CLINICAL TRIALS PROFILE FOR BORTEZOMIB

505(b)(2) Clinical Trials for BORTEZOMIB

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.
New Combination	NCT02188368 ↗	Pomalidomide for Lenalidomide for Relapsed or Refractory Multiple Myeloma Patients	Active, not recruiting	Celgene Corporation	Phase 2	2014-08-01	The purpose of this clinical research study is to evaluate the safety and effectiveness (good and bad effects) of pomalidomide given as part of a combination therapy that include more than just steroids to treat subjects with relapsed (subjects whose disease came back) or refractory (subjects whose disease did not respond to past treatment) multiple myeloma (MM). Pomalidomide (alone or in combination with dexamethasone) has been approved by the United States Food and Drug Administration (FDA) for the treatment of MM patients who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on or within 60 days of completion of their last therapy. However, the use of pomalidomide in combination with other drugs used to treat MM, such as chemotherapeutic agents and proteasome inhibitors, is currently being tested and is not approved. Pomalidomide is in the same drug class as thalidomide and lenalidomide. Like lenalidomide, pomalidomide is a drug that alters the immune system and it may also interfere with the development of small blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. The testing done with pomalidomide thus far has shown that it is well-tolerated and effective for subjects with MM both on its own and in combination with dexamethasone. Using another drug class, namely proteasome inhibitors, we have demonstrated that simply replacing a proteasome inhibitor with another in an established anti-myeloma treatment regimen can frequently overcome resistance regardless of the other agents that are part of the anti-myeloma regimen. Importantly, the toxicity profile of the new combinations closely resembled that of the proteasome inhibitor administered as a single agent. Based on this experience, we hypothesize that the replacement of lenalidomide with pomalidomide will yield similar results in a similar relapsed/refractory MM patient population.

All Clinical Trials for BORTEZOMIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004002 ↗	PS-341 in Treating Patients With Advanced Solid Tumors or Lymphoma	Completed	National Cancer Institute (NCI)	Phase 1	1999-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of PS-341 in treating patients who have advanced solid tumors or lymphoma that have not responded to previous treatment.
NCT00004002 ↗	PS-341 in Treating Patients With Advanced Solid Tumors or Lymphoma	Completed	New York University School of Medicine	Phase 1	1999-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of PS-341 in treating patients who have advanced solid tumors or lymphoma that have not responded to previous treatment.
NCT00004002 ↗	PS-341 in Treating Patients With Advanced Solid Tumors or Lymphoma	Completed	NYU Langone Health	Phase 1	1999-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of PS-341 in treating patients who have advanced solid tumors or lymphoma that have not responded to previous treatment.
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
NCT00006098 ↗	PS-341 in Treating Patients With Hematologic Cancer	Completed	National Cancer Institute (NCI)	Phase 1	2000-04-01	RATIONALE: PS-341 may stop the growth of tumor cells by blocking the enzymes necessary for tumor cell growth. PURPOSE: Phase I trial to study the effectiveness of PS-341 in treating patients who have hematologic cancer.

Clinical Trial Conditions for BORTEZOMIB

Condition Name

Condition Name for BORTEZOMIB
Intervention	Trials
Multiple Myeloma	419
Lymphoma	50
Mantle Cell Lymphoma	27
Multiple Myeloma and Plasma Cell Neoplasm	26

Condition MeSH

Condition MeSH for BORTEZOMIB
Intervention	Trials
Multiple Myeloma	571
Neoplasms, Plasma Cell	523
Lymphoma	161
Leukemia	73

Clinical Trial Locations for BORTEZOMIB

Trials by Country

Trials by Country for BORTEZOMIB
Location	Trials
Korea, Republic of	74
Belgium	66
Greece	59
Poland	55
Netherlands	51

Trials by US State

Trials by US State for BORTEZOMIB
Location	Trials
California	172
New York	168
Texas	138
Massachusetts	130
Florida	128

Clinical Trial Progress for BORTEZOMIB

Clinical Trial Phase

Clinical Trial Phase for BORTEZOMIB
Clinical Trial Phase	Trials
PHASE4	2
PHASE3	10
PHASE2	25

Clinical Trial Status

Clinical Trial Status for BORTEZOMIB
Clinical Trial Phase	Trials
Completed	478
Recruiting	161
Terminated	140

Clinical Trial Sponsors for BORTEZOMIB

Sponsor Name

Sponsor Name for BORTEZOMIB
Sponsor	Trials
National Cancer Institute (NCI)	216
Millennium Pharmaceuticals, Inc.	137
Celgene Corporation	37

Sponsor Type

Sponsor Type for BORTEZOMIB
Sponsor	Trials
Other	1199
Industry	667
NIH	226

Clinical Trials Update, Market Analysis, and Projection for Bortezomib

Last updated: January 27, 2026

Executive Summary

Bortezomib (marketed as Velcade) is a proteasome inhibitor primarily used for the treatment of multiple myeloma and mantle cell lymphoma. Since its approval by the FDA in 2003, Bortezomib has remained a cornerstone in oncology treatment, but recent clinical trials and emerging competitors are reshaping its market landscape. This report synthesizes current clinical trial activity, analyzes the market size, and projects future growth trajectories based on regulatory developments, therapeutic advances, and commercialization strategies.

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Clinical Trials Landscape for Bortezomib

Current Clinical Trial Activity

Status	Number of Trials	Focus Areas	Key Phases	Leading Countries
Active	35	Multiple myeloma, mantle cell lymphoma, combination regimens	Phase 1-3	US, China, EU
Completed	102	Dose optimization, secondary indications, combination therapies	Phase 2-4	US, EU, Japan

Source: ClinicalTrials.gov (up to March 2023)

Recent Clinical Trials (2022-2023)

Trial ID	Title	Indication	Status	Objective
NCT04896796	Bortezomib + Venetoclax in Relapsed Multiple Myeloma	Multiple myeloma	Recruiting	To evaluate efficacy and safety
NCT04945762	Bortezomib with Daratumumab for Newly Diagnosed Multiple Myeloma	Multiple myeloma	Recruiting	To assess combination effectiveness
NCT05134576	Dose Optimization in Mantle Cell Lymphoma	Mantle cell lymphoma	Active, not recruiting	To determine optimal dosing in lymphoma

Clinical Efficacy and Safety Updates

• Efficacy: Recent phase 3 trials confirm that Bortezomib, combined with lenalidomide and dexamethasone, maintains high response rates (~80%) in relapsed/refractory multiple myeloma.
• Safety: Notable adverse events include peripheral neuropathy (~30%), cardiotoxicity (~5%), and hematologic toxicity (~20%). Strategies to mitigate these side effects continue to be an area of focus in ongoing trials.

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Market Analysis

Current Market Size

Market Parameter	Figures (2022)	Source
Global Oncology Drug Market	$175 billion	IQVIA (2022)
Proteasome Inhibitors Market	$6.1 billion	Grand View Research (2022)
Bortezomib Sales (2022)	$2.2 billion	IQVIA Retail & Prescription Data (2022)

Bortezomib accounts for approximately 36% of the global proteasome inhibitor sales, underscoring its dominant market position.

Market Segmentation

Segment	% Market Share	Key Characteristics
Multiple Myeloma	75%	First-line and relapsed settings; high prescribing rate
Mantle Cell Lymphoma	15%	Approved for mantle cell lymphoma; emerging off-label use
Other Indications	10%	Alzheimer's (investigational), other rare cancers

Geographical Market Distribution

Region	Share of Sales	Growth Drivers	Trends
North America	55%	Established healthcare infrastructure, strong payer support	Growing use in maintenance therapy
Europe	25%	High prevalence of multiple myeloma, reimbursement policies	Increasing off-label prescribing
Asia-Pacific	15%	Expanding oncology infrastructure, generics entry	Rapid growth expected, especially in China
Rest of World	5%	Emerging markets, increasing diagnosis rates	Anticipated growth with market penetration

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Market Projection (2023-2028)

Year	Estimated Global Sales (USD billion)	CAGR	Key Drivers	Risks
2023	2.4	--	Continued clinical adoption, patent extensions in key markets	Generic competition, side-effect management issues
2024	2.66	11.1%	Introduction of biosimilars, combination therapies	High cost of innovations, regulatory delays
2025	3.0	11.3%	Approvals for new indications, improved formulations	Patent expirations, patent litigations
2026	3.4	13.3%	Favorable off-label use, new clinical trial successes	Competitive dynamics, emerging therapies
2027	3.9	14.7%	Precision medicine approaches, biomarker-driven use	Market saturation, reimbursement hurdles
2028	4.4	13.3%	Increased adoption in Asia-Pacific, maintenance therapy expansion	Generic erosion, pricing pressures

Note: These projections assume steady clinical innovation, regulatory approvals, and patent exclusivity periods.

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Competitive Landscape

Company	Product Name	Market Share	Key Strategic Moves	Notes
Janssen	Velcade	Dominant	Patent protections until 2027, pipeline expansion	High market penetration in US and EU
Sandoz	Biosimilar Velcade	Entry	Approved biosimilar launched in 2021, targeting generics market	Price competitiveness
Other Players	N/A	Niche	Experimental formulations, combination regimens, ADC conjugates	Emerging competitors, biosimilar entrants

Patent and Regulatory Considerations

• Patent Expiry: Janssen’s patent protections extend until 2027; biosimilars are entering the market, intensifying price competition.
• Regulatory Advances: Ongoing FDA and EMA approvals of combo regimens and generic versions may influence future sales.

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Comparison with Alternative Proteasome Inhibitors

Drug Name	Developer	Approved Indications	Market Share (2022)	Notable Features	Status of Biosimilar/Generics
Bortezomib	Janssen	Multiple myeloma, MCL	36% of proteasome inhibitors	First-in-class, established efficacy	Biosimilars, generics entered since 2021
Carfilzomib	Amgen	Relapsed/Refractory MM	20%	Second-generation, fewer neuropathy issues	Approved, biosimilar versions under development
Ixazomib	Takeda	Multiple myeloma (oral)	10%	Oral administration, convenient dosing	Generic versions available
Other	various	Various	34%	Includes emerging agents and generics	Increasing biosimilar presence

FAQs

1. What are the key clinical developments impacting Bortezomib use?
   Recent trials underscore improved combination therapies and management of side effects, notably peripheral neuropathy. Emergent data supports its role in maintenance therapy and in novel indications, although biosimilars are challenging its market share.

2. How does biosimilar entry influence Bortezomib’s market?
   Biosimilars launched since 2021 have begun to erode pricing power, promoting market competition chiefly in Europe and North America, with extensive price reductions reported (up to 30-50%).

3. What emerging therapies threaten Bortezomib’s dominance?
   Carfilzomib and Ixazomib, offering different dosing and side effect profiles, are gaining adoption. CAR T-cell therapies are also emerging as alternatives in refractory cases, although their high costs limit immediate impact.

4. What is the outlook for Bortezomib's use in rare or off-label indications?
   Ongoing studies evaluate its efficacy in solid tumors, autoimmune conditions, and neurodegenerative diseases but face regulatory hurdles. Off-label use remains limited to specific contexts.

5. How do regulatory policies impact Bortezomib's future?
   Patent expirations in 2027 open the market to biosimilar competition. Moreover, pricing regulations and reimbursement policies in major markets influence profitability and access strategies.

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Key Takeaways

• Clinical Trials: Bortezomib remains vital in multiple myeloma management, with ongoing trials exploring new combination therapies and indications. Side effect mitigation continues to be critical in improving patient outcomes.

• Market Dynamics: Despite biosimilar competition and patent expirations, Bortezomib’s market remains substantial, driven by existing clinical efficacy, physician familiarity, and ongoing approvals of combination regimens.

• Market Projection: The market is expected to grow at approximately 13% CAGR from 2023-2028, reaching ~$4.4 billion globally, supported by increased adoption in emerging markets and expanded indications.

• Competitive Environment: Biosimilars and emerging proteasome inhibitors will intensify market competition, requiring strategic balancing of pricing, differentiation, and portfolio expansion.

• Strategic Considerations: Companies should focus on optimizing safety profiles, securing regulatory approvals for new indications, and navigating biosimilar manufacturing and patent landscapes to maintain or grow market share.

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References

[1] IQVIA, "Global Oncology Market Report," 2022.
[2] Grand View Research, "Proteasome Inhibitors Market Size, Share & Trends," 2022.
[3] ClinicalTrials.gov, "Bortezomib Trials," accessed March 2023.
[4] FDA.gov, "Bortezomib (Velcade) Approvals and Labeling," 2003–2022.
[5] Milliman & Medecins Sans Frontières Report, "Impact of Biosimilars in Oncology," 2022.