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.

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

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

Bortezomib: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

Introduction

Bortezomib, marketed as Velcade®, is a pioneering proteasome inhibitor primarily approved for multiple myeloma and mantle cell lymphoma. Since its approval in 2003, bortezomib has revolutionized hematological cancer treatment. As the drug's development landscape evolves, an up-to-date analysis of ongoing clinical trials, market trends, and future projections is essential for stakeholders assessing its global impact and growth potential.

---

Clinical Trials Landscape: Current Status and Advancements

Ongoing and Recent Clinical Trials

The clinical trial environment for bortezomib has evolved beyond its initial indications, exploring diverse oncologic areas, combination therapies, and novel formulations. As per ClinicalTrials.gov data, over 150 trials involving bortezomib are active or recruiting globally.

• Multiple Myeloma and Lymphomas: The bulk of trials (approximately 60%) focuses on refractory or relapsed multiple myeloma, often assessing combinations with immunomodulatory drugs (IMiDs), monoclonal antibodies, or novel agents. For example, a phase III trial (NCT03376959) evaluates bortezomib combined with daratumumab in relapsed/refractory multiple myeloma (RRMM).

• Novel Indications: Investigations include trials in solid tumors such as pancreatic, gastric, and breast cancers, aiming to leverage proteasome inhibition in broader oncologic contexts. While early phase, these trials indicate ongoing scientific interest.

• Combination Therapies: Trials increasingly explore synergistic use with agents like carfilzomib, melphalan, and emerging immunotherapies. Notably, phase II studies examine bortezomib with checkpoint inhibitors, signaling a trend toward immunomodulation.

• Formulation Innovations: The advent of subcutaneous (SC) formulations has led to phase IV trials assessing safety, efficacy, and patient quality of life. For instance, a study (NCT02293630) compares SC versus IV bortezomib.

Emerging Data and Innovations

Recent updates include:

• Enhanced Dosing Schedules: Trials are evaluating once-weekly dosing strategies to optimize efficacy and reduce side effects.

• Biomarker-driven Approaches: Trials integrating genomic and proteomic profiling aim to identify responders, advancing personalized therapy paradigms.

• Oral Formulations: Developing oral proteasome inhibitors, though not bortezomib itself, has prompted investigations into relative efficacy and safety, potentially impacting future use.

Regulatory and Label Expansion Trials

While the drug's primary label remains for hematologic malignancies, trials exploring off-label uses, such as autoimmune conditions (e.g., multiple sclerosis), are ongoing but in early phases.

---

Market Analysis: Current and Future Dynamics

Market Overview and Key Players

Bortezomib remains a market leader within proteasome inhibitors but faces increasing competition from newer agents such as carfilzomib and ixazomib. In 2022, the global Bortezomib market was valued at approximately $2.5 billion, with projected annual growth of 4.5% over the next five years[1].

Major pharmaceutical companies involved include:

• Takeda Pharmaceuticals: Vertically integrated, initial patent holder.
• Millennium/Takeda: Continues to dominate the market.
• Novel Entrants: Emerging competitors aim to improve efficacy and safety profiles.

Market Drivers

• Prevalence of Multiple Myeloma and Lymphomas: Increasing incidence, especially with aging populations.
• Combination Therapy Approvals: Expanding indications and broader use in treatment regimens bolster revenue.
• Patient Preference for Subcutaneous Formulations: Enhanced tolerability increases adherence, incentivizing formulations conducive to outpatient settings.

Market Challenges

• Patent Expiry and Generics: Patent expiration, expected around 2027, will lead to generic competition, substantially reducing prices.
• Side Effect Profile: Neurotoxicity and thrombocytopenia limit dosages; newer agents with better safety profiles threaten market share.
• Competition from Next-gen Proteasome Inhibitors: Carfilzomib and ixazomib are gaining approvals, offering alternative mechanisms of action, broader administration options, and improved safety profiles.

---

Future Projections: Growth Opportunities and Challenges

Market Growth Outlook

Despite impending patent expirations, the bortezomib market is projected to sustain moderate growth, driven by:

• Expanding Indications: Trials for solid tumors and autoimmune diseases could open new revenue streams.
• Ageing Demographics: Increasing prevalence of multiple myeloma and lymphomas with age sustains demand.
• Formulation Improvements: Subcutaneous and oral formulations enhance patient compliance.

Forecasts estimate a compound annual growth rate (CAGR) of approximately 3.8% through 2030, reaching a market value of $4 billion[2].

Innovation and Pipeline Opportunities

• Biosimilars and Generics: Entry post-patent expiry will likely lead to significant price reductions and wider accessibility.
• Combination Regimens: Integration into multi-drug regimens will sustain demand, especially as clinical trial data support superior efficacy.
• Personalized Therapy: Biomarker-driven approaches and companion diagnostics could optimize treatment selection, boosting bortezomib adoption.

Potential Barriers

• Competitive Displacement: Next-generation proteasome inhibitors, with improved safety and convenience profiles, threaten initial market dominance.
• Regulatory Hurdles: Expanding indications require robust trial data and regulatory approvals, which can be costly and time-consuming.
• Side Effect Management: Neurotoxicity remains a critical concern; innovations mitigating these effects could influence market dynamics.

---

Conclusion

Bortezomib continues to be a cornerstone in hematologic cancer therapeutics, with ongoing clinical trials expanding its potential uses and optimizing its delivery. Marketwise, it maintains a strong position, albeit facing competition from newer agents and coming generics. Strategic adaptation involving combination therapies, formulation innovations, and personalized medicine approaches will determine its trajectory in the evolving oncology landscape.

---

Key Takeaways

• Clinical Trials Drive Innovation: Continuous trials are assessing bortezomib's efficacy in diverse indications, including combination regimens and novel formulations, informing future therapeutic strategies.

• Market Resilience Amidst Competition: Despite the upcoming patent expiry and emerging competitors, bortezomib remains significant, supported by a strong base in multiple myeloma treatment and ongoing regimen expansions.

• Generics and Biosimilars Will Shape Pricing and Access: Post-patent loss, market penetration of lower-cost versions will influence revenue streams and accessibility.

• Formulation and Biomarker Innovation Are Critical: Subcutaneous administration and biomarker-driven therapies will enhance patient compliance and treatment personalization.

• Future Growth Will Depend on Regulatory Approvals and Clinical Evidence: Robust data supporting expanded indications and improved safety profiles are essential for sustained market relevance.

---

FAQs

Q1: What are the primary clinical indications for bortezomib?
A1: Bortezomib is primarily indicated for multiple myeloma and mantle cell lymphoma, especially in relapsed or refractory cases.

Q2: Are there ongoing trials exploring bortezomib's use in solid tumors?
A2: Yes, several early-phase trials are investigating bortezomib in pancreatic, gastric, and breast cancers, although these are not yet approved indications.

Q3: How will patent expirations affect the market for bortezomib?
A3: Patent expiry around 2027 will likely introduce biosimilars and generics, leading to decreased prices and increased competition.

Q4: What are the main side effects limiting bortezomib's use?
A4: Neurotoxicity and thrombocytopenia are common limiting toxicities, though formulation improvements have helped mitigate these issues.

Q5: What trends are shaping the future of bortezomib in oncology?
A5: Trends include combination therapies with immunotherapies, development of subcutaneous and oral formulations, and personalized treatment approaches based on biomarkers.

---

References

[1] MarketResearch.com, "Global Proteasome Inhibitors Market," 2022.
[2] Grand View Research, "Proteasome Inhibitors Market Size & Trends," 2022.