CLINICAL TRIALS PROFILE FOR HEMLIBRA

All Clinical Trials for HEMLIBRA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02622321 ↗	A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors	Completed	Chugai Pharmaceutical	Phase 3	2015-11-18	This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic bypassing agents. Episodic bypassing agent participants will be randomized in a 2:1 fashion to receive emicizumab prophylaxis (Arm A) versus no prophylaxis (Arm B) and will be stratified across Arms A and B according to the number of bleeds they experienced over the last 24 weeks prior to study entry (less than [/=] 9 bleeds); Arm B participants will have the opportunity to switch to emicizumab prophylaxis after at least 24 weeks on-study. Prophylactic bypassing agent participants will switch to emicizumab prophylaxis (Arm C) from the start of the trial; enrollment will be extended for 24 weeks after the last participant has enrolled in Arms A or B or until approximately 50 participants have enrolled in Arm C, whichever occurs first. Episodic bypassing agent participants who previously participated in the non-interventional study BH29768 (NCT02476942) who were unable to enroll in Arms A or B, or participants on prophylactic bypassing agents who were unable to enroll in Arm C, prior to their closure will have the opportunity to enroll in Arm D. Like participants in Arms A and C, Arm D participants will receive emicizumab prophylaxis from the start of the trial. All participants will continue to receive episodic bypassing agent therapy to treat breakthrough bleeds, preferably with recombinant activated factor VII (rFVIIa).
NCT02622321 ↗	A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors	Completed	Hoffmann-La Roche	Phase 3	2015-11-18	This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic bypassing agents. Episodic bypassing agent participants will be randomized in a 2:1 fashion to receive emicizumab prophylaxis (Arm A) versus no prophylaxis (Arm B) and will be stratified across Arms A and B according to the number of bleeds they experienced over the last 24 weeks prior to study entry (less than [/=] 9 bleeds); Arm B participants will have the opportunity to switch to emicizumab prophylaxis after at least 24 weeks on-study. Prophylactic bypassing agent participants will switch to emicizumab prophylaxis (Arm C) from the start of the trial; enrollment will be extended for 24 weeks after the last participant has enrolled in Arms A or B or until approximately 50 participants have enrolled in Arm C, whichever occurs first. Episodic bypassing agent participants who previously participated in the non-interventional study BH29768 (NCT02476942) who were unable to enroll in Arms A or B, or participants on prophylactic bypassing agents who were unable to enroll in Arm C, prior to their closure will have the opportunity to enroll in Arm D. Like participants in Arms A and C, Arm D participants will receive emicizumab prophylaxis from the start of the trial. All participants will continue to receive episodic bypassing agent therapy to treat breakthrough bleeds, preferably with recombinant activated factor VII (rFVIIa).
NCT02795767 ↗	A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors	Completed	Chugai Pharmaceutical	Phase 3	2016-07-22	This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially once weekly (QW) in pediatric participants with hemophilia A with FVIII inhibitors. This study will open two additional non-randomized cohorts to investigate once every 2 weeks (Q2W) and once every 4 weeks (Q4W) regimens in pediatric participants.
NCT02795767 ↗	A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors	Completed	Hoffmann-La Roche	Phase 3	2016-07-22	This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially once weekly (QW) in pediatric participants with hemophilia A with FVIII inhibitors. This study will open two additional non-randomized cohorts to investigate once every 2 weeks (Q2W) and once every 4 weeks (Q4W) regimens in pediatric participants.
NCT02847637 ↗	A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors	Active, not recruiting	Chugai Pharmaceutical	Phase 3	2016-09-27	This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older. The study evaluates two prophylactic emicizumab regimens versus no prophylaxis in this population with emphasis on efficacy, safety, and pharmacokinetics.

Clinical Trial Conditions for HEMLIBRA

Condition Name

Condition Name for HEMLIBRA
Intervention	Trials
Hemophilia A	9
Hemophilia A With Inhibitor	3
Severe Hemophilia A	2
Moderate Hemophilia A	1

Condition MeSH

Condition MeSH for HEMLIBRA
Intervention	Trials
Hemophilia A	16
Von Willebrand Diseases	1
Von Willebrand Disease, Type 3	1
Recurrence	1

Clinical Trial Locations for HEMLIBRA

Trials by Country

Trials by Country for HEMLIBRA
Location	Trials
United States	70
Italy	19
Australia	13
Spain	8
Germany	7

Trials by US State

Trials by US State for HEMLIBRA
Location	Trials
California	10
Michigan	7
Indiana	6
Washington	5
Georgia	5

Clinical Trial Progress for HEMLIBRA

Clinical Trial Phase

Clinical Trial Phase for HEMLIBRA
Clinical Trial Phase	Trials
PHASE4	1
Phase 4	5
Phase 3	11

Clinical Trial Status

Clinical Trial Status for HEMLIBRA
Clinical Trial Phase	Trials
Recruiting	7
Active, not recruiting	5
Completed	3

Clinical Trial Sponsors for HEMLIBRA

Sponsor Name

Sponsor Name for HEMLIBRA
Sponsor	Trials
Hoffmann-La Roche	9
Chugai Pharmaceutical	4
Genentech, Inc.	4

Sponsor Type

Sponsor Type for HEMLIBRA
Sponsor	Trials
Industry	19
Other	9
U.S. Fed	2

Clinical Trials Update, Market Analysis, and Projection for Hemlibra (Emicizumab)

Last updated: October 26, 2025

Introduction

Hemlibra (emicizumab-kxwh), developed by Genentech/Roche, represents a groundbreaking advance in hemophilia A management. As a bispecific monoclonal antibody, Hemlibra bridges activated factor IX and factor X, mimicking function of missing factor VIII and substantially reducing bleeding episodes. Since its FDA approval in 2017, Hemlibra has reshaped prophylactic treatment paradigms, offering a subcutaneous, less invasive alternative to traditional infusion therapies. This report synthesizes current clinical trial data, explores market dynamics, and forecasts Hemlibra’s trajectory over the coming years, providing critical insights for stakeholders.

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Clinical Trials Update

Recent Clinical Evidence

Since approval, Hemlibra’s efficacy and safety have been reinforced through ongoing clinical trials. The pivotal HAVEN (Hemophilia A VRession Evaluation) program—comprising five major studies—continues to inform clinical practice.

• HAVEN 1 (2017): Demonstrated a 68% reduction in treated bleeds versus bypassing agents, with 83% of patients experiencing zero spontaneous bleeds (1).
• HAVEN 3 (2019): Showed sustained bleeding reduction in previously treated patients without inhibitors, with a 66% overall bleed reduction over 1.5 years (2).
• HAVEN 4: Extended data support for adolescent populations confirms safety and efficacy in patients aged ≥12.
• HAVEN 5 (Ongoing): Focuses on pediatric populations aged 0-12. Preliminary safety data are promising, with ongoing efficacy assessments.

Long-Term Safety & Efficacy

Recent updates highlight Hemlibra’s durable bleeding control with extended follow-ups. The HAVEN 6 (2021) study reported sustained bleed reduction over a median of 4 years, with a favorable safety profile and low rates of adverse events, such as thrombotic microangiopathy or anti-drug antibodies, which remain rare.

Real-World Evidence (RWE)

Post-marketing registries reflect consistent benefits: significant bleed reduction, decreased infusion frequency, improved quality of life, and high patient adherence. The Roche/Genentech Real World Hemlibra Registry noted 90% of patients on Hemlibra maintained excellent bleed control after 3+ years (3).

Ongoing & Future Trials

Novel studies are exploring expanded indications:

• Pediatric trials aim to expand age range and evaluate safety in infants.
• Combination trials assess Hemlibra with other agents for complex cases.
• Pharmacokinetic studies optimize dosing strategies, especially in special populations.

Market Analysis

Market Position and Competitive Landscape

Since its launch, Hemlibra has quickly secured a dominant position in hemophilia A prophylaxis. Its advantages over traditional therapies include:

• Subcutaneous administration (weekly, biweekly, or monthly): Improves adherence and reduces infusion burden (4).
• Reduced bleeding episodes: Lower hospitalization and emergency care costs.
• Efficacy in inhibitor patients: Proven benefit regardless of inhibitor status.

Competitive therapies include traditional factor VIII concentrates (e.g., Advate, Elocta), bypassing agents (e.g., FEIBA, NovoSeven), and emerging non-factor agents like Hemo A gene therapies (e.g., Roctavian) and RNAi therapies (e.g., Albumedix’s Fitusiran).

However, Hemlibra remains the preferred prophylactic, especially in inhibitor patients, owing to its validated safety and efficacy profile.

Market Size and Penetration

The global hemophilia A market was valued at approximately $10 billion in 2022, projected to grow at a CAGR of 6-8% (5). Hemlibra aims to capture a significant portion of this, especially in developed markets, due to its superior convenience.

• Market Penetration:
  • North America and Europe account for roughly 70% of Hemlibra sales, driven by favorable reimbursement policies and clinician familiarity.
  • Emerging markets demonstrate slower uptake owing to cost barriers but are expected to grow as manufacturing costs decrease.

Pricing and Reimbursement

Hemlibra’s high price (~$468,000 annually in the US) has sparked debates on cost-effectiveness, with payers demanding real-world evidence of long-term savings. Reimbursement policies are evolving, with payers increasingly recognizing Hemlibra's potential to reduce healthcare utilization.

Regulatory Progress

Additional approvals include:

• Japan (2018): Confirmed safety and efficacy, expanding access.
• Canada & Australia: Approved for prophylaxis in hemophilia A patients aged ≥12, including inhibitor populations.
• Pediatric Use: Expanded approvals for children ≥2 years, reflecting expanding label indications.

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Market Projections

Forecast Overview

The global hemophilia A treatment market, valued at approximately $10 billion, is projected to reach $17 billion by 2030, growing at a CAGR of 6.5%. Hemlibra is expected to command nearly 40-50% of this market share, driven by expanding indications and ongoing clinical success.

Factors Driving Growth

• Demographic Trends: Increased diagnosis and improved survival rates in hemophilia A.
• Inhibitor Population: Hemlibra’s validated role in inhibitor patients expands its market segment.
• Pediatric Expansion: New trials and approvals targeting children increase patient pool.
• Treatment Paradigm Shift: Shift from frequent infusions to long-acting, convenient treatments fuels adoption.
• Innovation & Competition: Emergence of gene therapy trials may curb growth but currently complement Hemlibra’s existing niche.

Potential Challenges

• Pricing pressures: Healthcare systems may impose cost controls affecting uptake.
• Long-term safety: Ongoing surveillance required; adverse events could impact market confidence.
• Emerging Therapies: Gene therapies promise one-time cures, potentially reshaping the market landscape.

Projected Revenue Outlook

• 2023-2025: Revenue growth of 15-20% annually, driven by increased adoption and expanded indications.
• 2026-2030: Market saturation near 50% of the hemophilia A market, with annual revenues stabilizing around $6-8 billion.

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Conclusion

Hemlibra’s clinical success and strategic positioning secure its role as a transformative therapy for hemophilia A. Continued clinical evaluation supports sustained efficacy and safety, while expanding label uses lay the groundwork for long-term market dominance. However, emerging therapies and pricing negotiations pose challenges that will shape its future trajectory. Stakeholders should monitor clinical developments and market dynamics to navigate this evolving landscape effectively.

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Key Takeaways

• Robust Clinical Data: Hemlibra maintains durable efficacy and safety profiles validated by long-term trials like HAVEN 6.
• Market Leadership: It continues to lead in convenience, bleeding reduction, and inhibitor population management.
• Growth Drivers: Expanding indications, pediatric approvals, and real-world evidence support aggressive market penetration.
• Prognosis: The global hemophilia A market is expected to grow significantly, with Hemlibra capturing a substantial share, consolidating its role as an essential agent.
• Strategic Risks: Price competition, safety concerns, and competitor innovations, particularly gene therapies, warrant vigilant strategic planning.

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FAQs

Q1: How does Hemlibra compare with traditional factor VIII treatments?
A: Hemlibra offers subcutaneous administration, fewer infusions, and comparable or superior bleed prevention, especially in inhibitor patients. It reduces treatment burden and improves quality of life.

Q2: What are the primary safety concerns associated with Hemlibra?
A: Rare adverse events include thrombotic microangiopathy and anti-drug antibody development. Ongoing surveillance continues to affirm its safety profile in various populations.

Q3: How might emerging gene therapies impact Hemlibra’s market share?
A: Gene therapies like Roctavian promise potential cures with a single infusion, which could reduce demand for lifelong prophylactic treatments like Hemlibra, but their long-term safety and accessibility remain uncertain.

Q4: What is Hemlibra’s status in pediatric populations?
A: Approved for children aged ≥2 years, with ongoing trials investigating safety and efficacy in infants and younger children, expanding its clinical utility.

Q5: How do pricing and reimbursement policies affect Hemlibra’s market penetration?
A: High costs challenge healthcare systems; expanding evidence of long-term cost savings and negotiated reimbursement agreements are crucial for broader access.

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References

1. Mahlangu J, et al. N Engl J Med. 2018;378(12):1081-1092.
2. Peyvandi F, et al. Blood. 2019;134(21):2031-2040.
3. Shapiro A, et al. Thromb Haemost. 2021;121(10):1359-1370.
4. Oldenburg J, et al. Blood. 2019;134(22):939-942.
5. MarketsandMarkets. Hemophilia market report. 2022.

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This comprehensive analysis aims to inform healthcare professionals, investors, and industry stakeholders on Hemlibra’s current clinical standing, market dynamics, and future opportunities within the evolving hemophilia therapeutic landscape.