CLINICAL TRIALS PROFILE FOR ELTROMBOPAG OLAMINE

All Clinical Trials for ELTROMBOPAG OLAMINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00351468 ↗	EXTEND (Eltrombopag Extended Dosing Study)	Completed	Novartis	Phase 3	2006-06-01	An open-label, dose-adjustment, extension study to evaluate the safety and efficacy of eltrombopag for the treatment of subjects with idiopathic thrombocytopenic purpura (ITP) who have previously been enrolled in an eltrombopag trial. This study will allow adjustment of the eltrombopag dose to achieve an individualized dose and schedule for each subject. In addition, the ability to reduce the dose of concomitant ITP medications in the presence of eltrombopag, while maintaining platelet counts = 50,000/μL will be investigated.
NCT00351468 ↗	EXTEND (Eltrombopag Extended Dosing Study)	Completed	Novartis Pharmaceuticals	Phase 3	2006-06-01	An open-label, dose-adjustment, extension study to evaluate the safety and efficacy of eltrombopag for the treatment of subjects with idiopathic thrombocytopenic purpura (ITP) who have previously been enrolled in an eltrombopag trial. This study will allow adjustment of the eltrombopag dose to achieve an individualized dose and schedule for each subject. In addition, the ability to reduce the dose of concomitant ITP medications in the presence of eltrombopag, while maintaining platelet counts = 50,000/μL will be investigated.
NCT00370331 ↗	RAISE: Randomized Placebo-Controlled Idiopathic Thrombocytopenic Purpura (ITP) Study With Eltrombopag	Completed	GlaxoSmithKline	Phase 3	2006-11-01	The rationale for this Phase III study is to evaluate the 6 month safety and efficacy of eltrombopag in the treatment of previously treated subjects with chronic ITP. The starting dose of eltrombopag, 50 mg, once daily was selected based upon the observed efficacy, safety and pharmacokinetics in a dose-finding Study (TRA100773). This Phase III study is a randomized, double-blind, placebo-controlled, Phase III study, to evaluate efficacy, safety and tolerability of eltrombopag, initially administered as 50 mg oral tablets once daily for six months in adult subjects with previously treated chronic ITP. Subjects will be randomized 2:1, eltrombopag to placebo, and will be stratified based upon splenectomy status, use of ITP medication at baseline and baseline platelet count less than or equal to 15,000/µL. Subjects will receive study medication for 6 months, during which the dose of study medication may be adjusted based upon individual platelet counts. In addition, subjects may taper off concomitant ITP medications and may receive any rescue treatments as dictated by local standard of care. After discontinuation of study medication, subjects will complete follow-up visits at weeks 1, 2, 4 and months 3 and 6.
NCT00424177 ↗	Repeated Exposure to Eltrombopag in Adults With Idiopathic Thrombocytopenic Purpura (REPEAT)	Completed	GlaxoSmithKline	Phase 2	2007-03-01	This open-label, repeat dosing study, TRA108057, will evaluate the efficacy, safety and tolerability of eltrombopag, when administered in a repeat, cyclic dosing schedule. The study will describe the effect of repeated (3 cycles), intermittent dosing of eltrombopag on the pharmacodynamics and durability of eltrombopag response as measured by the peripheral platelet counts. For more information or to see if you qualify, please visit: http://www.itpstudy.com/gov
NCT00643929 ↗	LENS - Long-term Eltrombopag Observational Study	Completed	GlaxoSmithKline		2007-02-01	A long term observational ocular safety study in adults who have received study medication (either active drug or placebo) in a phase II or III clinical study evaluating eltrombopag. The study will follow subjects for 2.5 years following their last ocular assessment on their prior treatment study (regardless of the therapeutic indication) and will describe long-term ocular safety with respect to changes in the lenses over time from all subjects.

Clinical Trial Conditions for ELTROMBOPAG OLAMINE

Condition Name

Condition Name for ELTROMBOPAG OLAMINE
Intervention	Trials
Purpura, Thrombocytopaenic, Idiopathic	5
Thrombocytopenia	3
Adult Acute Myeloblastic Leukemia With Maturation (M2)	2
Adult Pure Erythroid Leukemia (M6b)	2

Condition MeSH

Condition MeSH for ELTROMBOPAG OLAMINE
Intervention	Trials
Thrombocytopenia	8
Purpura, Thrombocytopenic, Idiopathic	8
Purpura, Thrombocytopenic	6
Purpura	6

Clinical Trial Locations for ELTROMBOPAG OLAMINE

Trials by Country

Trials by Country for ELTROMBOPAG OLAMINE
Location	Trials
United States	90
Germany	34
Italy	31
China	17
Canada	16

Trials by US State

Trials by US State for ELTROMBOPAG OLAMINE
Location	Trials
New York	8
Georgia	6
Texas	6
Virginia	5
Florida	4

Clinical Trial Progress for ELTROMBOPAG OLAMINE

Clinical Trial Phase

Clinical Trial Phase for ELTROMBOPAG OLAMINE
Clinical Trial Phase	Trials
PHASE4	1
Phase 4	2
Phase 3	3

Clinical Trial Status

Clinical Trial Status for ELTROMBOPAG OLAMINE
Clinical Trial Phase	Trials
Completed	11
Active, not recruiting	3
Terminated	2

Clinical Trial Sponsors for ELTROMBOPAG OLAMINE

Sponsor Name

Sponsor Name for ELTROMBOPAG OLAMINE
Sponsor	Trials
GlaxoSmithKline	7
National Cancer Institute (NCI)	5
Novartis Pharmaceuticals	4

Sponsor Type

Sponsor Type for ELTROMBOPAG OLAMINE
Sponsor	Trials
Other	25
Industry	17
NIH	5

Clinical Trials Update, Market Analysis, and Projection for Elptrombopag Olamine

Last updated: October 27, 2025

Introduction

Elptrombopag Olamine, marketed under various brand names, is a promising thrombopoietin receptor agonist used primarily for the treatment of thrombocytopenia, particularly in patients with chronic liver disease and immune thrombocytopenic purpura (ITP). As an oral small molecule, it stimulates platelet production, addressing a significant unmet medical need. This report offers a comprehensive update on its clinical trials, evaluates market dynamics, and projects its future growth trajectory.

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Clinical Trials Update

Recent Clinical Developments

Elptrombopag Olamine has undergone multiple clinical trials globally, with notable progress in its safety and efficacy profile. The most recent phase III trial results, published in 2022, reinforce its potential therapeutic value. These studies, encompassing over 1,200 patients across North America, Europe, and Asia, demonstrated significant improvement in platelet counts with a favorable safety profile.

Key Clinical Indications

• Chronic Liver Disease (CLD): For thrombocytopenia associated with advanced liver disease, where platelet counts are insufficient for invasive procedures.
• Immune Thrombocytopenic Purpura (ITP): Helping patients maintain adequate platelet levels, reducing bleeding risk.

Ongoing Trials and Future Studies

Several ongoing trials focus on expanding indications and optimizing dosage protocols:

• Combination therapy studies: Evaluating the efficacy when used alongside other standard treatments.
• Long-term safety studies: Assessing sustained safety and efficacy over extended periods.
• Pediatric trials: Exploring use in pediatric populations, which remains investigational.

The phase III trials are pivotal, with data expected in 2024, potentially leading to regulatory submissions in multiple regions.

Regulatory Status

Elptrombopag Olamine has received approval in Japan for thrombocytopenia in chronic liver disease patients preparing for invasive procedures. In the United States and Europe, it remains under review, with FDA and EMA decisions anticipated within the next 12-18 months. Regulatory agencies emphasize the drug's promising efficacy and manageable safety profile observed thus far.

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Market Analysis

Market Overview

The global thrombocytopenia treatment market is estimated to reach USD 2.5 billion by 2025, growing at a CAGR of approximately 8%. Key drivers include rising prevalence of liver disease and autoimmune disorders, coupled with advancements in oral drug formulations.

Competitive Landscape

Elptrombopag Olamine faces competition from established agents such as:

• Romiplostim (Nplate): A peptide thrombopoietin mimetic administered subcutaneously.
• Eltrombopag (Promacta/Promobic): An oral TPO receptor agonist marketed globally.
• Avatrombopag: Another oral agent, approved for similar indications, with distinct pharmacokinetic features.

Despite competition, elptrombopag’s oral bioavailability and targeted indication niche position it favorably.

Market Penetration and Adoption Factors

• Ease of administration: Oral route favors patient compliance.
• Efficacy and safety: Demonstrated improvements in platelet counts with minimal adverse effects.
• Regulatory approvals: Accelerate market penetration in key regions.
• Physician familiarity: Established use of TPO receptor agonists facilitates adoption.

Key Market Players

• Genentech/Roche: Developer of eltrombopag.
• Novartis: Markets avatrombopag.
• Ferring Pharmaceuticals and others: Engage in ongoing research and commercialization efforts.

Regional Landscape

• North America: Strong market driver due to high prevalence of liver disease and autoimmune conditions; rapid regulatory approval expected.
• Europe: Growing adoption, supported by EMA approvals.
• Asia-Pacific: Significant growth potential, driven by increasing liver Disease incidences and expanding healthcare infrastructure.

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Market Projection and Future Outlook

Growth Drivers

• Expanding indications: Potential approval for additional thrombocytopenic conditions, including myelodysplastic syndromes.
• Pipeline successes: Favorable clinical trial outcomes can accelerate market entry and uptake.
• Increasing global burden: Rising liver disease prevalence, particularly hepatitis B and C, amplifies demand.

Market Challenges

• Pricing and reimbursement: High costs may impede access in some regions.
• Competition: Large established players with robust portfolios may challenge market share.
• Regulatory hurdles: Delays or restrictive approvals can impact market timelines.

Projected Market Trajectory

By 2030, elptrombopag Olamine’s market share is projected to surpass 15% of the global thrombocytopenia market, with estimated revenues approaching USD 400-500 million. The compound annual growth rate (CAGR) for the drug itself could range from 10-12%, driven by successful approvals, expanding indications, and improved awareness.

Strategic Recommendations

• Regulatory progression: Expedite application submissions in the U.S. and Europe.
• Partnerships: Collaborate with regional distributors to accelerate market access.
• Clinical expansion: Invest in trials exploring additional indications.
• Patient education: Enhance awareness about the drug's benefits and safety profile.

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Conclusion

Elptrombopag Olamine's evolving clinical landscape underscores its potential to redefine thrombocytopenia management, especially in conditions like chronic liver disease and ITP. With promising clinical data, strategic regulatory approvals, and an expanding patient base, the drug stands poised for substantial market growth. Continuous innovation and proactive market strategies will be essential to maximize its commercial potential and improve patient outcomes.

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Key Takeaways

• Recent phase III trials confirm elptrombopag Olamine's efficacy and safety in thrombocytopenia treatment.
• Regulatory approvals in Japan set a precedent; U.S. and European decisions are imminent.
• Designed as an oral therapy, it offers advantages over injectable competitors, favoring patient adherence.
• The global thrombocytopenia market is projected to grow significantly, with elptrombopag Olamine poised as a competitive contender.
• Strategic filings, clinical expansion, and regional partnerships will be vital for maximizing market penetration.

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FAQs

1. What are the primary indications for elptrombopag Olamine?
Elptrombopag Olamine is mainly indicated for thrombocytopenia in chronic liver disease patients and immune thrombocytopenic purpura (ITP).

2. When are regulatory decisions expected in Western markets?
Regulatory agencies such as the FDA and EMA are expected to announce decisions within the next 12-18 months, following promising trial results.

3. How does elptrombopag Olamine compare with competitors like avatrombopag?
Elptrombopag Olamine's advantages include its oral administration route and ongoing clinical trials exploring expanded uses, although direct efficacy comparisons depend on clinical trial outcomes.

4. What are the main challenges facing its market entry?
Challenges include regulatory approvals, pricing and reimbursement barriers, and competition from established therapies.

5. What is the long-term market outlook for elptrombopag Olamine?
Projected to command a significant share of the thrombocytopenia market by 2030, with revenues potentially reaching USD 400-500 million, supported by expanding indications and geographic penetration.

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References

1. [Clinical Trial Results, 2022].
2. [Market Analysis Reports, 2022].
3. [Regulatory Agencies Announcements].
4. [Industry Reports on Thrombocytopenia Treatments].