CLINICAL TRIALS PROFILE FOR POMALIDOMIDE

505(b)(2) Clinical Trials for POMALIDOMIDE

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	NCT02103335 ↗	Combination Study of Pomalidomide, Marizomib, and Low-Dose Dexamethasone in Relapsed and Refractory Multiple Myeloma	Completed	Celgene Corporation	Phase 1	2014-06-05	This is a Phase 1 clinical trial to evaluate a new combination of drugs for the treatment of relapsed or refractory (drug-resistant) multiple myeloma. The drugs being studied are: - Pomalidomide (POMALYST®) is a drug that affects the immune system (an immunomodulatory drug) that has been approved by the United States (US) Food and Drug Administration (FDA) for the treatment of multiple myeloma. - Marizomib is an investigational drug being developed by Triphase that is being studied for the treatment of multiple myeloma. Investigational drugs are drugs that have not yet been approved by health authorities, such as the FDA, for general use but have been approved for use in specific clinical studies. Marizomib inhibits a cellular machine called the proteasome, which destroys unnecessary or damaged proteins. Other proteasome inhibitors have been shown to be effective in the treatment of multiple myeloma. - Dexamethasone is a corticosteroid drug that affects the immune system (an immunomodulatory drug) that has been approved by the FDA for the treatment of multiple myeloma. This is the first study to evaluate the three-drug combination of pomalidomide (POM), marizomib (MRZ), and dexamethasone (LD-DEX) in humans. Pomalidomide, alone or in combination with dexamethasone, is approved by the FDA for the treatment of relapsed or refractory multiple myeloma. The primary objective of this study is to determine the best drug dosing levels for this three-drug combination, including the highest safe doses and/or the recommended doses for future clinical studies of this drug combination. The secondary purposes of this study are to determine the safety of this drug combination and its effectiveness in treating relapsed or refractory multiple myeloma. The study will include examination of levels of all three drugs in the blood during various time points during treatment.
New Combination	NCT02103335 ↗	Combination Study of Pomalidomide, Marizomib, and Low-Dose Dexamethasone in Relapsed and Refractory Multiple Myeloma	Completed	Celgene	Phase 1	2014-06-05	This is a Phase 1 clinical trial to evaluate a new combination of drugs for the treatment of relapsed or refractory (drug-resistant) multiple myeloma. The drugs being studied are: - Pomalidomide (POMALYST®) is a drug that affects the immune system (an immunomodulatory drug) that has been approved by the United States (US) Food and Drug Administration (FDA) for the treatment of multiple myeloma. - Marizomib is an investigational drug being developed by Triphase that is being studied for the treatment of multiple myeloma. Investigational drugs are drugs that have not yet been approved by health authorities, such as the FDA, for general use but have been approved for use in specific clinical studies. Marizomib inhibits a cellular machine called the proteasome, which destroys unnecessary or damaged proteins. Other proteasome inhibitors have been shown to be effective in the treatment of multiple myeloma. - Dexamethasone is a corticosteroid drug that affects the immune system (an immunomodulatory drug) that has been approved by the FDA for the treatment of multiple myeloma. This is the first study to evaluate the three-drug combination of pomalidomide (POM), marizomib (MRZ), and dexamethasone (LD-DEX) in humans. Pomalidomide, alone or in combination with dexamethasone, is approved by the FDA for the treatment of relapsed or refractory multiple myeloma. The primary objective of this study is to determine the best drug dosing levels for this three-drug combination, including the highest safe doses and/or the recommended doses for future clinical studies of this drug combination. The secondary purposes of this study are to determine the safety of this drug combination and its effectiveness in treating relapsed or refractory multiple myeloma. The study will include examination of levels of all three drugs in the blood during various time points during treatment.
New Combination	NCT02103335 ↗	Combination Study of Pomalidomide, Marizomib, and Low-Dose Dexamethasone in Relapsed and Refractory Multiple Myeloma	Completed	Triphase Research and Development I Corporation	Phase 1	2014-06-05	This is a Phase 1 clinical trial to evaluate a new combination of drugs for the treatment of relapsed or refractory (drug-resistant) multiple myeloma. The drugs being studied are: - Pomalidomide (POMALYST®) is a drug that affects the immune system (an immunomodulatory drug) that has been approved by the United States (US) Food and Drug Administration (FDA) for the treatment of multiple myeloma. - Marizomib is an investigational drug being developed by Triphase that is being studied for the treatment of multiple myeloma. Investigational drugs are drugs that have not yet been approved by health authorities, such as the FDA, for general use but have been approved for use in specific clinical studies. Marizomib inhibits a cellular machine called the proteasome, which destroys unnecessary or damaged proteins. Other proteasome inhibitors have been shown to be effective in the treatment of multiple myeloma. - Dexamethasone is a corticosteroid drug that affects the immune system (an immunomodulatory drug) that has been approved by the FDA for the treatment of multiple myeloma. This is the first study to evaluate the three-drug combination of pomalidomide (POM), marizomib (MRZ), and dexamethasone (LD-DEX) in humans. Pomalidomide, alone or in combination with dexamethasone, is approved by the FDA for the treatment of relapsed or refractory multiple myeloma. The primary objective of this study is to determine the best drug dosing levels for this three-drug combination, including the highest safe doses and/or the recommended doses for future clinical studies of this drug combination. The secondary purposes of this study are to determine the safety of this drug combination and its effectiveness in treating relapsed or refractory multiple myeloma. The study will include examination of levels of all three drugs in the blood during various time points during treatment.

All Clinical Trials for POMALIDOMIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00463385 ↗	A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia	Completed	Celgene	Phase 2	2007-04-01	The purpose of this study is to determine the safety of and to select a treatment regimen of pomalidomide (CC-4047) either as single-agent or in combination with prednisone to study further in patients with myelofibrosis with myeloid metaplasia (MMM).
NCT00463385 ↗	A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia	Completed	Celgene Corporation	Phase 2	2007-04-01	The purpose of this study is to determine the safety of and to select a treatment regimen of pomalidomide (CC-4047) either as single-agent or in combination with prednisone to study further in patients with myelofibrosis with myeloid metaplasia (MMM).
NCT00537511 ↗	A Phase I/II Study to Determine the Maximum Tolerated Dose (MTD) and Safety of CC-4047 (Pomalidomide) Administered in Conjunction With Cisplatin and Etoposide	Terminated	Celgene	Phase 1/Phase 2	2008-02-01	The purpose of this study is to determine the maximum tolerated dose and safety of CC-4047 (pomalidomide) given in combination with cisplatin and etoposide in patients with extensive disease small cell lung cancer.

Clinical Trial Conditions for POMALIDOMIDE

Condition Name

Condition Name for POMALIDOMIDE
Intervention	Trials
Multiple Myeloma	148
Multiple Myeloma in Relapse	17
Refractory Multiple Myeloma	12

Condition MeSH

Condition MeSH for POMALIDOMIDE
Intervention	Trials
Multiple Myeloma	217
Neoplasms, Plasma Cell	188
Recurrence	21

Clinical Trial Locations for POMALIDOMIDE

Trials by Country

Trials by Country for POMALIDOMIDE
Location	Trials
Canada	138
Spain	118
Japan	93
China	90
France	85

Trials by US State

Trials by US State for POMALIDOMIDE
Location	Trials
New York	73
California	71
Texas	59
Massachusetts	55
Florida	54

Clinical Trial Progress for POMALIDOMIDE

Clinical Trial Phase

Clinical Trial Phase for POMALIDOMIDE
Clinical Trial Phase	Trials
PHASE3	10
PHASE2	8
PHASE1	7

Clinical Trial Status

Clinical Trial Status for POMALIDOMIDE
Clinical Trial Phase	Trials
Recruiting	85
Completed	58
Active, not recruiting	49

Clinical Trial Sponsors for POMALIDOMIDE

Sponsor Name

Sponsor Name for POMALIDOMIDE
Sponsor	Trials
Celgene	60
Celgene Corporation	53
National Cancer Institute (NCI)	33

Sponsor Type

Sponsor Type for POMALIDOMIDE
Sponsor	Trials
Industry	290
Other	223
NIH	33

Clinical Trials Update, Market Analysis, and Projection for Pomalidomide

Last updated: October 28, 2025

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Introduction

Pomalidomide (brand names: Pomalyst, Imnovid) is an immunomodulatory agent primarily used to treat multiple myeloma, particularly in relapsed or refractory cases. As a derivative of thalidomide, it belongs to the class of immunomodulatory drugs (IMiDs) and exhibits anti-angiogenic, anti-inflammatory, and immunostimulatory properties. The evolving landscape of multiple myeloma therapeutics, combined with ongoing clinical research, shapes the strategic outlook for Pomalidomide.

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Clinical Trials Update

Current Clinical Development Landscape

Pomalidomide has undergone extensive Phase I, II, and III trials to establish efficacy, safety, and optimal dosing regimens. Its initial approval by the FDA in 2013 for relapsed and refractory multiple myeloma was based on pivotal trial data demonstrating significant response rates when combined with dexamethasone.[^1]

Recent clinical efforts focus on expanding indications, optimizing combination therapies, and exploring novel formulations. Key ongoing and completed trials include:

• Combination with Monoclonal Antibodies: Multiple trials assess Pomalidomide with daratumumab and elotuzumab, capitalizing on synergistic immunomodulatory effects. For example, NCT02951443 investigates Pomalidomide with daratumumab in relapsed multiple myeloma, aiming to improve overall response rates.[^2]

• Novel Formulations: Efforts are underway to develop sustained-release formulations to mitigate adverse events and improve patient compliance.

• Expanded Indications: Early-phase studies are evaluating Pomalidomide's efficacy in other hematologic malignancies, such as myelodysplastic syndromes and certain lymphomas.

Recent Trial Results and Observations

• The EMN12/MM-011 trial demonstrated that the addition of Pomalidomide to standard regimens elicits durable responses with manageable toxicity profiles.[^3]

• A phase II study (NCT03418789) exploring Pomalidomide and ixazomib combination yielded promising progression-free survival (PFS) metrics, indicating potential breadth in multiple myeloma management strategies.

• Safety profiles remain consistent with known immunomodulatory effects: neutropenia, thrombocytopenia, thromboembolic events, and peripheral neuropathy are commonly reported adverse events.[^4]

Market Analysis

Market Size and Growth Drivers

The global multiple myeloma therapeutics market was valued at approximately USD 10.5 billion in 2022 and is projected to grow at a compound annual growth rate (CAGR) of about 9% through 2030, driven by increasing incidence rates, improved diagnostics, and expanding treatment indications.[^5]

Pomalidomide secures a significant share, primarily in the relapsed/refractory segment due to its proven efficacy. Factors influencing market growth include:

• Rising Prevalence: According to the American Cancer Society, the incidence of multiple myeloma has increased by about 1.8% annually.[^6]

• Advancement in Combination Regimens: Growing evidence supports combination therapies involving Pomalidomide, expanding its clinical application.

• Patent and Regulatory Landscape: Patent expirations may influence generic manufacturing; however, strategic drug formulations and combination partnerships sustain commercial interest.

Competitive Landscape

Pomalidomide faces competition from other IMiDs, notably lenalidomide and thalidomide, alongside novel agents like CAR-T therapies (e.g., idecabtagene vicleucel) and bispecific antibodies. Nonetheless, its unique efficacy in refractory cases sustains its clinical relevance.[^7]

Emerging competitors incorporate Pomalidomide in combination protocols and investigational therapies. Clinical data favoring its safety and efficacy in specific patient subsets preserve its market position.

Regulatory Developments and Approvals

In addition to FDA approval for relapsed multiple myeloma, Pomalidomide holds approvals in several markets, including the European Union and Japan, with ongoing applications for expanded indications. This regulatory footprint underscores global acceptance and sets the stage for broader market penetration.

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Market Projection

Forecasting Pomalidomide's Trajectory

The outlook for Pomalidomide remains optimistic, characterized by:

• Increasing Adoption: As combination therapies demonstrate enhanced efficacy, Pomalidomide's utilization is expected to deepen, especially in refractory cases.

• Pipeline Success: Promising results from ongoing trials targeting multiple myeloma and other hematological malignancies could extend its market lifespan and expand indications.

• Manufacturing and Patent Strategies: Patent expirations in certain regions may catalyze generic competition, potentially reducing costs and expanding access, thus influencing overall sales volume.

Challenges and Opportunities

• Pricing and Reimbursements: Cost pressures and reimbursement policies influence prescribing behaviors. Competitive pricing strategies and demonstrating value through clinical benefits are critical.

• Resistance and Toxicity Concerns: Resistance development and adverse event profiles necessitate ongoing research and patient management strategies to sustain market share.

• Emerging Therapies: The advent of cellular therapies and bispecific antibodies might compress the market over the next decade, demanding continuous innovation.

Projected Sales Growth

Based on current trends, strategic clinical developments, and market forecasts, Pomalidomide’s global sales are projected to grow at a CAGR of approximately 7-8% over the next five years (2023–2028), reaching an estimated USD 3.5–4 billion by 2028.[^8] The growth trajectory will largely depend on approval expansions, competitive dynamics, and clinical trial success.

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Key Takeaways

• Robust Clinical Data: Pomalidomide continues to demonstrate efficacy and manageable safety profiles in relapsed multiple myeloma, with ongoing trials exploring expanded combinations and indications.

• Market Positioning: Its established role in refractory multiple myeloma sustains strong demand, supported by rising disease prevalence and combination regimen evidence.

• Growth Opportunities: Advancements in combination therapies and novel formulations, coupled with expanding regulatory approval, underpin future growth potential.

• Competitive Landscape: While facing competition from other IMiDs and emerging therapies, Pomalidomide's targeted niche in refractory cases ensures relevant positioning.

• Strategic Considerations: Cost management, resistance mitigation, and innovation in drug delivery remain central to maintaining market relevance amid evolving treatment paradigms.

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FAQs

1. What are the primary clinical indications for Pomalidomide?
It is approved primarily for relapsed or refractory multiple myeloma after failure of two prior therapies, including lenalidomide and bortezomib.

2. How does Pomalidomide differ from other IMiDs like lenalidomide?
Pomalidomide has demonstrated superior efficacy in certain refractory populations and exhibits distinct pharmacokinetic properties, although both share similar mechanisms of immunomodulation.

3. What are the common adverse effects associated with Pomalidomide?
Neutropenia, thrombocytopenia, fatigue, peripheral neuropathy, and increased risk of thromboembolism are typical side effects.

4. Are there ongoing trials exploring new uses of Pomalidomide?
Yes, ongoing trials are investigating its potential in other hematologic malignancies and combination regimens to broaden therapeutic options.

5. What is the outlook for Pomalidomide’s market presence in the next decade?
The outlook remains favorable, with anticipated growth driven by combination therapy approvals, pipeline advancements, and expanded indications, despite competitive pressures from emerging modalities.

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References

1. [1] FDA Approval Letter, Pomalidomide, 2013.
2. [2] ClinicalTrials.gov, NCT02951443.
3. [3] European Myeloma Network Study, EMN12/MM-011, 2021.
4. [4] Sarker, SI et al., “Safety Profile of Pomalidomide,” Hematology Reports, 2018.
5. [5] MarketWatch, "Global Multiple Myeloma therapeutics Market Size & Forecast," 2022.
6. [6] American Cancer Society, “Cancer Facts & Figures: Multiple Myeloma,” 2022.
7. [7] Kumar, SK et al., “Emerging Treatments in Multiple Myeloma,” Blood, 2021.
8. [8] MarketResearch.com, "Pomalidomide Market Outlook," 2023.

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This comprehensive analysis aims to guide pharmaceutical executives, investors, and healthcare strategists in understanding Pomalidomide’s current clinical landscape and future market prospects.