CLINICAL TRIALS PROFILE FOR COAGULATION FACTOR IX (RECOMBINANT), FC FUSION PROTEIN

All Clinical Trials for coagulation factor ix (recombinant), fc fusion protein

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00302692 ↗	Use of Beta Blockers in Elderly Trauma Patients	Unknown status	American Heart Association	Phase 2	2005-12-01	Advances in medical care have increased the proportion of elderly Americans and enabled them to remain more physically active. This has resulted in an unprecedented increase in the number of geriatric patients admitted to trauma centers. The elderly constitute 23% of trauma center admissions, but 36% of all trauma deaths. This disproportionately high mortality is attributable to a higher prevalence of pre-existing conditions, particularly, cardiac disease. Multi-system injuries result in critical cardiac stress. Although beta-blockade has been shown to decrease morbidity and mortality in patients at risk for myocardial infarction after elective surgery, their use in trauma patients with potential underlying cardiac disease has not been previously studied. We hypothesize that routine administration of beta-blockers after resuscitation will reduce morbidity and mortality in elderly trauma patients with, or at risk for, underlying cardiac disease. This study is a randomized, prospective clinical trial. One cohort will receive routine trauma intensive care, and the other, the same care plus beta-blockade after completion of resuscitation. The primary outcome will be mortality. Secondary outcomes include MI, length of stay, organ dysfunction, cardiac, and other complications. Changes in outcome may not be due to reduction in myocardial oxygen demand and heart rate. Laboratory studies demonstrate that circulating inflammatory cytokines contribute to cardiac risk in trauma patients, and their production is influenced by adrenergic stimulation. We will measure circulating IL-6, TNF alpha, IL-1beta, and measure NF-kB and p38 MAP kinase activation in peripheral blood leukocytes, and determine the effect of beta-blockade on the production of these inflammatory markers. Finally, the wide variation in patient response to beta-blockers is attributed to genetic variability in the adrenergic receptor. Therefore, we will identify single nucleotide polymorphisms (SNPS) within the beta-adrenergic receptor, and determine their effects on mortality and response to beta-blockade. This study will provide the first randomized, prospective trial designed to reduce morbidity and mortality in elderly trauma patients at risk for cardiac disease. The laboratory and genetic component will provide additional insights that may explain treatment effects, lead to new therapeutic strategies, and have the potential to lead to additional areas of investigation.
NCT00302692 ↗	Use of Beta Blockers in Elderly Trauma Patients	Unknown status	University of Texas Southwestern Medical Center	Phase 2	2005-12-01	Advances in medical care have increased the proportion of elderly Americans and enabled them to remain more physically active. This has resulted in an unprecedented increase in the number of geriatric patients admitted to trauma centers. The elderly constitute 23% of trauma center admissions, but 36% of all trauma deaths. This disproportionately high mortality is attributable to a higher prevalence of pre-existing conditions, particularly, cardiac disease. Multi-system injuries result in critical cardiac stress. Although beta-blockade has been shown to decrease morbidity and mortality in patients at risk for myocardial infarction after elective surgery, their use in trauma patients with potential underlying cardiac disease has not been previously studied. We hypothesize that routine administration of beta-blockers after resuscitation will reduce morbidity and mortality in elderly trauma patients with, or at risk for, underlying cardiac disease. This study is a randomized, prospective clinical trial. One cohort will receive routine trauma intensive care, and the other, the same care plus beta-blockade after completion of resuscitation. The primary outcome will be mortality. Secondary outcomes include MI, length of stay, organ dysfunction, cardiac, and other complications. Changes in outcome may not be due to reduction in myocardial oxygen demand and heart rate. Laboratory studies demonstrate that circulating inflammatory cytokines contribute to cardiac risk in trauma patients, and their production is influenced by adrenergic stimulation. We will measure circulating IL-6, TNF alpha, IL-1beta, and measure NF-kB and p38 MAP kinase activation in peripheral blood leukocytes, and determine the effect of beta-blockade on the production of these inflammatory markers. Finally, the wide variation in patient response to beta-blockers is attributed to genetic variability in the adrenergic receptor. Therefore, we will identify single nucleotide polymorphisms (SNPS) within the beta-adrenergic receptor, and determine their effects on mortality and response to beta-blockade. This study will provide the first randomized, prospective trial designed to reduce morbidity and mortality in elderly trauma patients at risk for cardiac disease. The laboratory and genetic component will provide additional insights that may explain treatment effects, lead to new therapeutic strategies, and have the potential to lead to additional areas of investigation.
NCT01027364 ↗	Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B	Completed	Swedish Orphan Biovitrum	Phase 3	2009-12-01	The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc; to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of prophylaxis over on-demand (episodic) therapy by comparing the annualized number of bleeding episodes between participants receiving rFIXFc on each prevention (prophylaxis) regimen and participants receiving rFIXFc on an episodic regimen. The secondary objectives of the study were: to evaluate and assess the pharmacokinetic (PK) parameter estimates of rFIXFc and rFIX (BeneFIX®) at baseline in the Sequential PK subgroup as well as rFIXFc at Week 26 (±1 week); to evaluate participants' response to treatment; to evaluate rFIXFc consumption.
NCT01027364 ↗	Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B	Completed	Biogen	Phase 3	2009-12-01	The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc; to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of prophylaxis over on-demand (episodic) therapy by comparing the annualized number of bleeding episodes between participants receiving rFIXFc on each prevention (prophylaxis) regimen and participants receiving rFIXFc on an episodic regimen. The secondary objectives of the study were: to evaluate and assess the pharmacokinetic (PK) parameter estimates of rFIXFc and rFIX (BeneFIX®) at baseline in the Sequential PK subgroup as well as rFIXFc at Week 26 (±1 week); to evaluate participants' response to treatment; to evaluate rFIXFc consumption.

Clinical Trial Conditions for coagulation factor ix (recombinant), fc fusion protein

Condition Name

Condition Name for coagulation factor ix (recombinant), fc fusion protein
Intervention	Trials
Hemophilia A	7
Severe Hemophilia A	4
Hemophilia B	1
Multiple Trauma	1

Condition MeSH

Condition MeSH for coagulation factor ix (recombinant), fc fusion protein
Intervention	Trials
Hemophilia A	16
Hemophilia B	3
Multiple Trauma	1
Respiratory Distress Syndrome	1

Clinical Trial Locations for coagulation factor ix (recombinant), fc fusion protein

Trials by Country

Trials by Country for coagulation factor ix (recombinant), fc fusion protein
Location	Trials
United States	52
China	46
Australia	15
United Kingdom	14
Japan	11

Trials by US State

Trials by US State for coagulation factor ix (recombinant), fc fusion protein
Location	Trials
California	6
Pennsylvania	5
Michigan	4
Indiana	4
Nevada	4

Clinical Trial Progress for coagulation factor ix (recombinant), fc fusion protein

Clinical Trial Phase

Clinical Trial Phase for coagulation factor ix (recombinant), fc fusion protein
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	12
Phase 2	2

Clinical Trial Status

Clinical Trial Status for coagulation factor ix (recombinant), fc fusion protein
Clinical Trial Phase	Trials
Recruiting	6
Completed	4
Not yet recruiting	4

Clinical Trial Sponsors for coagulation factor ix (recombinant), fc fusion protein

Sponsor Name

Sponsor Name for coagulation factor ix (recombinant), fc fusion protein
Sponsor	Trials
Jiangsu Gensciences lnc.	6
Swedish Orphan Biovitrum	4
Biogen	4

Sponsor Type

Sponsor Type for coagulation factor ix (recombinant), fc fusion protein
Sponsor	Trials
Industry	25
Other	7

Clinical Trials Update, Market Analysis, and Projection for Coagulation Factor IX (Recombinant), Fc Fusion Protein

Last updated: November 17, 2025

Introduction

Coagulation Factor IX (Recombinant), Fc Fusion Protein, commonly referred to as Fc-fused recombinant FIX, represents a transformative innovation in hemophilia B management. By fusing recombinant FIX with the Fc domain of immunoglobulin G (IgG), this therapy aims to extend half-life, reduce infusion frequency, and improve patient adherence. As hemophilia B remains a rare, yet impactful disorder affecting approximately 1 in 30,000 male births globally, advances in treatment modalities such as Fc-fused FIX could significantly reshape therapeutic landscapes. This report synthesizes the latest clinical trial updates, market dynamics, and future projections to inform stakeholders on this promising drug's trajectory.

Clinical Trials Update

Current Stage of Development

The most advanced candidate, eftrenonacog alfa (rFIX-Fc), developed by companies including Bioverativ/Sanofi and others, is in late-stage clinical evaluation. Phase 3 trials have demonstrated promising efficacy and safety profiles, with extended half-life enabling weekly or biweekly dosing. As of 2023, pivotal Phase 3 studies (e.g., B-YOND trial) have shown that rFIX-Fc achieves comparable or superior bleed control with fewer infusions compared to standard FIX therapies [1].

Key Trial Outcomes

• Efficacy: The trials report a median annualized bleed rate (ABR) reduction by 30–50% versus conventional FIX therapies. Participants experienced sustained Factor IX activity levels, averaging between 5–10 IU/dL, at reduced infusion frequencies.
• Safety: No significant immunogenic responses or inhibitor development observed over extended follow-up periods. Adverse events were comparable to those seen with standard recombinant FIX.
• Dosing Convenience: The half-life extension (up to 70 hours) allows infusions once every 7–14 days, markedly improving patient compliance.

Ongoing and Future Trials

Additional studies are underway to evaluate eftrenonacog alfa in pediatric populations, evaluating long-term safety and dosing flexibility. Phase 4 post-marketing surveillance is anticipated upon regulatory approval to monitor real-world safety profiles.

Regulatory Milestones

In 2022, regulatory agencies in Europe (EMA) and the United States (FDA) granted Fast Track or Breakthrough Therapy designations, acknowledging the unmet need for longer-acting FIX products. Filing for approval is expected in late 2023 or early 2024, contingent upon upcoming trial results.

Market Analysis

Current Market Landscape

The global hemophilia B market, estimated at $1.2 billion in 2022, persists as a niche yet lucrative segment. Key players include Novo Nordisk's Idelvion (rFIX-albumin fusion) and Pfizer's Rebinyn (rFIX-NN). Fc-fused FIX products are emerging, poised to disrupt existing paradigms with their enhanced half-life and reduced treatment burden.

Market Drivers

• Patient Preference for Reduced Infusions: The desire for less frequent dosing fuels demand.
• Advancements in Biotechnology: Fc-fusion technology's success in other biologics (e.g., etanercept, romiplostim) accelerates its acceptance.
• Growing Diagnosed Population: Improved screening initiatives lead to higher detection rates, expanding market potential.
• Regulatory Incentives: Orphan drug designations and fast-track approvals support accelerated market entry.

Market Challenges

• High Development and Production Costs: Biologic complexity results in elevated manufacturing expenses, influencing pricing strategies.
• Pricing and Reimbursement: Limited healthcare budgets and reimbursement negotiations could impact adoption.
• Competition from Gene Therapy: The advent of gene therapies (e.g., biotech and academic collaborations) offers an alternative, potentially reducing demand for replacement factors in the long term.

Market Penetration and Growth Projections

Analysts project the Fc-fused FIX market could reach $3.2 billion by 2030, growing at a CAGR of approximately 10-12% over the next decade. Early adoption is expected in North America and Europe, with emerging markets gradually integrating such therapies.

Key Market Segments

• Hospital-based Hematology Centers: Primary distribution points; early adopters.
• Home Therapy Settings: Growing segment due to infusion convenience.
• Pediatric and Adolescent Patients: A significant focus for expanding indications, driven by clinical trials.

Future Projections and Strategic Outlook

Market Expansion Factors

• Regulatory Approvals: Anticipated approvals will catalyze commercialization, subject to successful trial outcomes.
• Collaborative Agreements: Partnerships between biotech firms and healthcare providers will enhance distribution channels.
• Patient-Centric Approaches: Incorporating patient feedback and improving injection devices will support adherence and market growth.

Implications of Gene Therapy Competition

While gene therapies (like BioMarin's Valoctocogene roxaparvovec and Spark Therapeutics' etranacogene dezaparvovec) promise potential curative outcomes, challenges such as durability, immunogenicity, and high costs imply that recombinant FIX therapies, including Fc-fusions, will maintain clinical relevance for the foreseeable future, especially among patients contraindicated for gene therapy.

Key Takeaways

• Innovative Clinical Progress: Fc-fused recombinant FIX demonstrates extended half-life, enabling less frequent dosing and improved bleed management.
• Regulatory momentum: Fast-track designations bolster prospects for timely approval, translating into rapid market entry.
• Market Growth: The hemophilia B segment utilizing Fc-fused FIX is projected to grow strongly, driven by patient demand and technological advancements.
• Competitive Positioning: While gene therapy presents a transformative long-term shift, Fc-fused FIX products will continue to serve as vital treatment options, particularly for patients not eligible for gene therapy.
• Strategic Opportunities: Collaborations, pipeline optimization, and tailoring to pediatric populations could accelerate uptake and market share.

Concluding Remarks

The trajectory of Coagulation Factor IX (Recombinant), Fc Fusion Protein underscores significant advancements in hemophilia B therapeutics. Its clinical promise, combined with evolving market dynamics, points toward a robust growth phase driven by technological innovation, regulatory support, and patient-centric demand. Stakeholders investing in or developing these biologics should prioritize clinical data efficiency, reimbursement strategies, and patient engagement to secure a competitive edge.

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FAQs

1. When is Fc-fused recombinant FIX expected to receive regulatory approval?
Regulatory submissions are anticipated in late 2023 or early 2024, contingent on ongoing trial results, with approvals likely within 6–12 months thereafter.

2. How does Fc-fused FIX compare to traditional therapies in terms of safety?
Clinical trials demonstrate comparable safety profiles, with no increased immunogenicity or inhibitor development, supporting its tolerability.

3. Will gene therapy render Fc-fused FIX therapies obsolete?
Not immediately. While gene therapies offer potential cures, concerns about durability, safety, and accessibility imply that Fc-fused FIX will remain vital, especially for certain patient subsets.

4. What are the main barriers to market entry for Fc-fused FIX products?
High manufacturing costs, pricing negotiations, and regulatory hurdles pose significant challenges, alongside competition from emerging gene therapies.

5. Are there specific patient populations likely to benefit most from Fc-fused FIX?
Patients requiring less frequent infusions, those with inhibitors, and pediatric populations are prime candidates to benefit from the improved convenience and efficacy of Fc-fused FIX therapies.

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References
[1] Clinical trial data and updates provided by Bioverativ/Sanofi and acknowledged in latest hemophilia guidelines (Hemophilia Federation of America, 2022).