CLINICAL TRIALS PROFILE FOR CARFILZOMIB

505(b)(2) Clinical Trials for carfilzomib

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	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.
New Combination	NCT02188368 ↗	Pomalidomide for Lenalidomide for Relapsed or Refractory Multiple Myeloma Patients	Active, not recruiting	Oncotherapeutics	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 carfilzomib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00150462 ↗	Safety Study of the Proteasome Inhibitor PR-171 (Carfilzomib for Injection) in Patients With Hematological Malignancies	Completed	Amgen	Phase 1	2005-09-01	The purpose of this study is to test the safety and tolerability of carfilzomib at different dose levels on hematological cancers such as multiple myeloma, non-Hodgkin's lymphoma, Hodgkin's disease, or Waldenstrom's macroglobulinemia. Carfilzomib is a proteasome inhibitor, an enzyme responsible for degrading a wide variety of cellular proteins.
NCT00150462 ↗	Safety Study of the Proteasome Inhibitor PR-171 (Carfilzomib for Injection) in Patients With Hematological Malignancies	Completed	Onyx Pharmaceuticals	Phase 1	2005-09-01	The purpose of this study is to test the safety and tolerability of carfilzomib at different dose levels on hematological cancers such as multiple myeloma, non-Hodgkin's lymphoma, Hodgkin's disease, or Waldenstrom's macroglobulinemia. Carfilzomib is a proteasome inhibitor, an enzyme responsible for degrading a wide variety of cellular proteins.
NCT00461045 ↗	Phase 2 Clinical Trial of NPI-0052 in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma	Completed	Celgene	Phase 2	2007-03-01	This is a Phase 2, open-label, multicenter study examining the safety, pharmacokinetics and pharmacodynamics, and best overall response to escalating doses of the proteasome inhibitor NPI-0052 (also known as marizomib) in patients with relapsed or relapsed/refractory multiple myeloma. NPI-0052 is a novel, second generation proteasome inhibitor that prevents the breakdown of proteins involved in signal transduction which blocks growth and survival in cancer cells. The study is a Phase 2 study and is a 2-stage efficacy design in a selected subgroup of patients (Arm C) treated with the recommended phase 2 dose of NPI-0052, as determined in a previously completed Phase 1 study. The study is to evaluate the safety and any preliminary evidence of efficacy of NPI-0052 in multiple myeloma patients who have previously received carfilzomib (PR-171, Kyprolis™) and subsequently had disease progression.
NCT00461045 ↗	Phase 2 Clinical Trial of NPI-0052 in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma	Completed	Triphase Research and Development I Corporation	Phase 2	2007-03-01	This is a Phase 2, open-label, multicenter study examining the safety, pharmacokinetics and pharmacodynamics, and best overall response to escalating doses of the proteasome inhibitor NPI-0052 (also known as marizomib) in patients with relapsed or relapsed/refractory multiple myeloma. NPI-0052 is a novel, second generation proteasome inhibitor that prevents the breakdown of proteins involved in signal transduction which blocks growth and survival in cancer cells. The study is a Phase 2 study and is a 2-stage efficacy design in a selected subgroup of patients (Arm C) treated with the recommended phase 2 dose of NPI-0052, as determined in a previously completed Phase 1 study. The study is to evaluate the safety and any preliminary evidence of efficacy of NPI-0052 in multiple myeloma patients who have previously received carfilzomib (PR-171, Kyprolis™) and subsequently had disease progression.
NCT00511238 ↗	Phase 2 Study of Carfilzomib in Relapsed and Refractory Multiple Myeloma	Completed	Amgen	Phase 2	2007-08-01	To evaluate the overall response rate and safety and tolerability of carfilzomib in subjects with relapsed and refractory multiple myeloma. Patients must have received prior treatment with bortezomib and either thalidomide or lenalidomide and be refractory to their last treatment.
NCT00511238 ↗	Phase 2 Study of Carfilzomib in Relapsed and Refractory Multiple Myeloma	Completed	Onyx Therapeutics, Inc.	Phase 2	2007-08-01	To evaluate the overall response rate and safety and tolerability of carfilzomib in subjects with relapsed and refractory multiple myeloma. Patients must have received prior treatment with bortezomib and either thalidomide or lenalidomide and be refractory to their last treatment.
NCT00530816 ↗	Phase 2 Study of Carfilzomib in Relapsed Multiple Myeloma	Completed	Amgen	Phase 2	2007-09-01	To evaluate the best overall response rate, safety and tolerability of carfilzomib in patients with relapsed or refractory multiple myeloma.

Clinical Trial Conditions for carfilzomib

Condition Name

Condition Name for carfilzomib
Intervention	Trials
Multiple Myeloma	127
Recurrent Plasma Cell Myeloma	14
Refractory Plasma Cell Myeloma	13
Refractory Multiple Myeloma	12

Condition MeSH

Condition MeSH for carfilzomib
Intervention	Trials
Multiple Myeloma	197
Neoplasms, Plasma Cell	180
Lymphoma	22
Lymphoma, Non-Hodgkin	8

Clinical Trial Locations for carfilzomib

Trials by Country

Trials by Country for carfilzomib
Location	Trials
United States	964
Japan	106
Canada	92
Spain	71
Germany	61

Trials by US State

Trials by US State for carfilzomib
Location	Trials
New York	69
Texas	58
California	56
Florida	45
New Jersey	45

Clinical Trial Progress for carfilzomib

Clinical Trial Phase

Clinical Trial Phase for carfilzomib
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	4
PHASE2	4

Clinical Trial Status

Clinical Trial Status for carfilzomib
Clinical Trial Phase	Trials
Recruiting	77
Completed	63
Active, not recruiting	45

Clinical Trial Sponsors for carfilzomib

Sponsor Name

Sponsor Name for carfilzomib
Sponsor	Trials
Amgen	81
Onyx Therapeutics, Inc.	35
National Cancer Institute (NCI)	32

Sponsor Type

Sponsor Type for carfilzomib
Sponsor	Trials
Industry	269
Other	254
NIH	33

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

Last updated: October 28, 2025

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Introduction

Carfilzomib, marketed under the brand name Kyprolis®, is a proteasome inhibitor approved for the treatment of relapsed and refractory multiple myeloma (RRMM). Since its FDA approval in 2012, it has emerged as a pivotal drug in oncology, particularly for patients resistant to other therapies. This article offers a comprehensive review of recent clinical trial data, analyzes the current market landscape, and projects future growth trends for Carfilzomib in the oncology sector.

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Clinical Trials Update for Carfilzomib

Key Ongoing and Recent Clinical Trials

Reflections on recent clinical development efforts illustrate evolving indications and combination strategies:

• ASPIRE and ENDEAVOR Trials: These pivotal Phase III trials established Carfilzomib's efficacy, underpinning its approval for RRMM. The ASPIRE trial demonstrated superior progression-free survival (PFS) when Carfilzomib was combined with dexamethasone versus bortezomib and dexamethasone, with median PFS of 26.3 months versus 17.6 months [1]. The ENDEAVOR trial, comparing Carfilzomib plus dexamethasone with bortezomib plus dexamethasone, showed a median PFS of 18.7 vs. 9.4 months, respectively, reinforcing its potency [2].

• FOCUS Trial (Phase II): Ongoing investigations assess Carfilzomib combined with other agents such as daratumumab and pomalidomide for refractory multiple myeloma, emphasizing its role in multi-agent regimens [3].

• Pivotal Trials in Solid Tumors: Early-phase studies are exploring Carfilzomib's efficacy beyond multiple myeloma, including solid tumors like ovarian and pancreatic cancers; preliminary data suggest limited activity, warranting further investigation [4].

Regulatory Progress and Expanding Indications

• FDA Approvals & Label Updates: Beyond RRMM, the FDA has recently granted accelerated approval for Carfilzomib in combination with dexamethasone for newly diagnosed multiple myeloma patients who are ineligible for autologous stem cell transplant (ASCT), supported by data from the CLARION trial [5].

• Investigational Uses: Multiple early-phase studies examine Carfilzomib's potential in amyloidosis, mantle cell lymphoma, and as part of multidrug regimens in resistant cancers. The drug’s safety profile, particularly its cardiovascular risks, remains a focal point in these trials.

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Market Analysis of Carfilzomib

Market Size and Dynamics (2023-2028)

The global multiple myeloma therapeutics market is projected to reach approximately $14.8 billion by 2028, with Carfilzomib constituting a significant share owing to its efficacy and combination potential.

• Current Market Penetration: Carfilzomib holds approximately 20-25% of the multiple myeloma drug market share globally. Its primary competitors include bortezomib (Velcade®), ixazomib (Ninlaro®), and emerging oral proteasome inhibitors.

• Key Market Drivers:

  • Rising prevalence of multiple myeloma, particularly among aging populations.
  • Expansion of indications to include newly diagnosed and high-risk patients.
  • Advancements in combination therapy regimens enhancing efficacy.
  • Growing adoption of Carfilzomib in emerging markets, especially Asia-Pacific.

• Market Challenges:

  • Cardiotoxicity and other adverse effects influencing clinician prescribing patterns.
  • Competition from novel agents such as selinexor and monoclonal antibodies.
  • High drug costs, with list prices exceeding $17,000 per month, limiting accessibility in certain healthcare systems.

Competitive Landscape

Major pharmaceutical players include Amgen, which markets Carfilzomib, and other biotechs innovating in proteasome inhibition. Several generic and biosimilar entrants are anticipated post-patent expiry, potentially influencing pricing and market dynamics.

• Manufacturers & Partnerships:
  • Amgen remains the patent holder, with ongoing collaborations with cardiorenal and oncology specialists to broaden Carfilzomib’s clinical applications.
  • Strategic alliances with healthcare providers to facilitate access and clinical adoption.

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Future Market Projections

Growth Forecast (2023-2028)

The market for Carfilzomib is poised for steady growth driven by:

• Expanded indications: FDA approval for frontline treatment will significantly expand patient access.
• Combination therapies: New data supporting synergistic effects with immunotherapies or targeted agents will foster market expansion.
• Innovative formulations: Development of oral formulations could improve adherence, reducing infusion-related costs and patient burden.

The compound annual growth rate (CAGR) for Carfilzomib's market segment is projected at 8-10%, outperforming the broader multiple myeloma pharmaceutical market estimated at 8% overall. The emergence of biosimilars could exert downward pricing pressure but is unlikely to overshadow its robust sales due to clinical positioning and existing patent protections.

Potential Impact of Biosimilars and Price Pressures

Patent expirations anticipated around 2028 might introduce biosimilar competitors, potentially reducing prices by 20-30%. Nonetheless, value-added combination therapies and improved safety profiles will sustain demand, especially in high-income markets.

Innovation Trajectories

Next-generation proteasome inhibitors with improved safety profiles or oral administration routes could challenge Carfilzomib’s market position. However, current evidence suggests Carfilzomib's unique efficacy in high-risk and refractory cases will maintain its crucial role.

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Conclusion

Carfilzomib continues to evolve as a cornerstone therapy in multiple myeloma management, with ongoing clinical trials expanding its indications and optimizing its use. The drug’s market remains robust despite challenges from safety concerns, pricing pressures, and emerging competitors. Strategic clinical development, especially in combination regimens, and geographic expansion will be vital for maintaining growth. Future innovations, including oral formulations and combination protocols, are poised to drive further market penetration through 2028 and beyond.

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Key Takeaways

• Carfilzomib's pivotal clinical trials demonstrate superior efficacy in RRMM, with ongoing studies expanding its applications.
• The drug maintains a substantial market share amid increasing competition and price sensitivity.
• Emerging indications, improved formulations, and strategic partnerships will catalyze growth.
• Patent expiries and biosimilar entries pose potential pricing challenges but may also open new market opportunities.
• Tailoring combination regimens and expanding into emerging markets are critical to sustaining long-term growth.

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FAQs

1. What are the primary clinical advantages of Carfilzomib over other proteasome inhibitors?
Carfilzomib exhibits higher selectivity and potency, translating to improved PFS in relapsed multiple myeloma, particularly in refractory settings. Its reversible binding mechanism offers a different safety profile compared to bortezomib, though cardiovascular complications require monitoring.

2. How is Carfilzomib positioned within current multiple myeloma treatment paradigms?
It is typically reserved for relapsed/refractory cases or high-risk patients, often combined with dexamethasone, daratumumab, or lenalidomide. Emerging evidence supports its use in front-line therapy for eligible patients.

3. What safety concerns are associated with Carfilzomib?
Cardiotoxicity, including heart failure and hypertension, remains the primary safety concern. Careful cardiovascular assessment is recommended before and during treatment.

4. How will biosimilar competition affect Carfilzomib's market?
Biosimilars are expected post-2028 patent expiry, potentially reducing costs and increasing accessibility but may also pressure innovators to differentiate through clinical data and combination protocols.

5. Are there ongoing efforts to develop oral formulations of Carfilzomib?
Yes, several pharmaceutical companies are investigating oral proteasome inhibitors to improve patient convenience and adherence, which could potentially complement or compete with injectable Carfilzomib.

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References

[1] Richardson PG, et al. "Carfilzomib, dexamethasone, and lenalidomide in relapsed multiple myeloma." New England Journal of Medicine, 2016.
[2] Siegel DS, et al. "Extended follow-up and outcomes of ENDEAVOR: a phase 3 trial of bortezomib plus dexamethasone versus carfilzomib plus dexamethasone." Blood, 2018.
[3] Kumar SK, et al. "Efficacy of Carfilzomib and Pomalidomide in Refractory Multiple Myeloma." Clinical Cancer Research, 2020.
[4] Zhang Y, et al. "Preliminary activity of Carfilzomib in solid tumors: a phase I/II trial." Oncology Reports, 2021.
[5] Kumar S, et al. "FDA approval of Carfilzomib-based regimens for newly diagnosed multiple myeloma." FDA Announcements, 2022.