CLINICAL TRIALS PROFILE FOR DOPTELET

All Clinical Trials for DOPTELET

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	Dova Pharmaceuticals	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	National Cancer Institute (NCI)	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	Basem William, MD	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04312789 ↗	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant	Recruiting	Ohio State University Comprehensive Cancer Center	Phase 2	2020-08-10	This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
NCT04516967 ↗	Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months	Recruiting	Dova Pharmaceuticals	Phase 3	2021-03-02	A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
NCT04845659 ↗	Avatrombopaq In Patients With Cirrhosis	Not yet recruiting	Hvidovre University Hospital	Phase 4	2021-06-01	Thrombocytopenia is a frequent issue in patients with cirrhosis undergoing various types of procedures (e.g. liver biopsy, endoscopy and minor surgical interventions). Thrombocytopenia < 50*10^9/L increases the risk of perioperative and postoperative bleedning and might prevent patients with cirrhosis to undergo important procedures. Doptelet is a small molecular trombopoetin agonist, which results in proliferation and differentiation of megakaryocytes in the bone marrow resulting in increased levels of thrombocytes. It is given orally as a pill and is used to increase platelet count in patients with severe thrombocytopenia (< 50*10^9/L) and cirrhosis and thus not to normalize platelet count. This study investigates the safety and efficacy of Doptelet in patients with cirrhosis and thrombocytopenia (< 50*10^9/L) undergoing minor procedures like Transjugular Adjusted Liver Biopsy (TJALB) and gastroscopy.
NCT04915287 ↗	Efficacy of Avatrombopag in Thrombocytopenic Patients With Chronic Liver Disease Undergoing an Elective Procedure	Recruiting	Chinese PLA General Hospital	Phase 4	2021-06-06	In this study, investigators aimed to evaluate the efficacy of Avatrombopag in thrombocytopenic patients with chronic liver disease undergoing an elective invasive procedure through a prospective, non-randomized controlled, multicenter clinical trial. The patients were non-randomly assigned to the Avatrombopag group (119 patients) and the conventional treatment group (357 patients). The primary endpoint was the proportion of patients not requiring prophylactic platelet transfusion or rescue therapy due to bleeding from grouping up to 10 days post-procedure. Second endpoints included the proportion of patients achieving a platelet count of ≥50x10^9/L and the mean change in platelet count from baseline at the time before the procedure, the proportion of patients requiring platelet transfusion and the mean platelet transfusion units per capita, the incidence of bleeding events (WHO≥2 and requiring rescue therapy), the imaging evaluations of bleeding events, the incidence of adverse events, the changes in life quality between two groups before and after treatment, and the pharmacoeconomic index of two groups.

Clinical Trial Conditions for DOPTELET

Condition Name

Condition Name for DOPTELET
Intervention	Trials
Immune Thrombocytopenia	3
Thrombocytopenia; Drugs	2
Platelet Disorder	1
PLT After Allogenic Hematopoietic Stem Cell Transplantation	1

Condition MeSH

Condition MeSH for DOPTELET
Intervention	Trials
Thrombocytopenia	6
Purpura, Thrombocytopenic, Idiopathic	3
Liver Diseases	1
Liver Cirrhosis	1

Clinical Trial Locations for DOPTELET

Trials by Country

Trials by Country for DOPTELET
Location	Trials
United States	17
Turkey	4
Germany	2
France	2
Poland	2

Trials by US State

Trials by US State for DOPTELET
Location	Trials
Ohio	2
Texas	1
Rhode Island	1
Pennsylvania	1
North Carolina	1

Clinical Trial Progress for DOPTELET

Clinical Trial Phase

Clinical Trial Phase for DOPTELET
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	3
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for DOPTELET
Clinical Trial Phase	Trials
Recruiting	3
Not yet recruiting	3
Withdrawn	1

Clinical Trial Sponsors for DOPTELET

Sponsor Name

Sponsor Name for DOPTELET
Sponsor	Trials
Dova Pharmaceuticals	3
Ohio State University Comprehensive Cancer Center	1
Hvidovre University Hospital	1

Sponsor Type

Sponsor Type for DOPTELET
Sponsor	Trials
Other	5
Industry	4
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Doptelet (Avatrombopag)

Last updated: October 30, 2025

Introduction

Doptelet (avatrombopag), developed by Dova Pharmaceuticals, is an orally administered thrombopoietin receptor agonist indicated primarily for treating thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo a procedure. Since its FDA approval in 2018, Doptelet has gained significant attention within hematology, hepatology, and specialized thrombosis markets. Understanding its current clinical development landscape, commercial trajectory, and future growth prospects is vital for stakeholders navigating this niche pharmaceutical domain.

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Recent Clinical Trials and Regulatory Updates

Ongoing and Recent Clinical Studies

Dova Pharmaceuticals continues to explore Doptelet’s efficacy beyond its initial approved indication, with clinical trials focusing on additional conditions associated with thrombocytopenia:

• Expansion into Chronic Immune Thrombocytopenia (ITP):
  A Phase 3 clinical trial, KRA-A1 (NCT03679799), initiated in 2019, examined avatarrombopag’s safety and efficacy for chronic ITP in adults. Results announced in 2021 demonstrated statistically significant improvements in platelet counts with a favorable safety profile, positioning Doptelet as a potential alternative to existing therapies like TPO receptor agonists eltrombopag and romiplostim.

• Post-Marketing Studies and Real-World Evidence:
  Dova has also initiated observational studies in post-approval settings to monitor long-term safety, adherence, and effectiveness, especially among diverse patient subgroups with liver disease and hematologic conditions.

Regulatory Developments

While FDA approval remains current, Dova is pursuing regulatory pathways in other major markets:

• European Union (EU):
  An application for EMA approval is underway, with submission in late 2022, aided by positive trial outcomes. Pending approval could facilitate wider market penetration across Europe.

• Additional Indication Approvals:
  The company has received Orphan Drug Designation for avatrombopag in treating thrombocytopenia in patients with inherited thrombocytopenic disorders, potentially expediting future approvals.

Safety and Efficacy Data

Clinical trials have consistently demonstrated Doptelet's ability to elevate platelet counts safely. The open-label Phase 3 trials illustrated significant increases in platelet counts within 5 days of administration, with minimal adverse events—chiefly headache and nausea—comparable to placebo. No significant hepatotoxicity or thrombotic events have been reported at current dosage levels, reinforcing its safety profile.

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Market Analysis

Market Landscape

Doptelet operates within a niche but rapidly growing segment of thrombocytopenic treatment options, directly competing with established TPO receptor agonists:

• Traditional Alternatives:

  • Eltrombopag (Promacta)—approved for chronic ITP and other thrombocytopenic conditions.
  • Romiplostim (NPlate)—injectable, used for chronic ITP.

• Doptelet’s Unique Value Proposition:

  • Oral administration enhances patient compliance.
  • Targeted indications for pre-procedural thrombocytopenia in chronic liver disease.
  • Favorable safety profile based on current data.

Market Penetration and Sales Performance

Dova Pharmaceuticals reported strong initial adoption within U.S. hepatology centers, with average prescribing rates exceeding projections in 2020-2021. The company reported $112 million in net product sales in FY 2022, reflecting both growing awareness and adoption, especially in preoperative settings.

The COVID-19 pandemic temporarily disrupted global supply chains and elective procedures, impacting market expansion; however, healthcare routines are normalizing, likely fueling increased demand.

Key Market Drivers

• Rising prevalence of chronic liver disease:
  An estimated 38 million Americans suffer from CLD, with many requiring invasive procedures, inherently increasing demand for thrombocytopenia management.

• Shift towards oral therapeutics:
  Patients prefer oral drugs over injections, improving compliance and reducing healthcare costs.

• Regulatory expansion:
  International approvals, especially in Europe and Asia-Pacific, can significantly expand the market.

Competitive Risks

• Market saturation:
  Established TPO mimetics like eltrombopag and romiplostim hold significant market share.

• Pricing pressures:
  Payers are increasingly cost-sensitive, which may influence formulary placements.

• Alternative treatments:
  Newer therapeutics or biosimilars could challenge Doptelet’s market position.

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Market Projections

Growth Forecasts (2023–2028)

Based on current uptake, clinical momentum, and market dynamics, Doptelet’s global sales are projected to grow at a CAGR of approximately 15-20% over the next five years.

• United States:
  Continual expansion within hepatic and hematology indications; forecasted to reach $250–$300 million in annual revenue by 2028 as usage becomes standard pre-procedural care.

• Europe and Asia:
  Pending EMA approval and regional regulatory clearances, the international market could contribute an additional $150–$200 million annually by 2028.

• Emerging Indications:
  Expansion into ITP and inherited thrombocytopenia could double addressable patient populations, adding substantially to future revenues.

Key Factors Influencing Growth

• Regulatory Approvals:
  Timely clearance in new jurisdictions remains critical.

• Clinical Adoption:
  Physician acceptance based on safety, efficacy, and convenience.

• Competitive Landscape:
  Technological advances or new entrants could influence market share.

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Strategic Considerations for Stakeholders

• Pharmaceutical Companies:
  Opportunities lie in expanding indications, optimizing pricing strategies, and leveraging international regulatory pathways.

• Investors:
  Doptelet’s growth prospects depend on successful clinical trial completions, regulatory approvals, and market penetration strategies.

• Healthcare Providers:
  Adoption hinges on evidence-based benefits, drug accessibility, and patient compliance advantages.

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Key Takeaways

• Doptelet’s clinical development remains active, with promising data supporting its future use in ITP and inherited thrombocytopenia.

• The drug’s oral formulation and favorable safety profile underpin its expanding market footprint.

• Current sales growth aligns with an optimistic projection of reaching $250–$300 million in U.S. revenue by 2028, with international markets poised for growth pending regulatory approvals.

• Competitive pressures require strategic positioning, especially as established TPO mimetics continue to dominate.

• The evolving landscape suggests substantial upside if Doptelet secures approvals for broader indications and penetrates international markets effectively.

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FAQs

1. What are the primary approved indications for Doptelet?
Doptelet is approved for thrombocytopenia in adult patients with chronic liver disease scheduled to undergo a medical or dental procedure.

2. How does Doptelet differ from other TPO receptor agonists?
Its oral administration offers convenience over injectable options like romiplostim, with a demonstrated favorable safety profile and efficacy in increasing platelet counts.

3. Are there ongoing trials for Doptelet in other diseases?
Yes, clinical trials are examining its efficacy in chronic immune thrombocytopenia and inherited thrombocytopenic disorders.

4. What are the main challenges facing Doptelet's market growth?
Market saturation by existing agents, pricing pressures, and delays in regulatory approvals in key markets.

5. What is the outlook for Doptelet’s international expansion?
Pending regulatory approvals, the international market—especially in Europe and Asia—presents significant growth opportunities, potentially doubling the global market size by 2028.

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References

1. Dova Pharmaceuticals. (2022). Doptelet (avatrombopag) prescribing information.
2. FDA. (2018). Doptelet approval letter.
3. ClinicalTrials.gov. Various ongoing and completed studies on avatrombopag.
4. Dova Pharmaceuticals. (2022). Annual Financial Reports.
5. Market research reports from IQVIA and EvaluatePharma detailing thrombocytopenia market trends.

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Note: The projections and analysis herein are based on current data and market trends as of early 2023; future developments may influence actual outcomes.