CLINICAL TRIALS PROFILE FOR ROMIDEPSIN

505(b)(2) Clinical Trials for romidepsin

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	NCT01755975 ↗	Romidepsin in Combination With Lenalidomide in Adults With Relapsed or Refractory Lymphomas and Myeloma	Active, not recruiting	Biologics, Inc.	Phase 1/Phase 2	2012-12-01	The treatments used to treat lymphoma and multiple myeloma sometimes do not always work well or they may only work for a short period of time. This is why new treatments are being tested. This study will test a new combination of two drugs that are already approved by the Food and Drug Administration for treatment of certain kinds of blood cancers. These drugs are romidepsin and lenalidomide. Both these drugs by themselves have been used to treat lymphoma or multiple myeloma. However, while these drugs are routinely used alone, this is the first time they will be tested together. The mechanism of action of both drugs is not well understood but both have been shown to to be effective by themselves in lymphoma and multiple myeloma.
New Combination	NCT01755975 ↗	Romidepsin in Combination With Lenalidomide in Adults With Relapsed or Refractory Lymphomas and Myeloma	Active, not recruiting	Celgene Corporation	Phase 1/Phase 2	2012-12-01	The treatments used to treat lymphoma and multiple myeloma sometimes do not always work well or they may only work for a short period of time. This is why new treatments are being tested. This study will test a new combination of two drugs that are already approved by the Food and Drug Administration for treatment of certain kinds of blood cancers. These drugs are romidepsin and lenalidomide. Both these drugs by themselves have been used to treat lymphoma or multiple myeloma. However, while these drugs are routinely used alone, this is the first time they will be tested together. The mechanism of action of both drugs is not well understood but both have been shown to to be effective by themselves in lymphoma and multiple myeloma.
New Combination	NCT01755975 ↗	Romidepsin in Combination With Lenalidomide in Adults With Relapsed or Refractory Lymphomas and Myeloma	Active, not recruiting	Saint Francis/Mount Sinai Regional Cancer Center	Phase 1/Phase 2	2012-12-01	The treatments used to treat lymphoma and multiple myeloma sometimes do not always work well or they may only work for a short period of time. This is why new treatments are being tested. This study will test a new combination of two drugs that are already approved by the Food and Drug Administration for treatment of certain kinds of blood cancers. These drugs are romidepsin and lenalidomide. Both these drugs by themselves have been used to treat lymphoma or multiple myeloma. However, while these drugs are routinely used alone, this is the first time they will be tested together. The mechanism of action of both drugs is not well understood but both have been shown to to be effective by themselves in lymphoma and multiple myeloma.
New Combination	NCT01755975 ↗	Romidepsin in Combination With Lenalidomide in Adults With Relapsed or Refractory Lymphomas and Myeloma	Active, not recruiting	University of Nebraska	Phase 1/Phase 2	2012-12-01	The treatments used to treat lymphoma and multiple myeloma sometimes do not always work well or they may only work for a short period of time. This is why new treatments are being tested. This study will test a new combination of two drugs that are already approved by the Food and Drug Administration for treatment of certain kinds of blood cancers. These drugs are romidepsin and lenalidomide. Both these drugs by themselves have been used to treat lymphoma or multiple myeloma. However, while these drugs are routinely used alone, this is the first time they will be tested together. The mechanism of action of both drugs is not well understood but both have been shown to to be effective by themselves in lymphoma and multiple myeloma.

All Clinical Trials for romidepsin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00007345 ↗	Depsipeptide to Treat Patients With Cutaneous T-Cell Lymphoma and Peripheral T-Cell Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	2001-03-08	Background: NSC630176 is a depsipeptide fermentation product from Chromobacterium violaceum with potent cytotoxic activity against human tumor cell lines and in vivo efficacy against both human tumor xenografts and murine tumors (1-3). NSC 630176, herein referred to as depsipeptide, shows a lack of cross resistance with several commonly used cytotoxic agents such as vincristine, 5-fluorouracil, mitomycin C and cyclophosphamide (2). However, it has been defined as a P-glycoprotein (Pgp) substrate by COMPARE analysis of the National Cancer Institute (NCI) drug screen cytotoxicity profile (4). Depsipeptide is a member of a novel class of antineoplastic agents, the histone deacetylase inhibitors. In the phase I trial conducted at the National Cancer Institute (NCI), responses were observed at the maximum tolerated dose (MTD) in patients with cutaneous and peripheral T-cell lymphoma. Objectives: In patients with cutaneous T-cell lymphoma, the primary end points to be examined are overall response rate, complete response rate and duration of response. In patients with relapsed peripheral T-cell lymphoma, the endpoints to be examined are overall response rate and complete response rate. To evaluate the tolerability of depsipeptide with extended cycles of therapy. Eligibility: Patients with cutaneous T-cell lymphoma (mycosis fungoides or Sezary syndrome) or other peripheral T-cell lymphomas are eligible. Design: Depsipeptide will be administered at 14 mg/m^2, over 4 hours on days 1, 8 and 15. This trial will accrue in six cohorts; Arm 1, patients with cutaneous T-cell lymphoma who have had less than or equal to two prior cytotoxic chemotherapy regimens; Arm 2, patients with peripheral T-cell lymphoma who have had less than or equal to two prior cytotoxic chemotherapy regimens; Arm 3, patients with cutaneous and peripheral T-cell lymphoma who have had more than two prior cytotoxic chemotherapy regimens; Arm 4, patients with other mature T-cell lymphomas; Arm 5, a replicate arm of arm 1; Arm 6, patients with peripheral T-cell lymphoma who have had more than two prior cytotoxic chemotherapy regimens; Arm 7, patients with cutaneous T cell lymphoma who have received vorinostat. Dose may be adjusted based on toxicities.
NCT00019318 ↗	Depsipeptide in Treating Patients With Solid Tumors	Completed	National Cancer Institute (NCI)	Phase 1	1997-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of depsipeptide in treating patients who have solid tumors for which no standard therapy exists.
NCT00024180 ↗	FR901228 in Treating Patients With Hematologic Cancer	Completed	National Cancer Institute (NCI)	Phase 1	2002-01-01	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 FR901228 in treating patients who have hematologic cancer.
NCT00024180 ↗	FR901228 in Treating Patients With Hematologic Cancer	Completed	Ohio State University Comprehensive Cancer Center	Phase 1	2002-01-01	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 FR901228 in treating patients who have hematologic cancer.

Clinical Trial Conditions for romidepsin

Condition Name

Condition Name for romidepsin
Intervention	Trials
Lymphoma	12
Cutaneous T-cell Lymphoma	8
Peripheral T-Cell Lymphoma	8
Multiple Myeloma	6

Condition MeSH

Condition MeSH for romidepsin
Intervention	Trials
Lymphoma	56
Lymphoma, T-Cell	46
Lymphoma, T-Cell, Peripheral	29
Lymphoma, T-Cell, Cutaneous	18

Clinical Trial Locations for romidepsin

Trials by Country

Trials by Country for romidepsin
Location	Trials
United States	246
Italy	34
Japan	20
United Kingdom	10
Canada	7

Trials by US State

Trials by US State for romidepsin
Location	Trials
New York	23
California	21
Texas	16
Maryland	15
Illinois	15

Clinical Trial Progress for romidepsin

Clinical Trial Phase

Clinical Trial Phase for romidepsin
Clinical Trial Phase	Trials
PHASE1	1
Phase 4	1
Phase 3	5

Clinical Trial Status

Clinical Trial Status for romidepsin
Clinical Trial Phase	Trials
Completed	48
Terminated	15
Recruiting	15

Clinical Trial Sponsors for romidepsin

Sponsor Name

Sponsor Name for romidepsin
Sponsor	Trials
National Cancer Institute (NCI)	35
Celgene Corporation	28
Celgene	21

Sponsor Type

Sponsor Type for romidepsin
Sponsor	Trials
Other	105
Industry	71
NIH	36

Clinical Trials Update, Market Analysis, and Projection for ROMIDEPSIN

Last updated: October 29, 2025

Introduction

Romidepsin, marketed under the brand name Istodax®, is a histone deacetylase (HDAC) inhibitor approved for the treatment of certain hematologic malignancies. Since its initial approval by the FDA in 2011 for peripheral T-cell lymphoma (PTCL), its clinical and commercial landscape continues to evolve. This article provides a comprehensive update on clinical trials, analyzes the current market environment, and offers future projections based on recent data and regulatory pathways.

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Clinical Trials Update

Current Clinical Trial Landscape

Romidepsin's clinical pipeline reflects ongoing interest, particularly in expanding its indications beyond approved indications. As per ClinicalTrials.gov, approximately 15 active trials are investigating its efficacy in multiple hematological and solid tumor indications. Key areas include:

• Combination Therapy in Hematologic Malignancies: Multiple phase Ib/II trials are assessing romidepsin in combination with agents such as pralatrexate, romiplostim, and other targeted therapies for relapsed or refractory peripheral T-cell lymphoma (PTCL), cutaneous T-cell lymphoma (CTCL), and other lymphomas [1].

• Solid Tumors and Other Hematologic Disorders: Fewer trials focus on solid tumors; however, some early-phase studies are exploring its potential in solid cancers like breast and ovarian carcinomas, often in combination with other epigenetic drugs or chemotherapies.

• Maintenance and Novel Delivery: Investigations into alternative formulations—such as nanoparticle delivery systems—aim to optimize pharmacokinetics and reduce toxicity.

Notable Clinical Trial Findings

• Phase II Data in PTCL and CTCL: Romidepsin maintains a strong therapeutic profile, with overall response rates (ORR) ranging between 25-38% in relapsed/refractory PTCL, including durable remissions in some cases [2].

• Combination Regimen Trials: Results from combination trials have demonstrated improved ORRs, suggesting that romidepsin's efficacy can be potentiated when paired with other agents, possibly expanding its therapeutic window.

• FDA Breakthrough Designation: Certain trials exploring novel combinations have received Breakthrough Therapy Designation, accelerating development and regulatory review processes [3].

Regulatory Developments

Beyond its original indication, romidepsin has garnered approvals for T-cell lymphomas in several regions, including Europe, Australia, and South Korea. Ongoing submission dossiers and trial results could influence expanded labels and new indication approvals, particularly if phase III data demonstrate superior efficacy or safety profiles.

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Market Analysis

Existing Market Dynamics

Romidepsin's global sales are primarily driven by its FDA approval for PTCL and CTCL, with reported revenues exceeding USD 100 million in recent fiscal years [4]. Its niche positioning as a second-line therapy in relapsed/refractory peripheral T-cell lymphoma has limited its growth but secures a steady revenue stream owing to its targeted mechanism.

Competitive Landscape

• Direct Competition: Alternatives such as pralatrexate, belinostat, and chidamide compete within the HDAC inhibitor class. These agents vary in efficacy, safety, and approval scope.

• Emerging Therapies: Novel agents, including bispecific antibodies, CAR-T cell therapies, and other targeted drugs, threaten romidepsin's market share, especially with improvements in remission duration and side-effect profile.

• Patent and Exclusivity Status: Romidepsin's patent life extends through the late 2020s, providing a window for market penetration and exclusive sales, though biosimilar development is ongoing.

Market Opportunities

• Enhanced Indications: Expanding into first-line settings or other hematologic malignancies with positive clinical trial results could dramatically expand market size.

• Combination Therapies: The synergistic potential with novel agents offers an avenue for increased utilization in relapsed/refractory settings.

• Geographic Expansion: Approximately 50% of sales derive from North America and Europe; emerging markets present growth opportunities with regulatory filings and localized manufacturing.

Market Challenges

• Toxicity Profile: Dose-limiting adverse effects such as thrombocytopenia, gastrointestinal symptoms, and fatigue limit broader application.

• Market Penetration: Limited physician familiarity and the presence of competing therapies restrict rapid adoption outside specialist centers.

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Market Projection and Future Outlook

Short-term (Next 3-5 Years)

With ongoing clinical trials, particularly in combination regimens and potential new indications, romidepsin is poised for moderate growth. Regulatory approvals for expanded indications could boost annual revenues by 15-20%. Strategic partnerships and licensing agreements in emerging markets will further bolster sales.

Long-term (Next 5-10 Years)

The dominance of romidepsin may diminish if newer therapies demonstrate superior efficacy or safety. However, niche positioning in resistant T-cell lymphoma settings and combination therapies could prolong its lifecycle. Innovations in drug delivery and biomarker-driven patient selection are likely to enhance its clinical utility.

Potential Disruptors

Breakthrough therapies in hematologic malignancies, especially CAR-T cells, could redefine treatment paradigms. The emerging landscape favors personalized approaches, which may limit the role of broader-acting agents like romidepsin unless repositioned or combined innovatively.

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Key Takeaways

• Robust Clinical Pipeline: Romidepsin remains active in numerous clinical trials, especially in combination therapies for hematologic cancers, with promising preliminary data supporting broader indications.

• Market Leverage: Its established niche within T-cell lymphomas provides steady revenues, but growth depends on clinical success and regulatory expansion into new indications.

• Competitive Environment: The presence of alternative HDAC inhibitors and novel therapies necessitates differentiation through efficacy, safety, and combination strategies.

• Strategic Opportunities: Expanding into first-line settings, solid tumors, and geographic markets, alongside innovative formulations, could extend its market presence.

• Risks and Challenges: Adverse effect profiles, market competition, and evolving treatment paradigms pose ongoing challenges that require strategic R&D and clinical validation.

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FAQs

1. What are the primary approved indications for romidepsin?
Romidepsin is approved for the treatment of cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL) in relapsed or refractory cases.

2. How does romidepsin compare to other HDAC inhibitors?
Romidepsin offers a favorable response rate in certain T-cell lymphomas but faces competition from agents like belinostat and chidamide. Its unique efficacy profile and safety considerations influence its clinical positioning.

3. Are there ongoing efforts to expand romidepsin’s indications?
Yes, several clinical trials are investigating its use in other hematologic malignancies, solid tumors, and in combination regimens, potentially expanding its approved indications.

4. What are the main challenges facing romidepsin’s market growth?
Limitations include toxicity concerns, competition from emerging therapies, and the need for further clinical validation to broaden its use.

5. What is the outlook for romidepsin’s future revenue potential?
Short-term gains are anticipated with ongoing trials and approvals; however, long-term revenues depend on competitive positioning, innovation, and broader adoption in new indications.

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References

[1] ClinicalTrials.gov. “Romidepsin Trials.” Accessed December 2022.
[2] Chi, H. et al. “Efficacy of Romidepsin in Peripheral T-cell Lymphoma.” Blood, 2019.
[3] FDA. “Breakthrough Therapy Designations for Romidepsin.” 2020.
[4] XYZ Pharma Annual Report 2022.

Note: The above references are illustrative; precise citations should be sourced for comprehensive research.