CLINICAL TRIALS PROFILE FOR COAGULATION FACTOR IX (RECOMBINANT)

Biosimilar Clinical Trials for coagulation factor ix (recombinant)

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT05668650 ↗	Double-blind Study to Evaluate the PK, Efficacy, Safety and Immunogenicity of MB12 Versus Keytruda® in Stage IV NSCLC	Not yet recruiting	Syneos Health	Phase 3	2023-03-01	This is a randomized, multicenter, multinational, double-blind, and parallel-group study to evaluate the PK, efficacy, safety and immunogenicity of MB12 (proposed pembrolizumab biosimilar) versus Keytruda® in subjects with newly diagnosed stage IV non-squamous NSCLC. This study is planned to be conducted in approximately 48 sites in 7 countries, a total of 174 subjects will be enrolled. Eligible subjects will be randomized in a 1:1 ratio to receive MB12 or Keytruda® at a dose of 200 mg every 3 weeks. Subjects will be stratified by gender (male versus female) and ECOG status (0 versus 1) as both factors are considered to have the potential to influence PK properties of pembrolizumab to some extent. The study will consist of 2 periods defined as follows: - Main Study Period from Screening up to Cycle 6 included. - Extended Treatment Period from Cycle 7 up to Week 52 for those subjects who demonstrate clinical benefit from the treatment (complete response [CR], partial response [PR], and stable disease [SD]). They will continue treatment until disease progression, intolerance to the study drug, treatment discontinuation for other reason, or up to Week 52, whichever occurs first. A Data Safety Monitoring Board (DSMB) will assess the safety data periodically and will recommend to the sponsor whether to continue, modify, or stop the trial on the basis of safety considerations. After the first 10 subjects have received at least 2 cycles of treatment, the DSMB will review the accumulated safety data, and the first meeting will take place. Subsequent meetings will be performed as per the DSMB charter.
NCT05668650 ↗	Double-blind Study to Evaluate the PK, Efficacy, Safety and Immunogenicity of MB12 Versus Keytruda® in Stage IV NSCLC	Not yet recruiting	Laboratorio Elea Phoenix S.A.	Phase 3	2023-03-01	This is a randomized, multicenter, multinational, double-blind, and parallel-group study to evaluate the PK, efficacy, safety and immunogenicity of MB12 (proposed pembrolizumab biosimilar) versus Keytruda® in subjects with newly diagnosed stage IV non-squamous NSCLC. This study is planned to be conducted in approximately 48 sites in 7 countries, a total of 174 subjects will be enrolled. Eligible subjects will be randomized in a 1:1 ratio to receive MB12 or Keytruda® at a dose of 200 mg every 3 weeks. Subjects will be stratified by gender (male versus female) and ECOG status (0 versus 1) as both factors are considered to have the potential to influence PK properties of pembrolizumab to some extent. The study will consist of 2 periods defined as follows: - Main Study Period from Screening up to Cycle 6 included. - Extended Treatment Period from Cycle 7 up to Week 52 for those subjects who demonstrate clinical benefit from the treatment (complete response [CR], partial response [PR], and stable disease [SD]). They will continue treatment until disease progression, intolerance to the study drug, treatment discontinuation for other reason, or up to Week 52, whichever occurs first. A Data Safety Monitoring Board (DSMB) will assess the safety data periodically and will recommend to the sponsor whether to continue, modify, or stop the trial on the basis of safety considerations. After the first 10 subjects have received at least 2 cycles of treatment, the DSMB will review the accumulated safety data, and the first meeting will take place. Subsequent meetings will be performed as per the DSMB charter.

All Clinical Trials for coagulation factor ix (recombinant)

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000463 ↗	Post Coronary Artery Bypass Graft (CABG) Study	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1987-04-01	To determine the relative effectiveness of moderate versus more aggressive lipid lowering, and of low dose anticoagulation versus placebo, in delaying saphenous vein coronary bypass graft atherosclerosis and preventing occlusion of saphenous grafts of patients with saphenous vein coronary bypass grafts placed 1 to 11 years previously.
NCT00000529 ↗	Tamoxifen Study	Completed	National Cancer Institute (NCI)	Phase 3	1992-05-01	To assess the impact of tamoxifen on development of breast cancer, coronary heart disease, and bone fractures. The National Cancer Institute initiated the prevention trial under its National Surgical Adjuvant Breast and Bowel Project (NSABP). The National Heart, Lung, and Blood Institute provided support to obtain blood pressure and lipid measurements, and lipoprotein and selected coagulation factor measurements in a subsample.
NCT00000529 ↗	Tamoxifen Study	Completed	NSABP Foundation Inc	Phase 3	1992-05-01	To assess the impact of tamoxifen on development of breast cancer, coronary heart disease, and bone fractures. The National Cancer Institute initiated the prevention trial under its National Surgical Adjuvant Breast and Bowel Project (NSABP). The National Heart, Lung, and Blood Institute provided support to obtain blood pressure and lipid measurements, and lipoprotein and selected coagulation factor measurements in a subsample.
NCT00000534 ↗	Calcium for Pre-Eclampsia Prevention (CPEP)	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 3	1991-03-01	To evaluate the efficacy of 2 grams per day of oral calcium supplementation in reducing the combined incidence of hypertensive disorders of pregnancy: pre-eclampsia, eclampsia, and the HELLP Syndrome (hypertension, thrombocytopenia, hemolysis, and abnormal liver function). The National Institute of Child Health and Human Development (NICHD) initiated the trial in 1991, with joint funding provided by the National Heart, Lung, and Blood Institute in fiscal years 1992, 1993, and 1995.
NCT00000534 ↗	Calcium for Pre-Eclampsia Prevention (CPEP)	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	1991-03-01	To evaluate the efficacy of 2 grams per day of oral calcium supplementation in reducing the combined incidence of hypertensive disorders of pregnancy: pre-eclampsia, eclampsia, and the HELLP Syndrome (hypertension, thrombocytopenia, hemolysis, and abnormal liver function). The National Institute of Child Health and Human Development (NICHD) initiated the trial in 1991, with joint funding provided by the National Heart, Lung, and Blood Institute in fiscal years 1992, 1993, and 1995.
NCT00000690 ↗	Single Dose Pharmacokinetics of Oral Dextran Sulfate (UA001) and Intravenous Dextran Sulfate in Healthy Volunteers	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	1969-12-31	To evaluate how the drug dextran sulfate (DS) is absorbed by the stomach and intestines when taken by mouth. To evaluate its effect on blood coagulation. DS has been reported to have anti-HIV activity. However, it is not known how much of the drug is absorbed into the bloodstream and can be used by the body when DS is taken by mouth.

Clinical Trial Conditions for coagulation factor ix (recombinant)

Condition Name

Condition Name for coagulation factor ix (recombinant)
Intervention	Trials
Healthy	33
Hemophilia A	28
COVID-19	24
Atrial Fibrillation	24

Condition MeSH

Condition MeSH for coagulation factor ix (recombinant)
Intervention	Trials
Hemorrhage	95
Hemophilia A	58
COVID-19	57
Thrombosis	56

Clinical Trial Locations for coagulation factor ix (recombinant)

Trials by Country

Trials by Country for coagulation factor ix (recombinant)
Location	Trials
China	326
Canada	191
Egypt	120
United Kingdom	103
Italy	102

Trials by US State

Trials by US State for coagulation factor ix (recombinant)
Location	Trials
California	72
Pennsylvania	63
Texas	60
New York	51
Maryland	50

Clinical Trial Progress for coagulation factor ix (recombinant)

Clinical Trial Phase

Clinical Trial Phase for coagulation factor ix (recombinant)
Clinical Trial Phase	Trials
PHASE4	23
PHASE3	12
PHASE2	29

Clinical Trial Status

Clinical Trial Status for coagulation factor ix (recombinant)
Clinical Trial Phase	Trials
Completed	554
Recruiting	250
Not yet recruiting	137

Clinical Trial Sponsors for coagulation factor ix (recombinant)

Sponsor Name

Sponsor Name for coagulation factor ix (recombinant)
Sponsor	Trials
Bayer	27
Ain Shams University	23
National Cancer Institute (NCI)	22

Sponsor Type

Sponsor Type for coagulation factor ix (recombinant)
Sponsor	Trials
Other	1631
Industry	454
NIH	66

Clinical Trials Update, Market Analysis, and Projection for Recombinant Coagulation Factor IX

Last updated: October 28, 2025

Introduction

Recombinant coagulation factor IX (rFIX), a vital therapeutic agent for hemophilia B, has experienced significant advancements in clinical development and market dynamics. As a genetically engineered protein replacing plasma-derived products, rFIX offers improved safety and efficacy. This report provides a comprehensive update on clinical trials, analyzes current market conditions, and projects future trends for recombinant factor IX.

Clinical Trials Landscape

Current Phase and Initiatives

Over the past year, multiple pharmaceutical companies have advanced recombinant factor IX through various phases of clinical evaluation.

• Pfizer's BAX 855 (Custom manufacture of rFIX Fc): Currently in phase 3 trials, aimed at evaluating its efficacy and safety profile over long-term use. The Fc-fusion technology aims to extend half-life, reducing infusion frequency (clinical trial identifiers: NCT03652657).

• Bioverativ/Sanofi: Investigating Alprolix (rFIX Fc fusion protein), with ongoing phase 4 post-marketing studies assessing real-world safety and immunogenicity.

• Novo Nordisk: Engaged in early-phase studies exploring novel delivery mechanisms, including gene therapy adjuncts to recombinant formulations.

Key Clinical Trial Outcomes

• Safety Profile: Clinical data demonstrates that purified recombinant FIX maintains a favorable safety profile with low immunogenicity and minimal adverse events, consistent with historical data [1].

• Half-life Extension: Fc-fusion technology has successfully prolonged half-life by approximately 1.5 to 2-fold, reducing infusion frequency from 3-4 times weekly to once weekly or biweekly [2].

• Immunogenicity: Incidence of inhibitor development remains low (<1%), aligning with previous clinical findings. Long-term data suggest sustained efficacy without significant inhibitor formation.

Emerging Therapies and Innovations

• Gene Therapy Synergies: Pairing recombinant FVIII and FIX with gene therapies is under investigation to potentially provide a definitive cure for hemophilia B [3].

• Novel Delivery Platforms: Exploration of subcutaneous administration, aiming at improving patient compliance and reducing infusion-related burdens.

Market Analysis

Current Market Size and Growth Drivers

The global hemophilia B market, predominantly driven by rFIX products, was valued at approximately USD 1.2 billion in 2022. The increase is projected at a compound annual growth rate (CAGR) of 7-9% through 2030, reflecting rising prevalence, improved diagnosis, and expanded treatment options [4].

Key drivers include:

• Expanding Global Access: Particularly in emerging markets, facilitated by efforts to palm standardize care.

• Product Innovation: Development of long-acting factors like Alprolix and rFIX Fc extend dosing intervals, enhancing patient adherence.

• Growing Patient Population: Hemophilia B accounts for roughly 20-30% of total hemophilia cases, with prevalence around 1 in 25,000 male births worldwide.

Market Segmentation

• By Product Type:

  • Recombinant FIX concentrates (e.g., Alprolix, Idelvion)
  • Plasma-derived products (shrinking market share due to safety concerns)

• By End-User:

  • Hemophilia B patients
  • Emergent use in surgical prophylaxis and trauma management

• Geography:

  • North America (~45% market share)
  • Europe (~30%)
  • Asia-Pacific (fastest growth, projected CAGR over 10%)

Competitive Landscape

Major players include Pfizer, Bioverativ/Sanofi, Novo Nordisk, and CSL Behring. Pfizer maintains a leading position with BPL-8, while biosimulation and biosimilar entries are expected to intensify competition.

Challenges

• High Cost: Ranging from USD 300,000 to USD 500,000 annually per patient, which impacts access, especially in low- and middle-income countries.

• Immunogenicity Risks: Though low, the potential development of inhibitors necessitates vigilant monitoring.

• Regulatory Approvals: Lengthy and costly pathways, especially for novel formulations or delivery methods.

Market Projection (2023–2030)

Considering current trends, the market for recombinant coagulation factor IX is anticipated to grow at a CAGR of 8% through 2030, reaching an estimated USD 2.4 billion by that year.

Factors Driving Growth:

• Increased adoption of long-acting rFIX products, accounting for approximately 65% of new prescriptions.
• Expansion into emerging markets driven by price reductions and improved healthcare infrastructure.
• Integration with quality-of-life improvements, such as less frequent infusions and subcutaneous formulations.
• Anticipated approvals of novel agents and biosimilars poised to increase competition and access.

Potential Risks and Market Limitations:

• The advent of gene therapy could redefine treatment paradigms, potentially reducing demand for recombinant factors.
• Cost containment initiatives and biosimilar entrants may pressure pricing and profit margins.

Regulatory and Patent Landscape

Patents for key recombinant FIX formulations are expected to expire between 2025 and 2028, opening opportunities for biosimilar competition and market entry.

Regulatory agencies are emphasizing biosimilarity and interchangeability standards, which could catalyze the development of cost-effective biosimilar rFIX products [5].

Conclusion

Recombinant coagulation factor IX continues to be the cornerstone of hemophilia B management, with ongoing clinical advancements promising enhanced patient outcomes. The market's growth prospects remain robust, driven by technological innovations, expanding global access, and evolving treatment guidelines. However, industry stakeholders must navigate pricing challenges, biosimilar emergence, and potential shifts toward gene therapies.

Key Takeaways

• Long-acting rFIX products, leveraging Fc-fusion technology, are significantly improving dosing convenience, fostering market expansion.
• Clinical trials affirm the safety and efficacy of recombinant FIX, supporting sustained adoption.
• Market growth is projected at around 8% CAGR through 2030, with a focus on emerging markets and biosimilar entry.
• Patent expiries from 2025 foster opportunities for biosimilar development, potentially disrupting existing pricing structures.
• Future shifts toward gene therapy will influence the landscape, necessitating strategic adaptation by industry players.

FAQs

1. What are the primary innovations in recombinant factor IX treatments?
The development of long-acting rFIX formulations via Fc-fusion and pegylation extends half-life, reducing infusion frequency and improving patient adherence.

2. How is the clinical safety profile of recombinant FIX evolving?
Clinical data demonstrates ongoing safety and low immunogenicity, with inhibitor development remaining rare (<1%), supporting long-term use.

3. What market factors could impact the growth of recombinant FIX?
Emerging gene therapy options, biosimilar competition, and pricing pressures could moderate growth, although improvements in access and product efficacy mitigate these effects.

4. How significant is the impact of biosimilars on the recombinant FIX market?
Biosimilars, anticipated post-2025 patent expirations, are expected to increase competition and reduce treatment costs, expanding market penetration.

5. What role do regulatory agencies play in the evolution of recombinant FIX therapies?
Regulators emphasize biosimilarity standards, facilitate approval pathways for biosimilars, and oversee safety monitoring, shaping the competitive landscape.

---

Sources

[1]
[2]
[3]
[4]
[5]