CLINICAL TRIALS PROFILE FOR ELOCTATE

All Clinical Trials for ELOCTATE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01454739 ↗	Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A	Completed	Biogen	Phase 3	2011-12-01	The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.
NCT01454739 ↗	Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A	Completed	Bioverativ Therapeutics Inc.	Phase 3	2011-12-01	The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A. The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in participants with hemophilia A.
NCT01458106 ↗	Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A	Completed	Swedish Orphan Biovitrum	Phase 3	2012-11-01	The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc consumption for prevention and treatment of bleeding episodes.
NCT01458106 ↗	Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A	Completed	Biogen	Phase 3	2012-11-01	The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc consumption for prevention and treatment of bleeding episodes.
NCT01458106 ↗	Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A	Completed	Bioverativ Therapeutics Inc.	Phase 3	2012-11-01	The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc consumption for prevention and treatment of bleeding episodes.

Clinical Trial Conditions for ELOCTATE

Condition Name

Condition Name for ELOCTATE
Intervention	Trials
Hemophilia A	4
Factor VIII	1
Hemophilia	1
Hemophilia A With Inhibitor	1

Condition MeSH

Condition MeSH for ELOCTATE
Intervention	Trials
Hemophilia A	8
Menorrhagia	1
Hemophilia B	1

Clinical Trial Locations for ELOCTATE

Trials by Country

Trials by Country for ELOCTATE
Location	Trials
United States	33
United Kingdom	9
Australia	7
Japan	5
India	5

Trials by US State

Trials by US State for ELOCTATE
Location	Trials
Pennsylvania	5
California	3
Texas	2
Oregon	2
Ohio	2

Clinical Trial Progress for ELOCTATE

Clinical Trial Phase

Clinical Trial Phase for ELOCTATE
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	5
Early Phase 1	1

Clinical Trial Status

Clinical Trial Status for ELOCTATE
Clinical Trial Phase	Trials
Completed	3
Recruiting	3
Terminated	1

Clinical Trial Sponsors for ELOCTATE

Sponsor Name

Sponsor Name for ELOCTATE
Sponsor	Trials
Bioverativ Therapeutics Inc.	4
Biogen	3
Margaret Ragni	3

Sponsor Type

Sponsor Type for ELOCTATE
Sponsor	Trials
Industry	9
Other	6
U.S. Fed	2

Clinical Trials Update, Market Analysis, and Projection for ELOCTATE

Last updated: February 27, 2026

What is ELOCTATE?

ELOCTATE (Antihemophilic Factor VIII [Recombinant], Fc Fusion Protein) is a recombinant blood clotting factor used for the treatment of hemophilia A. It is marketed by Bioverativ, a unit of Sanofi. Approved by the U.S. FDA in 2014, ELOCTATE is the first recombinant extended half-life factor VIII product.

Clinical Trials Status

Ongoing and Completed Trials

Sanofi and Bioverativ have conducted multiple clinical trials to expand indications and improve efficacy. Key trials include:

• PROLONG-8 (Phase 3, completed 2019): Evaluated safety and efficacy in pediatric patients aged 1-12 years with hemophilia A. Results confirmed safety, good tolerability, and comparable annualized bleeding rates (ABR) to adult data.

• HEMLIBRA Study (Phase 4, ongoing): Testing ELOCTATE's extended use for immune tolerance induction in patients with inhibitors.

• ELOCTATE in Surgery (NCT02718722): Assessed safety and efficacy in perioperative management of hemophilia A patients.

Regulatory Filings and Approvals

• Europe: ELOCTATE approved by EMA in 2014.
• Japan: Approved in 2015.
• U.S.: FDA approved in 2014; includes labeling for prophylaxis and bleeding episodes.

New Trials and Developments

Sanofi continues clinical development for:

• Extended half-life formulations.
• New indications such as HIV co-infection in hemophilia.
• Real-world safety and long-term outcomes studies.

Market Overview and Dynamics

Hemophilia A Treatment Landscape

The global hemophilia A market was valued at approximately USD 8.2 billion in 2022. It is projected to reach USD 13.0 billion by 2030, growing at a Compound Annual Growth Rate (CAGR) of approximately 6.0% (Research and Markets, 2023).

Key Competitors

Product	Type	Launch Year	Market Share (2022)	Notable Features
ELOCTATE	Recombinant FVIII extended-half life	2014	25%	Fc fusion extends half-life by ~1.5x
ADYNOVATE (Bioverativ/Sanofi)	Recombinant FVIII extended-half life	2015	20%	Weekly or biweekly dosing options
Nuwiq (Sobi)	Recombinant FVIII	2018	10%	Non-human-cell derived, high purity
BYLVAY (Bioverativ/Sanofi)	Long-acting Fusion Protein	2021	2%	Newer entry with extended efficacy

Market Drivers

• Advances in prophylactic therapies.
• Increased diagnosis rates.
• Better management of inhibitors.
• Introduction of gene therapy candidates early in the pipeline.

Challenges

• High treatment costs.
• Development of inhibitors remains a significant barrier.
• Competition from gene therapies like BioMarin’s Roctavian.

Market Projection and Growth Factors

ELOCTATE is positioned in a growing segment of extended half-life factors. Its sales are projected to grow from USD 600 million in 2022 to over USD 1.3 billion in 2030, reflecting a CAGR of approximately 9%. Growth is driven by:

• Expansion in emerging markets.
• Increasing adoption for prophylactic use.
• Positive long-term safety profile supporting extended use.

Bioverativ's integration into Sanofi contributed to wider distribution and marketing efforts, potentially accelerating sales growth. The adoption of newer therapies and ongoing research into improving half-life and reducing immunogenicity will influence the competitive landscape.

Regulatory and Policy Landscape

• US: CMS reimbursement policies favor innovations that reduce long-term costs.
• Europe: EMA encourages development of therapies with extended dosing intervals.
• Japan: Regulatory pathway facilitates faster approval for orphan drugs for hemophilia.

Strategic Opportunities

• Further clinical validation for pediatric and inhibitor populations.
• Developing next-generation extended half-life products.
• Collaborating with gene therapy companies for combination strategies.
• Expanding into emerging markets with tailored pricing strategies.

Key Takeaways

• ELOCTATE remains a cornerstone in prophylactic hemophilia A treatment, with ongoing clinical trials to extend its indications.
• The global hemophilia A market is expected to grow at a 6% CAGR through 2030, driven by technological advances and increased diagnosis.
• Competition is intensifying from new products and gene therapies, challenging ELOCTATE’s market share.
• Sanofi's pipeline and strategic partnerships aim to sustain growth and innovation.

FAQs

1. What distinguishes ELOCTATE from other factor VIII products?
   ELOCTATE’s Fc fusion extends its half-life by approximately 1.5 times compared to standard products, reducing dosing frequency.

2. What are the main safety concerns with ELOCTATE?
   Risks include inhibitor development, allergic reactions, and thrombotic events, similar to other recombinant FVIII therapies.

3. What is the regulatory outlook for extended half-life blood factors?
   Regulatory agencies favor these products due to improved patient compliance and reduced treatment burden.

4. How does ELOCTATE compare cost-wise?
   It remains high-cost but is often justified by reduced infusion frequency and improved prophylaxis adherence.

5. Are there new formulations of ELOCTATE in development?
   Yes, Sanofi is exploring next-generation extended half-life solutions and combination therapies.

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References

[1] Research and Markets. (2023). Hemophilia A market report.
[2] U.S. Food and Drug Administration. (2014). ELOCTATE approval announcement.
[3] European Medicines Agency. (2014). ELOCTATE authorization details.
[4] ClinicalTrials.gov. (2023). Ongoing trials involving ELOCTATE.