Program Overview

   • https://www.hcp.vpriv.com/clinical-trials

5. Treatment-Experienced Switch Study | VPRIV® (velaglucerase alfa) for injection

   • https://www.hcp.vpriv.com/treatment-experienced/switch-study