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	149
Multiple Myeloma in Relapse	17
Refractory Multiple Myeloma	12

Condition MeSH

Condition MeSH for POMALIDOMIDE
Intervention	Trials
Multiple Myeloma	219
Neoplasms, Plasma Cell	188
Recurrence	22

Clinical Trial Locations for POMALIDOMIDE

Trials by Country

Trials by Country for POMALIDOMIDE
Location	Trials
Canada	138
Spain	118
Japan	93
China	91
France	86

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	11
PHASE2	10
PHASE1	7

Clinical Trial Status

Clinical Trial Status for POMALIDOMIDE
Clinical Trial Phase	Trials
RECRUITING	86
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	293
Other	225
NIH	33

Pomalidomide clinical trials update, market analysis, and exclusivity/competition outlook (2026)

Last updated: July 26, 2026

Pomalidomide (Pomalyst, Celgene/Bristol Myers Squibb) remains a cornerstone therapy in relapsed/refractory multiple myeloma (RRMM), with the commercial and IP position shaped by: (1) the original small-molecule estate, (2) life-cycle extensions around formulations and new combinations, and (3) the entry timing and competitive pressure from generic lenalidomide substitutes and established anti-myeloma regimens rather than a single dominant next-generation competitor.

What is the current clinical trial pipeline for pomalidomide in multiple myeloma (RRMM)?

Pomalidomide’s clinical development is focused on combination expansion and earlier-line use rather than changing the core mechanism (IMiD cereblon modulation). Trial strategy largely tracks the standard RRMM landscape: pairing with proteasome inhibitors, monoclonal antibodies, and targeted agents to deepen responses in patients who have exhausted lenalidomide.

Which combinations are most active?

Commercially meaningful expansion typically centers on these pairing archetypes:

• Pomalidomide + dexamethasone as the backbone comparator in RRMM
• Pomalidomide + anti-CD38 (e.g., daratumumab) to intensify depth and durability
• Pomalidomide + proteasome inhibitor (e.g., bortezomib/carfilzomib-class regimens) for anti-myeloma synergy
• Pomalidomide + next-gen immunotherapies in later-line trials (including T-cell redirectors and BCMA-targeted approaches in investigational settings)

What endpoints matter for market impact?

Trials that drive label updates and payer access in RRMM usually target:

• Overall response rate (ORR) and stringent complete response rates
• MRD negativity rates (where measured)
• Duration of response (DoR) and progression-free survival (PFS)
• Overall survival (OS), especially in lenalidomide-exposed or refractory subgroups
• Safety/tolerability profiles in older, heavily pretreated populations (neutropenia, infection risk, thrombosis prevention)

How to interpret “pipeline velocity” for pomalidomide?

The most market-relevant “velocity” is not the count of trials. It is the share of trials with:

• Readouts expected to support label expansions
• Randomized comparisons against accepted standards (pomalidomide backbone or alternative IMiDs plus steroids)
• Evidence in lenalidomide-refractory and high-risk cytogenetic subgroups

How strong is pomalidomide’s market position in 2024-2026 and what is the revenue sensitivity?

Pomalidomide’s revenue is structurally resilient because RRMM remains a chronic, multi-line disease with persistent demand for effective oral regimens after lenalidomide failure. The sensitivity is tied to:

• Share gains from improved regimens in real-world settings
• Formulary positioning versus alternative oral IMiDs and anti-CD38 combinations
• Adverse-event management capacity (hematologic toxicity, infection monitoring)
• Patient access and co-pay dynamics in the US and EU

Market demand drivers

• Continued need after lenalidomide refractoriness: Pomalidomide is a key post-lenalidomide option with established clinical evidence.
• Combination adoption: Anti-CD38 and proteasome inhibitor combinations can shift treatment sequencing, sustaining usage even when monotherapy becomes less dominant.
• Oral administration advantage: Facilitates adherence and outpatient management.

Market headwinds

• Competitive substitution: Other IMiDs and combination regimens can displace pomalidomide in specific lines.
• Safety/tolerability: Management of neutropenia/infection risk can constrain uptake, particularly in frail or comorbid populations.
• Pricing pressure: As generic competition and therapeutic alternatives expand, contracting and rebate pressure increase.

When does pomalidomide lose exclusivity and how much patent life is left?

Pomalidomide’s exclusivity and patent life are driven by a combination of:

• Core compound/early compositions
• Life-cycle extensions (formulation, polymorphs/solid-state, and method-of-use for specific combinations)
• Regulatory exclusivities tied to US FDA reference listed drug history (and exclusivity stacking, where applicable)

For business planning, you should treat the risk as not a single “cliff”. Entry dynamics are driven by the Orange Book patent list granularity and whether generic challengers can fully design around both active ingredient and protected use.

US exclusivity mechanics (how generic entry typically happens)

• If an FDA-listed patent is challenged via Paragraph IV, generic entry timing is controlled by the litigation and any settlement terms.
• If formulation or method-of-use patents remain unexpired, a generic may still be blocked from certain approved indications or dosing instructions even when the compound is technically off-patent.

What patents protect pomalidomide (Pomalyst) and how many are in the estate?

The patent estate for pomalidomide is commonly dense because manufacturers add incremental protections around:

• Composition of matter and salts/solid forms
• Specific dose regimens and compositions
• Formulation technologies (capsule/solid oral drug product characteristics)
• Combination therapies and method-of-use

Practical takeaway: For generic entry, the relevant question is not the total count. It is whether Orange Book-listed patents cover:

• The exact approved strength(s) and dosage regimen
• The approved indications and combinations (method-of-use)
• Any key manufacturing or formulation steps that cannot be designed around

What is the Orange Book status of pomalidomide (Pomalyst) and which patents are likely Paragraph IV targets?

In most branded IMiD products, Paragraph IV interest clusters around:

• Late-expiring formulation or method-of-use patents
• Weakly defended process patents only if the challenger expects design-around feasibility
• Combination-related patents if the brand label includes specific regimen language

The generic strategy typically aims to:

1. avoid infringing compound/formulation claims, and
2. carve out protected uses or rely on non-infringing instructions.

How does pomalidomide compare with lenalidomide and other RRMM options on competitive landscape?

Pomalidomide operates in a distinct clinical niche: lenalidomide-exposed and lenalidomide-refractory patients. Competitive pressure includes:

• Other IMiDs and next-line oral regimens
• Proteasome inhibitor-based combinations
• Anti-CD38 monoclonal antibodies and BCMA-targeted therapies

Competitive positioning summary

• Where pomalidomide is strong: post-lenalidomide RRMM, combination intensification with anti-CD38 and steroid backbones
• Where it can lose: earlier-line sequences where newer combinations and cellular/targeted therapies earn share, or where payer policies favor alternate regimens with faster escalation of deeper response

What patent litigation affects pomalidomide generics and settlements?

Pomalidomide’s litigation profile has historically followed the US generic playbook:

• Patent infringement suits following Paragraph IV challenges
• Potential settlements that trigger “at-risk” or delayed launch dates
• Ongoing disputes about claim scope and design-around

For market impact, monitor:

• Whether generic entrants secure “skinny” approvals or carve-outs
• Settlement entry dates that align with late-expiring method-of-use or formulation patents
• Post-settlement injunction dynamics, particularly around label scope

What generic entry risks exist for pomalidomide in the US and which dosing strengths are most exposed?

Generic risk is strongest where:

• Orange Book patents are fewer or expiring earlier for specific dosage forms
• Design-around is feasible for formulation claims
• Generic challengers win a non-infringement or invalidity outcome

Dose-strength exposure can differ if:

• The brand’s Orange Book coverage is strength-specific
• Specific tablet/capsule manufacturing or excipient formulation claims map to particular strengths

Which international markets pose the next biggest launch threat for pomalidomide?

Outside the US, generic entry and regulatory competition depend on:

• National patent status and enforcement strength
• Local data exclusivity regimes
• Parallel import and price competition dynamics
• EMA/National HTA formularies that influence uptake speed

High-impact geographies for commercial planning typically include EU-5 (Germany, France, Italy, Spain, UK) and key ROW markets where Indian and other generic suppliers set pricing benchmarks.

How do formulation and method-of-use patents affect competition and biosimilar risk for pomalidomide?

Pomalidomide is a small molecule; biosimilar risk does not apply. Competition risk comes from:

• Generic small molecules (composition/formulation and process design-around)
• Switching of clinical practice if other regimens supersede pomalidomide in treatment guidelines

Formulation and method-of-use patents matter because they can delay:

• Full label substitution
• Indication-by-indication switching
• Use in combination regimens where method-of-use claims exist

Clinical and regulatory milestones: what label expansions could move pomalidomide utilization?

Market-shaping label expansions tend to be:

• Earlier line RRMM settings (moving upward from later-line use)
• Specific lenalidomide-refractory subgroups
• Combination regimens that become guideline standard

The highest commercial lift typically comes from randomized data that supports broad-line positioning rather than narrow post-hoc subgroup updates.

Market projection for pomalidomide through 2028: base case, downside, and upside

A defensible projection framework for pomalidomide needs to incorporate three variables: (1) generic penetration speed, (2) durability of brand usage in combination regimens, and (3) payer-driven substitution against alternative RRMM standards.

Base case (status quo competitive substitution)

• Modest erosion driven by contracting pressure and gradual displacement by alternative regimens
• Uptake of pomalidomide combinations in eligible RRMM lines remains steady
• Revenue declines slower than pure off-patent small-molecule declines because of clinical niche persistence post-lenalidomide

Downside (faster substitution and pricing compression)

• Higher-than-expected uptake of alternative anti-CD38 + targeted regimens in earlier lines
• Increased generic/authorized generic pressure if US exclusivity weakens faster than expected
• Greater restrictions from HTA and payer policies tied to cost-effectiveness

Upside (successful trial readouts and guideline reinforcement)

• Label support for combinations that prove superior in PFS/DoR without unacceptable toxicity
• Expanded payer coverage due to clearer sequencing advantage in lenalidomide-refractory patients
• Continued growth in outpatient regimen adoption

What filings and trial readouts should be monitored to update the projection?

High-signal events for market forecasts are:

• Phase 3 readouts with OS/PFS in RRMM stratified by lenalidomide refractoriness
• FDA supplemental NDA outcomes for new combinations
• Safety profile updates that reduce discontinuation rates in older patients
• Guideline committee changes reflecting new standard-of-care regimens

Key Takeaways

• Pomalidomide’s market durability comes from its RRMM niche after lenalidomide exposure, with competition shifting toward combination-based standards rather than replacing the drug’s mechanism outright.
• The near-term clinical catalyst is combination expansion that can strengthen or broaden label positioning and payer uptake.
• Patent risk is governed by Orange Book granularity and method-of-use/formulation coverage, which affects launch timing and label scope more than compound off-patent status alone.
• Market projection hinges on generic penetration speed and payer-driven regimen switching in earlier-line RRMM.

FAQs

1. What is pomalidomide’s best-selling line of therapy in RRMM and why does it persist?
2. Which clinical trials most directly influence pomalidomide label expansion in lenalidomide-refractory patients?
3. How do Orange Book method-of-use patents delay generic substitution even after compound expiration?
4. What combination regimens are most likely to displace pomalidomide monotherapy in routine practice?
5. How should a payer formulary strategy treat pomalidomide when anti-CD38 and BCMA therapies expand?

References

1. FDA. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations (Pomalyst, pomalidomide). U.S. Food and Drug Administration.
2. FDA. Drug Approval Package and labeling for Pomalyst (pomalidomide). U.S. Food and Drug Administration.
3. ClinicalTrials.gov. Pomalidomide trials in multiple myeloma (RRMM) and combination studies. U.S. National Library of Medicine.
4. EMA. Public assessment documents and product information for pomalidomide-containing products. European Medicines Agency.