CLINICAL TRIALS PROFILE FOR VELCADE

505(b)(2) Clinical Trials for VELCADE

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT00116961 ↗	Velcade, Doxil, and Dexamethasone (VDd) as First Line Therapy for Multiple Myeloma	Completed	University of Michigan Cancer Center	Phase 2	2005-06-01	This is a research study for patients with newly diagnosed multiple myeloma. Multiple myeloma remains a non-curable disease however, newer medications and their combinations appear to provide higher response rates and higher complete response rates than current treatment options. One of the new medications in multiple myeloma is Velcade. Preliminary results from a study using a combination of Velcade with Doxil have shown high response rates (disease reduction). Preliminary results also show that an addition of dexamethasone to Velcade in patients not responding to Velcade alone showed improved response rates. This study involves treatment with a new combination of three standard medications: Velcade, Doxil, and dexamethasone (VDd combination). The proposed combination of all three drugs may improve efficacy and response. Velcade is approved by the Food and Drug Administration (FDA) for treatment in multiple myeloma patients who have received at least two prior therapies and have demonstrated disease progression on the last therapy. Velcade is still currently under investigation for other indications. Doxil is not approved for use in multiple myeloma but is an approved drug for use in patients with some other cancers. Several published clinical trials provide evidence that Doxil is an active agent in multiple myeloma and it is used in treatment combinations for multiple myeloma in general practice. Dexamethasone is a standard therapy for multiple myeloma, but is not approved by the FDA for that use. The combination of all three drugs is experimental (not FDA approved). The goals of this study are to determine if this new combination therapy with Velcade, Doxil and dexamethasone is an effective treatment and also to determine the side effects that occur when this combination treatment is given.
New Combination	NCT00116961 ↗	Velcade, Doxil, and Dexamethasone (VDd) as First Line Therapy for Multiple Myeloma	Completed	University of Michigan Rogel Cancer Center	Phase 2	2005-06-01	This is a research study for patients with newly diagnosed multiple myeloma. Multiple myeloma remains a non-curable disease however, newer medications and their combinations appear to provide higher response rates and higher complete response rates than current treatment options. One of the new medications in multiple myeloma is Velcade. Preliminary results from a study using a combination of Velcade with Doxil have shown high response rates (disease reduction). Preliminary results also show that an addition of dexamethasone to Velcade in patients not responding to Velcade alone showed improved response rates. This study involves treatment with a new combination of three standard medications: Velcade, Doxil, and dexamethasone (VDd combination). The proposed combination of all three drugs may improve efficacy and response. Velcade is approved by the Food and Drug Administration (FDA) for treatment in multiple myeloma patients who have received at least two prior therapies and have demonstrated disease progression on the last therapy. Velcade is still currently under investigation for other indications. Doxil is not approved for use in multiple myeloma but is an approved drug for use in patients with some other cancers. Several published clinical trials provide evidence that Doxil is an active agent in multiple myeloma and it is used in treatment combinations for multiple myeloma in general practice. Dexamethasone is a standard therapy for multiple myeloma, but is not approved by the FDA for that use. The combination of all three drugs is experimental (not FDA approved). The goals of this study are to determine if this new combination therapy with Velcade, Doxil and dexamethasone is an effective treatment and also to determine the side effects that occur when this combination treatment is given.
New Combination	NCT00135187 ↗	Study of Combination Therapy With VELCADE, Doxil, and Dexamethasone (VDd) in Multiple Myeloma	Completed	University of Michigan Cancer Center	N/A	2004-07-01	Patients are being asked to take part in this research study because they have multiple myeloma which has relapsed after (come back), or is refractory to (unaffected by), initial therapy. For patients who have relapsed or are refractory to therapy, there is no agreed upon standard treatment. Treatment options include chemotherapy and, for some patients, bone marrow transplants. None of the available treatments are curative and investigators are continually looking for more effective treatments. This study involves treatment with a new combination of standard drugs: VELCADE, Doxil, and Dexamethasone. Preliminary results from a study using a combination of VELCADE with Doxil showed high response rates (disease reduction). Two other studies showed that an addition of Dexamethasone to VELCADE in patients not responding to VELCADE alone improved response rate. The proposed combination of all three drugs may improve efficacy and response. VELCADE is approved by the Food and Drug Administration (FDA) for use in multiple myeloma. Doxil is not approved for use in multiple myeloma but is an approved drug for use in patients with some other cancers. Several published clinical trials provide evidence that Doxil is an active agent in multiple myeloma and it is used in treatment combinations for multiple myeloma in general practice. Dexamethasone is approved for use in multiple myeloma. The combination of all three drugs is experimental (not FDA approved). The goals of this study are to determine if this new combination therapy with VELCADE, Doxil and Dexamethasone is an effective treatment, and also to determine the side effects that occur when this combination treatment is given.
New Combination	NCT00135187 ↗	Study of Combination Therapy With VELCADE, Doxil, and Dexamethasone (VDd) in Multiple Myeloma	Completed	University of Michigan Rogel Cancer Center	N/A	2004-07-01	Patients are being asked to take part in this research study because they have multiple myeloma which has relapsed after (come back), or is refractory to (unaffected by), initial therapy. For patients who have relapsed or are refractory to therapy, there is no agreed upon standard treatment. Treatment options include chemotherapy and, for some patients, bone marrow transplants. None of the available treatments are curative and investigators are continually looking for more effective treatments. This study involves treatment with a new combination of standard drugs: VELCADE, Doxil, and Dexamethasone. Preliminary results from a study using a combination of VELCADE with Doxil showed high response rates (disease reduction). Two other studies showed that an addition of Dexamethasone to VELCADE in patients not responding to VELCADE alone improved response rate. The proposed combination of all three drugs may improve efficacy and response. VELCADE is approved by the Food and Drug Administration (FDA) for use in multiple myeloma. Doxil is not approved for use in multiple myeloma but is an approved drug for use in patients with some other cancers. Several published clinical trials provide evidence that Doxil is an active agent in multiple myeloma and it is used in treatment combinations for multiple myeloma in general practice. Dexamethasone is approved for use in multiple myeloma. The combination of all three drugs is experimental (not FDA approved). The goals of this study are to determine if this new combination therapy with VELCADE, Doxil and Dexamethasone is an effective treatment, and also to determine the side effects that occur when this combination treatment is given.

All Clinical Trials for VELCADE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00005064 ↗	PS-341 in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myeloid Leukemia in Blast Phase, or Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 1	2000-02-01	Phase I trial to study the effectiveness of PS-341 in treating patients who have refractory or relapsed acute myeloid leukemia, acute lymphoblastic leukemia, chronic myeloid leukemia in blast phase, or myelodysplastic syndrome. PS-341 may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth
NCT00006184 ↗	Chemotherapy, Stem Cell Transplantation and Donor and Patient Vaccination for Treatment of Multiple Myeloma	Completed	National Cancer Institute (NCI)	Phase 2	2001-02-08	Background: The mainstay of therapy for newly diagnosed multiple myeloma patients remains systemic chemotherapy. Although partial remissions of up to 60% are obtained with conventional regimens, multiple myeloma is essentially an incurable disease with a median survival of approximately 30 months. Allogeneic stem cell transplantation (SCT) results in a high percentage of complete remissions, but it can be associated with significant treatment-related mortality, which has been primarily attributed to conventional myeloablative transplant regimens. Recent clinical studies have shown that highly immunosuppressive yet non-myeloablative doses of fludarabine-based chemotherapy can result in alloengraftment. Even with a reduction in treatment related mortality, success with allogeneic SCT is limited by a significant risk of relapse. Donor immunization with myeloma Id in the setting of a non-myeloablative allogeneic SCT may represent a novel strategy for the treatment of multiple myeloma. Objectives: Primary Objectives: To induce cellular and humoral immunity in allogeneic stem cell donors and recipients against the unique idiotype expressed by the recipient's myeloma. To determine whether antigen-specific immunity, induced in the stem cell donor, can be passively transferred to the allogeneic SCT recipient in the setting of a non-myeloablative conditioning regimen. Secondary Objectives: To evaluate the effect of the Fludarabine-(etoposide, doxorubicin, vincristine, prednisone, cyclophosphamide) EPOCH regimen on host T cell depletion and myeloid depletion prior to allogeneic SCT. To determine the efficacy of a novel conventional chemotherapy regimen (Fludarabine-EPOCH) in the setting multiple myeloma. To determine the treatment-related morbidity and mortality of allogeneic stem cell transplantation using a non-myeloablative conditioning regimen in multiple myeloma. To determine if the re-vaccination of allogeneic stem cell donors with the unique idiotype expressed by the recipient's myeloma will enhance cellular and humoral immunity to patient specific-idiotype prior to lymphocyte donation for the treatment of patients with recurrent or progressive disease after transplantation. Eligibility: Patients 18-75 years of age with Immunoglobulin G (IgG) or Immunoglobulin A (IgA) multiple myeloma. Patients must have achieved at least a partial remission following initial conventional chemotherapy regimen or after autologous stem cell transplantation. Consenting first degree relative matched at 6/6 or 5/6 human leukocyte antigen (HLA) antigens. Design: Phase 2 trial using a non-myeloablative conditioning regimen to reduce treatment-related toxicity. Recipient will undergo a plasmapheresis to obtain starting material for the isolation of idiotype protein. Donors would be immunized with an Id vaccine prepared from the patient. Prior to transplantation patients would receive a conventional chemotherapy regimen which contains agents active in myeloma and is T cell depleting. The allogeneic SCT would be performed with a conditioning regimen consisting of cyclophosphamide and fludarabine. The stem cell source would be blood mobilized with filgrastim. Recipients will be immunized with the Id vaccine following transplantation.
NCT00006773 ↗	Bortezomib in Treating Patients With Recurrent Glioma	Terminated	National Cancer Institute (NCI)	Phase 1	2001-05-01	Phase I trial to study the effectiveness of bortezomib in treating patients who have recurrent glioma. Bortezomib may stop the growth of tumor cells by blocking the enzymes necessary for tumor cell growth
NCT00007878 ↗	Bortezomib, Fluorouracil, and Leucovorin Calcium in Treating Patients With Solid Tumors That Are Metastatic or Cannot Be Removed By Surgery	Completed	National Cancer Institute (NCI)	Phase 1	2000-09-01	This phase I trial studies the side effects and best dose of bortezomib when given together with fluorouracil and leucovorin calcium in treating patients with solid tumors that are metastatic or cannot be removed by surgery. Bortezomib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fluorouracil and leucovorin calcium, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving bortezomib together with chemotherapy may be an effective treatment for solid tumors.

Clinical Trial Conditions for VELCADE

Condition Name

Condition Name for VELCADE
Intervention	Trials
Multiple Myeloma	219
Lymphoma	29
Mantle Cell Lymphoma	18
Unspecified Adult Solid Tumor, Protocol Specific	17

Condition MeSH

Condition MeSH for VELCADE
Intervention	Trials
Multiple Myeloma	290
Neoplasms, Plasma Cell	277
Lymphoma	114
Leukemia	55

Clinical Trial Locations for VELCADE

Trials by Country

Trials by Country for VELCADE
Location	Trials
Canada	105
Spain	79
Germany	73
France	71
Italy	50

Trials by US State

Trials by US State for VELCADE
Location	Trials
California	100
Texas	85
New York	84
Massachusetts	79
Florida	77

Clinical Trial Progress for VELCADE

Clinical Trial Phase

Clinical Trial Phase for VELCADE
Clinical Trial Phase	Trials
PHASE4	1
PHASE2	1
Phase 4	13

Clinical Trial Status

Clinical Trial Status for VELCADE
Clinical Trial Phase	Trials
Completed	331
Terminated	107
Active, not recruiting	52

Clinical Trial Sponsors for VELCADE

Sponsor Name

Sponsor Name for VELCADE
Sponsor	Trials
National Cancer Institute (NCI)	151
Millennium Pharmaceuticals, Inc.	136
M.D. Anderson Cancer Center	26

Sponsor Type

Sponsor Type for VELCADE
Sponsor	Trials
Other	604
Industry	391
NIH	157

Velcade (Bortezomib): Clinical Trials Update, Market Analysis, and Future Projection

Last updated: October 28, 2025

Introduction

Velcade (bortezomib) stands as a cornerstone in the treatment of multiple myeloma and mantle cell lymphoma (MCL). Approved initially by the FDA in 2003 for multiple myeloma, Velcade has undergone extensive clinical validation and continues to evolve within therapeutic protocols. As the global oncology landscape advances with targeted therapies and immunotherapies, understanding Velcade's current clinical status, market dynamics, and future outlook is crucial for pharmaceutical stakeholders, investors, and healthcare providers.

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Clinical Trials Update

Ongoing and Recent Trials

Recent clinical research highlights Velcade's expanding therapeutic applications and combination regimens. The ClinicalTrials.gov database lists over 50 active initiatives involving Velcade, emphasizing its ongoing relevance. Notable recent trials include:

• Combination Therapies in Multiple Myeloma: Multiple phase III studies examine Velcade in combination with immunomodulatory drugs such as lenalidomide and pomalidomide. For instance, the CASSIOPEIA trial evaluated Velcade plus daratumumab with standard therapy, showing promising progression-free survival (PFS) benefits [1].

• First-line Treatments: Trials such as VICTOR (Velcade-based induction therapy) compare Velcade plus dexamethasone versus other regimens, seeking to optimize upfront therapy options.

• Novel Delivery Methods: Research explores subcutaneous administration (already FDA-approved in 2012), alongside efforts investigating sustained-release formulations and nano-delivery systems to improve patient adherence and minimize side effects.

• Expanded Indications: Trials are evaluating Velcade in other hematologic malignancies, including Waldenström’s macroglobulinemia and certain solid tumors, reflecting its versatility.

Emerging Data and Evidence

The latest phase III data reinforce Velcade’s role in relapsed/refractory multiple myeloma (RRMM). The APEX trial demonstrated improved overall response rates (ORR) and PFS compared to conventional therapies—a benchmark that established Velcade’s prominence [2].

Moreover, combination regimens pairing Velcade with novel agents such as selinexor or monoclonal antibodies continue to show efficacy, aligning with the shift toward personalized and precision oncology.

Regulatory and Approval Developments

Beyond initial approvals, regulatory agencies, including the European Medicines Agency (EMA), have approved indications for new combinations. The gene-therapy landscape's emergence emphasizes the need for Velcade’s integrations into combination protocols to sustain its market relevance.

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Market Analysis

Market Size and Revenue Trends

Velcade remains a leading drug within the hematology-oncology market. As per IQVIA data, the global Velcade market generated approximately $1.2 billion USD in 2022, with a compound annual growth rate (CAGR) near 4.5% over the past five years [3].

Geographical Market Dynamics

• North America: Dominates due to established healthcare infrastructure and high diagnosis rates of multiple myeloma (~34,000 cases annually). The US accounts for approximately 60% of Velcade sales, driven by reimbursement policies and clinical adoption.

• Europe: Significant market share, especially in Germany, France, and the UK, with incremental growth fueled by expanded indications and combination therapies.

• Asia-Pacific: Exhibits rapid growth, attributable to increasing healthcare access and awareness. Emerging markets like China and India are witnessing higher Velcade utilization, projected to grow at CAGR exceeding 7% through 2027.

Commercial Factors Influencing the Market

• Pricing and Reimbursement: Velcade’s high cost (~$7,000-$10,000 per treatment cycle) influences access, with reimbursement variations affecting sales. Biosimilar development is gaining momentum, potentially reducing prices and expanding patient access.

• Competitive Landscape: The market features rival proteasome inhibitors such as carfilzomib and ixazomib, with Velcade maintaining dominance due to its extensive clinical data and earlier approval.

• Innovation and Label Expansion: Ongoing trials for Velcade in new indications and as part of combination regimens bolster market stability and growth prospects.

Patent and Biosimilar Impact

Velcade’s patent expired in 2014 in the US, prompting biosimilar entries (e.g., Velcade biosimilars in India), which threaten pricing power but also facilitate broader access in emerging markets. Nonetheless, brand loyalty, extensive clinical backing, and ongoing patent protections for certain formulations sustain its market position.

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Future Market Projection

Growth Drivers

• Expanding Indications: Approval for additional hematologic malignancies and solid tumors predicted to contribute significantly to sales.

• Patient Demographics: Rising global incidence of multiple myeloma, especially in aging populations, underpins future demand.

• Combination Regimens: The trend towards multi-agent therapy, incorporating Velcade, enhances its position and cuts across various treatment lines, from first-line to salvage settings.

• New Formulations and Delivery Systems: Subcutaneous formulations have improved tolerability, increasing adherence and expanding eligible patient populations.

Challenges and Risks

• Competition from Orally Administered Agents: Oral proteasome inhibitors (e.g., ixazomib) offer convenience, threatening Velcade’s market share.

• Biosimilar Era: Biosimilar proliferation may pressure prices and profitability unless Velcade’s manufacturer adopts strategic measures.

• Resistance and Relapse Rates: Disease progression and resistance impact long-term sustainability; ongoing trials aim to mitigate these by combination strategies.

Projected Revenue Growth

Market analysts project Velcade’s global revenues will stabilize around $1.0-$1.3 billion USD annually through the next five years, with potential upticks driven by new indications and combination protocols. The CAGR is forecasted at approximately 3-5%, reflecting moderate growth in a competitive landscape.

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Conclusion

Velcade remains a pivotal molecule in hematologic oncology, with ongoing clinical trials reinforcing its therapeutic versatility. While market expansion faces headwinds from biosimilars and oral alternatives, strategic combination therapies and new formulations promise sustained revenues. Continued innovation and expansion into emergent indications will be critical for maintaining Velcade’s market leadership.

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Key Takeaways

• Velcade’s clinical pipeline emphasizes combination regimens and expanded indications, reinforcing its role in hematologic malignancy treatment.

• The global Velcade market is sizable and growing steadily, driven by aging populations, increased diagnosis, and regimen innovations.

• Biosimilar competition and alternative oral proteasome inhibitors present challenges but also offer opportunities for strategic positioning.

• Future growth hinges on regulatory approvals for new indications, improved formulations, and positioning within personalized medicine frameworks.

• Stakeholders should monitor emerging trial data and biosimilar developments to adapt strategies effectively.

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FAQs

1. What are the primary indications approved for Velcade?
Velcade is primarily approved for multiple myeloma and mantle cell lymphoma. Its use in combination therapies has expanded to include relapsed/refractory cases and as part of initial treatment regimens.

2. How does Velcade compare with newer proteasome inhibitors like carfilzomib?
Velcade has a longer clinical track record, with extensive data supporting its efficacy and tolerability. Carfilzomib shows higher potency and may have fewer peripheral neuropathy risks but faces similar competition; choice depends on patient profile and treatment setting.

3. What impact do biosimilars have on Velcade’s market share?
Biosimilars in certain markets pose pricing pressure, potentially reducing Velcade’s revenue. However, brand loyalty and clinical confidence sustain its market presence, especially in regions lacking biosimilar approval.

4. Are there any notable ongoing clinical trials that could expand Velcade’s indications?
Yes, trials investigating Velcade in solid tumors, amyloidosis, and other hematologic cancers are ongoing, with some promising early-phase results that could broaden its therapeutic scope.

5. What strategic measures can stakeholders adopt to maximize Velcade’s market potential?
Stakeholders should focus on expanding combination protocols, pursuing new indications, developing improved delivery systems, and engaging in partnerships to innovate therapies integrating Velcade.

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References

[1] ClinicalTrials.gov. "Evaluation of Velcade in Combination Regimens."
[2] Richardson PG, et al. "Phase III Results of Bortezomib in Relapsed Multiple Myeloma." J Clin Oncol. 2005.
[3] IQVIA. "Global Oncology Market Report 2022."