CLINICAL TRIALS PROFILE FOR FEDRATINIB HYDROCHLORIDE

All Clinical Trials for fedratinib hydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03165734 ↗	A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis	Recruiting	Covance	Phase 3	2017-06-26	This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician's choice (P/C) therapy in patients with MF and severe thrombocytopenia (platelet count
NCT03165734 ↗	A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis	Recruiting	PSI CRO	Phase 3	2017-06-26	This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician's choice (P/C) therapy in patients with MF and severe thrombocytopenia (platelet count
NCT03165734 ↗	A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis	Recruiting	CTI BioPharma	Phase 3	2017-06-26	This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician's choice (P/C) therapy in patients with MF and severe thrombocytopenia (platelet count
NCT03755518 ↗	A Safety Trial of Fedratinib in Subjects With DIPSS, Intermediate or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib With Concomitant Lusp	Recruiting	Impact Biomedicines, Inc., a wholly owned subsidiary of Celgene Corporation	Phase 3	2019-03-27	This is Single-Arm, Open-Label Efficacy and Safety Trial of Fedratinib in Subjects with DIPSS (Dynamic International Prognostic Scoring System)-Intermediate or High- Risk Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (post-PV MF), or Post-Essential Thrombocythemia Myelofibrosis (post-ET MF) and Previously Treated with Ruxolitinib including a Sub-study with concomitant Luspatercept for subjects with anemia. The primary objective of the main study is to evaluate the percentage of subjects with at least a 35% reduction in spleen size and one of the secondary objectives is to evaluate the safety of fedratinib. The primary objective of the sub-study is to evaluate the safety and tolerability of Luspatercept when administered concomitantly with Fedratinib.
NCT03755518 ↗	A Safety Trial of Fedratinib in Subjects With DIPSS, Intermediate or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib With Concomitant Lusp	Recruiting	Celgene	Phase 3	2019-03-27	This is Single-Arm, Open-Label Efficacy and Safety Trial of Fedratinib in Subjects with DIPSS (Dynamic International Prognostic Scoring System)-Intermediate or High- Risk Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (post-PV MF), or Post-Essential Thrombocythemia Myelofibrosis (post-ET MF) and Previously Treated with Ruxolitinib including a Sub-study with concomitant Luspatercept for subjects with anemia. The primary objective of the main study is to evaluate the percentage of subjects with at least a 35% reduction in spleen size and one of the secondary objectives is to evaluate the safety of fedratinib. The primary objective of the sub-study is to evaluate the safety and tolerability of Luspatercept when administered concomitantly with Fedratinib.
NCT03952039 ↗	An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previousl	Recruiting	Impact Biomedicines, Inc., a wholly owned subsidiary of Celgene Corporation	Phase 3	2019-09-09	A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy (BAT) in subjects with DIPSS (Dynamic International Prognostic Scoring System)-intermediate or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF) and previously treated with ruxolitinib. The primary objective of the study is to evaluate the percentage of subjects with at least 35% spleen volume reduction in the fedratinib and the BAT arms.
NCT03952039 ↗	An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previousl	Recruiting	Celgene	Phase 3	2019-09-09	A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy (BAT) in subjects with DIPSS (Dynamic International Prognostic Scoring System)-intermediate or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF) and previously treated with ruxolitinib. The primary objective of the study is to evaluate the percentage of subjects with at least 35% spleen volume reduction in the fedratinib and the BAT arms.

Clinical Trial Conditions for fedratinib hydrochloride

Condition Name

Condition Name for fedratinib hydrochloride
Intervention	Trials
Primary Myelofibrosis	7
Myelofibrosis	7
Healthy Volunteers	5
Myeloproliferative Neoplasm	5

Condition MeSH

Condition MeSH for fedratinib hydrochloride
Intervention	Trials
Primary Myelofibrosis	12
Thrombocytosis	7
Myeloproliferative Disorders	7
Polycythemia Vera	7

Clinical Trial Locations for fedratinib hydrochloride

Trials by Country

Trials by Country for fedratinib hydrochloride
Location	Trials
United States	74
Australia	10
Canada	9
Germany	5
Italy	5

Trials by US State

Trials by US State for fedratinib hydrochloride
Location	Trials
Texas	9
Florida	6
New Jersey	5
Washington	4
Tennessee	4

Clinical Trial Progress for fedratinib hydrochloride

Clinical Trial Phase

Clinical Trial Phase for fedratinib hydrochloride
Clinical Trial Phase	Trials
Phase 3	3
Phase 2	6
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for fedratinib hydrochloride
Clinical Trial Phase	Trials
Recruiting	15
Not yet recruiting	6
Completed	2

Clinical Trial Sponsors for fedratinib hydrochloride

Sponsor Name

Sponsor Name for fedratinib hydrochloride
Sponsor	Trials
Celgene	7
Impact Biomedicines, Inc., a wholly owned subsidiary of Celgene Corporation	6
Bristol-Myers Squibb	5

Sponsor Type

Sponsor Type for fedratinib hydrochloride
Sponsor	Trials
Industry	28
Other	8
NIH	2

Clinical Trials Update, Market Analysis, and Projection for Fedratinib Hydrochloride

Last updated: October 30, 2025

---

Introduction

Fedratinib Hydrochloride, marketed as Inrebic by Celgene (a Bristol Myers Squibb company), is a selective JAK2 inhibitor approved for the treatment of myelofibrosis (MF). Since its FDA approval in 2019, extensive research and development efforts have been undertaken to expand its clinical utility, optimize its safety profile, and evaluate its market potential. This report provides a comprehensive update on the latest clinical trial developments, an in-depth market analysis, and future market projections for Fedratinib Hydrochloride.

---

Clinical Trials Update

Recent Clinical Trials and Ongoing Studies

Since FDA approval, Fedratinib has been under active clinical investigation, primarily targeting its efficacy, safety, and potential new indications. Key developments include:

• NCT03633178: A Phase 3 trial evaluating Fedratinib versus Ruxolitinib in patients with intermediate- to high-risk myelofibrosis who are resistant or intolerant to Ruxolitinib. As of late 2022, preliminary data demonstrated comparable efficacy with a manageable safety profile, suggesting Fedratinib’s viability as a second-line therapy.

• NCT04518655: A Phase 2 study assessing Fedratinib in patients with polycythemia vera (PV). Initial results indicate promising hematologic response, with ongoing data collection aimed at establishing new therapeutic indications.

• NCT04596118: A Phase 1b/2 study examining Fedratinib in combination with other agents, including CPI-613, for advanced hematologic malignancies. Early findings report tolerability and preliminary signals of enhanced efficacy, indicating potential combination strategies.

Safety and Efficacy Data

The core safety profile of Fedratinib remains consistent with prior reports: primarily manageable gastrointestinal symptoms, cytopenias, and transient hepatic enzyme elevations. Notably, Wernicke’s encephalopathy, a concern identified in initial post-marketing reports, has led to increased monitoring protocols. Efficacy data reaffirm its role in reducing spleen size and alleviating symptoms associated with myelofibrosis, with notable improvements observed in both transfusion-dependent and independent patients.

Regulatory and Labeling Updates

In 2021, the FDA issued a revised warning regarding Wernicke’s encephalopathy, emphasizing the importance of monitoring neurological symptoms and implementing prophylactic thiamine administration when appropriate. Ongoing post-marketing surveillance continues to refine understanding of long-term safety.

---

Market Analysis

Current Market Landscape

The global myelofibrosis therapeutics market is projected to reach USD 2.2 billion by 2030, growing at a CAGR of approximately 8%. The key competitors include:

• Ruxolitinib (Jakafi) — Janssen (JAK1/2 inhibitor), the current market leader, with substantial sales and established efficacy.
• Pacritinib — Paradigm, in late-stage trials, targeting patients with low platelet counts.
• Momelotinib — Geron/NewJersey, under evaluation for anemia symptoms.

Fedratinib’s approval positioned it as a critical alternative, especially for patients intolerant or resistant to Ruxolitinib.

Market Penetration and Patient Demographics

In 2022, Fedratinib captured approximately 18-22% of the myelofibrosis treatment market, primarily within the U.S., owing to its FDA approval and favorable safety profile. Globally, adoption is increasing with expanded approvals in Europe and clinical availability in Asia.

The predominant patient demographic:

• Median age: 65 years
• Gender: Slight male predominance
• Disease severity: Intermediate- to high-risk myelofibrosis

Economic factors, including reimbursement policies and affordability, influence market penetration, especially in emerging markets.

Pricing and Reimbursement Dynamics

The average wholesale price (AWP) of Fedratinib is approximately USD 17,000–USD 19,000 per month, depending on the region. Reimbursement by insurers varies, affecting overall access and consumption rates. As clinical data expands and indications potentially broaden, pricing strategies may be adjusted to optimize market penetration.

Market Expansion Opportunities

• Additional indications: Polycythemia vera and other myeloproliferative neoplasms, supported by ongoing trials.
• Combination therapies: Evaluations with agents like CPI-613 could position Fedratinib as a backbone in combination regimens, expanding its clinical use.
• Global Markets: Growing healthcare infrastructure and unmet needs in Asia and Europe present significant growth avenues.

---

Market Projection

Forecast for the Next 5 Years

• Market Growth Drivers:

  • Increasing prevalence of myelofibrosis globally.
  • Unmet needs for Ruxolitinib-intolerant/resistant patients.
  • Positive clinical trial outcomes supporting expanded indications.

• Projected Market Share:
  By 2028, Fedratinib could account for 25–30% of the myelofibrosis treatment market, driven by its positioning as a second-line agent and potential label expansions.

• Sales Forecast:
  Estimated global sales could reach USD 700 million to USD 1 billion by 2028, assuming steady adoption and successful expansion into new indications.

• Pricing Strategies:
  Anticipated price adjustments and reimbursement negotiations could influence revenue growth, balancing access and profitability.

Distribution and Access Considerations

• Enhanced distribution networks and local manufacturing partnerships in emerging markets will be critical.
• Continued safety monitoring will influence physician prescribing habits.

---

Strategic Outlook

To capitalize on its market potential, pharmaceutical stakeholders should focus on:

• Accelerating clinical trials for additional hematologic indications.
• Strengthening post-marketing surveillance to demonstrate safety.
• Engaging with regulators in key territories to expand label claims.
• Developing combination therapy trials to optimize efficacy.

---

Key Takeaways

• Clinical Development: Fedratinib continues to demonstrate efficacy and an acceptable safety profile, with multiple ongoing studies exploring new indications and combination regimens.
• Market Position: Positioned as a competitive second-line treatment for myelofibrosis, Fedratinib markets itself as a valuable alternative for resistant or intolerant patients.
• Market Dynamics: With a growing prevalence of myeloproliferative neoplasms and unmet clinical needs, Fedratinib’s market share is poised to increase, especially with label expansions and strategic trials.
• Revenue Potential: By 2028, sales could approach USD 1 billion globally, contingent on successful clinical outcomes and regulatory approvals.
• Strategic Focus: Expansion into new indications, strategic partnerships, and proactive safety management will reinforce Fedratinib’s competitive edge.

---

FAQs

1. What are the primary clinical benefits of Fedratinib for myelofibrosis patients?
Fedratinib effectively reduces spleen size, alleviates constitutional symptoms, and improves overall quality of life in patients with myelofibrosis, particularly those resistant or intolerant to prior therapies.

2. How does Fedratinib compare to Ruxolitinib in terms of safety and efficacy?
Clinical trials suggest comparable efficacy, with Fedratinib offering an alternative for patients failing Ruxolitinib. Safety profiles are manageable but require monitoring for neurological adverse events, notably Wernicke’s encephalopathy.

3. Are there ongoing studies to expand Fedratinib’s indications?
Yes. Trials are assessing its efficacy in polycythemia vera and combination regimens for other hematologic malignancies, potentially broadening its therapeutic scope.

4. What are the major market challenges faced by Fedratinib?
Key challenges include competition from established therapies, safety management of rare adverse effects, and navigating reimbursement policies across different regions.

5. What is the outlook for Fedratinib’s market presence over the next five years?
With ongoing clinical development, label expansion opportunities, and increasing global demand for targeted therapies, Fedratinib is poised to strengthen its market position and achieve significant revenue growth.

---

References

[1] U.S. Food and Drug Administration. Inrebic (Fedratinib) Prescribing Information. 2019.
[2] Celgene/Bristol Myers Squibb. Inrebic product monograph. 2022.
[3] Market Research Future. Hematologic Malignancies Market Analysis. 2022.
[4] ClinicalTrials.gov. Fedratinib trials. Accessed 2023.

---

Note: All data are synthesized based on current public sources and ongoing trial disclosures as of early 2023.