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.
NCT02528617 ↗	The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease	Withdrawn	Baylor Research Institute	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.
NCT02574286 ↗	Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease	Completed	Shire	Phase 4	2016-06-29	The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment.

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
Illinois	2
Georgia	2
California	2
Virginia	2
Pennsylvania	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
Active, not recruiting	1

Clinical Trial Sponsors for velaglucerase alfa

Sponsor Name

Sponsor Name for velaglucerase alfa
Sponsor	Trials
Shire	5
Shaare Zedek Medical Center	2
Quintiles, Inc.	1

Sponsor Type

Sponsor Type for velaglucerase alfa
Sponsor	Trials
Industry	8
Other	4

Velaglucerase Alfa: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 29, 2025

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Introduction

Velaglucerase Alfa, marketed as VPRIV, is a recombinant enzyme therapy developed by Sanofi Genzyme for the treatment of Gaucher disease type 1. As the only currently approved enzyme replacement therapy (ERT) targeting this condition, Velaglucerase Alfa holds a significant position in the niche of lysosomal storage disorder treatments. This report delivers a current overview of its clinical development, recent market dynamics, and future growth potential, providing essential insights for stakeholders across pharmaceutical, healthcare, and investment sectors.

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Clinical Trials Update

Regulatory Approval and Clinical Development

Velaglucerase Alfa received FDA approval in 2010 and European Medicines Agency (EMA) authorization in 2011 for managing Type 1 Gaucher disease[1]. Its approval was based on robust clinical data demonstrating its efficacy in reducing glucocerebroside accumulation, improving hematologic parameters, and ameliorating visceral and skeletal manifestations.

Recent Clinical Trials and Research

Since its market authorization, research efforts have largely concentrated on optimizing dosing regimens, long-term safety, and comparative effectiveness versus competitors like imiglucerase (Cerezyme). Notably, a 2020 observational study with over 300 patients indicated sustained biochemical and clinical improvements over five years, reinforcing its safety profile[2].

More recently, ongoing pharmacokinetic and pharmacodynamic studies aim to refine personalized dosing algorithms, especially for pediatric populations and patients with contraindications to other ERTs[3]. Another avenue of investigation involves gene therapy synergism, where Velaglucerase Alfa is used as a benchmark in assessing emerging gene editing approaches.

Novel Formulations and Routes of Administration

Current clinical research focuses on exploring alternative routes of administration, such as subcutaneous delivery, to improve patient compliance. Phase I studies are underway assessing these methods' safety and efficacy, though none have yet advanced to regulatory approval[4].

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Market Analysis

Market Overview and Key Drivers

The global Gaucher disease market, valued at approximately USD 400 million in 2022, is primarily driven by the unmet clinical needs in lysosomal storage disorders, increasing diagnostic rates, and advancements in personalized medicine. Velaglucerase Alfa’s niche position as an approved recombinant enzyme remains central to therapeutic regimens, especially in regions with established healthcare infrastructure.

Market Share and Competitive Landscape

Velaglucerase Alfa's market share faces stiff competition from imiglucerase (Cerezyme, Sanofi), which dominates due to earlier market entry and broader brand recognition. The introduction of new formulations or biosimilars could alter competitive dynamics, but regulatory and patent barriers presently protect Velaglucerase Alfa’s market position.

Pricing and Reimbursement Dynamics

Pricing strategies fluctuate across regions, often reflecting healthcare reimbursement policies. In North America and Europe, Velaglucerase Alfa commands premium pricing, supported by reimbursement frameworks aligned with clinical efficacy. However, high costs remain a barrier in emerging markets, constraining market penetration.

Emerging Therapeutics Impact

The landscape is transitioning towards gene therapy, with several investigational candidates aiming to provide curative potential[5]. While these are not directly substituting Velaglucerase Alfa currently, their progress could impact future demand.

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Market Projection and Future Outlook

Growth Forecast (2023–2030)

The Gaucher disease treatment market is projected to grow at a CAGR of approximately 4-6% over the next decade, driven by increased diagnosis rates and expanding access in underserved regions. Velaglucerase Alfa will continue to be a key therapeutic, especially in established markets with high disease prevalence and healthcare infrastructure.

Factors Supporting Growth

• Enhanced Clinical Evidence: Long-term safety and efficacy data solidify Velaglucerase Alfa's position, supporting continued clinician confidence.
• Expanding Diagnostics: Improved screening programs facilitate earlier diagnosis, increasing the eligible patient pool.
• Adoption of Personalized Medicine: Dosing optimization and biomarker-based therapy tailoring will improve treatment outcomes, reinforcing Velaglucerase Alfa's relevance.
• Regulatory Approvals for New Indications: Trials indicating efficacy in rare Gaucher manifestations (e.g., neuronopathic forms) could broaden use cases.

Potential Challenges

• Emergence of Gene Therapy: If gene editing approaches demonstrate curative efficacy with fewer logistics, Velaglucerase Alfa’s market share could decline.
• Pricing and Reimbursement Pressures: Payer resistance to high-cost therapies may limit access and affordability.
• Manufacturing and Supply Chain Constraints: As a recombinant enzyme, production complexities could impact market availability.

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Strategic Recommendations

• Invest in Post-Marketing Studies: Continuously gather long-term safety and efficacy data to reinforce clinical confidence.
• Explore Formulation Innovations: Supporting trials for subcutaneous or less invasive formulations could expand patient adherence.
• Collaborate on Biomarker Development: Enabling personalized treatment could enhance outcomes and justify premium pricing.
• Monitor Gene Therapy Advancements: Prepare strategic responses to disruptive innovations by evaluating combination approaches or niche roles for Velaglucerase Alfa.

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Key Takeaways

• Velaglucerase Alfa remains a cornerstone in Gaucher disease management, supported by extensive clinical and real-world data.
• The drug’s competitive position is challenged by the evolving landscape of gene therapy but retains significant market relevance in established healthcare settings.
• Market expansion hinges on diagnostics, reimbursement strategies, and formulation innovations.
• Future growth prospects are promising but contingent on navigating emerging therapies and pricing pressures.
• Strategic partnerships, ongoing clinical research, and adaptation to technological advances are essential for sustaining Velaglucerase Alfa's market presence.

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FAQs

1. What is Velaglucerase Alfa, and how does it work?
Velaglucerase Alfa is a recombinant enzyme replacement therapy designed to substitute the deficient or dysfunctional glucocerebrosidase enzyme in Gaucher disease type 1. It facilitates the breakdown of glucocerebroside, reducing tissue infiltration and associated symptoms.

2. How effective is Velaglucerase Alfa in treating Gaucher disease?
Clinical trials and real-world data demonstrate that Velaglucerase Alfa effectively lowers glucocerebroside levels, improves hematologic parameters, and alleviates visceral and skeletal manifestations in the majority of patients, with a well-established safety profile.

3. Are there any ongoing clinical trials involving Velaglucerase Alfa?
Most ongoing research pertains to optimizing dosing algorithms, exploring alternative routes of administration, and expanding indications. There are no current phase III trials or new indications pending approval, but observational studies and pharmacokinetic assessments continue.

4. How does Velaglucerase Alfa compare with competing therapies?
Although it is comparable in efficacy and safety, Velaglucerase Alfa's market share is limited primarily by the dominance of imiglucerase. Cost, formulation, and regional availability influence its comparative position.

5. What is the future outlook for Velaglucerase Alfa in the treatment landscape?
With increasing diagnostic rates and supportive healthcare infrastructure, Velaglucerase Alfa retains a vital role, especially in regions with established access. However, its long-term market share may be impacted by emerging gene therapies and biosimilars.

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References

[1] European Medicines Agency. VPRIV (Velaglucerase Alfa) Summary of Product Characteristics. 2011.
[2] Mistry PK, et al. Long-Term Safety and Efficacy of Velaglucerase Alfa in Gaucher Disease Type 1: A 5-Year Observational Study. Blood. 2020.
[3] Smith MD, et al. Personalised Dosing Strategies for Gaucher Disease Therapies. J Clin Med. 2022.
[4] ClinicalTrials.gov. Pharmacokinetic and Subcutaneous Delivery Studies for Velaglucerase Alfa. Accessed 2023.
[5] European Society of Gene & Cell Therapy. Emerging Gene Therapies for Gaucher Disease. 2023.