CLINICAL TRIALS PROFILE FOR ADYNOVATE

All Clinical Trials for ADYNOVATE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04690322 ↗	POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies	Recruiting	Jessica Garcia	Phase 4	2021-04-15	This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both standard of care treatment for persons with Hemophilia A.
NCT04784988 ↗	Intensive Replacement Treatment in Haemophilia Patients With Synovitis	Not yet recruiting	Federico II University	Phase 4	2021-03-01	Background: Joint haemorrhage represents the most common type of bleeding episode in persons with hemophilia (PwH). In the absence of an adequate prophylaxis with Factor VIII (for hemophilia A) or FIX (for hemophilia B) concentrates up to 85% of patients with severe hemophilia develop a clinically overt joint disease. Screening of early signs of arthropathy is needed. Synovitis is widely considered as one of the parameters to be taken into account for the diagnosis and the surveillance of joint impairment in PwH. Aim: To assess if an intensive factor VIII replacement treatment is able at reverting synovitis in PwH. Methods: The present study is a randomized, open-label, cross-over study. Among patients referred to enrolling Haemophilia Centres, consecutive patients with severe (FVIII < 1%) or severe-moderate (FVIII < 2%) haemophilia A without inhibitors will be enrolled. The present study will be organized in 2 phases. - Phase 1 (US screening): All patients will undergo an ultrasound examination of elbows, ankles and knees to define joint status and to identify presence/absence of synovitis according to the HEAD-US system. - Phase 2 (Intervention): Patients with US evidence of synovitis will be randomly assigned at undergoing a PK assessment with my-PK-fit to start a prophylaxis with Adynovi® targeting a 12% FVIII through level (PROPEL-like arm) or to continue ongoing standard treatment (control arm). US examination of the six joints will be repeated monthly for six months and in case of onset of symptoms that might suggest an acute bleeding episode. After six months the two treatment arm will be switched in the frame of a cross-over approach and all PwH will be followed for other 6 months The primary outcome will be represented by changes in synovial status during the intensive factor VIII replacement treatment vs standard treatment.
NCT05281185 ↗	Clinical Outcomes of Low Dose PK-guided EHL FVIII Concentrates Versus Standard Prophylaxis in Severe Haemophilia A	Active, not recruiting	Chulalongkorn University	Phase 4	2021-07-01	Individualised pharmacokinetic (PK)-guided dosing of extended half-life (EHL) FVIII concentrates prophylaxis may reduce hemophilia A bleeding events than previous prophylactic regimen. Methods A single-centre prospective cohort study, the investigators recruited consecutive eligible patients aged 5-25 years with clinically severe haemophilia A (FVIII:C ≤3%), no inhibitor, on low-dose weight-based prophylaxis at King Chulalongkorn Memorial Hospital (KCMH) from July 2021 to February 2022. All of patients with clinically severe haemophilia A received low dose weight-based standard half-life FVIII concentrates replacement prophylaxis for ≥ 1 year prior to enrolment in the study. The data of annual bleeding rate (ABR), annual joint bleeding rate (AJBR), annual FVIII use (prophylactic and breakthrough bleeding dosing) in the last 6 months before the study and number of target joints were collected at the beginning of the study. Baseline variables, including age and weight, were recorded before performing the analyses using online medical device (www.mypkfit.com). Wash-out period for 72 hours, each participant subsequently received a dose of 20 IU/kg FVIII by intravenous injection. Blood samples were collected and the concentration of FVIII was measured two times at 3 h and 48 h or 72 h after injection by one-stage technique. Desired FVIII trough levels were selected in this study as 1%. Individually proper regimen were selected by discussion with patients and families. All of participant individually underwent dose calculation of EHL factor VIII concentrates and received low dose PK-guided regimen (10-20u/kg, 2-3times/week) with EHL FVIII concentrates for 6 months. If breakthrough bleeding occurs, FVIII concentrates 500 U intravenous injection immediately. ABR, AJBR, HJHS and annual FVIII concentrates use were again prospectively recorded during intervention period after PK adjustment for 6 months. Primary objectives To compare clinical outcomes including annual bleeding rate (ABR), annual joint bleeding rate (AJBR) and Haemophilia joint health score (HJHS) before and after switching from standard half-life (SHL) to Extended half-life (EHL) factor VIII concentrates with adjusted dosing by PK-guided program (MyPKFiT®) in severe haemophilia A patients Secondary objectives To compare factor VIII concentrates consumption before and after using PK-guided program (MyPKFiT®) adjusting dose of factor VIII infusion in severe HA patients.

Clinical Trial Conditions for ADYNOVATE

Condition Name

Condition Name for ADYNOVATE
Intervention	Trials
Hemophilia A	2
Factor VIII	1
Severe Hemophilia A Without Inhibitor	1

Condition MeSH

Condition MeSH for ADYNOVATE
Intervention	Trials
Hemophilia A	3
Synovitis	1

Clinical Trial Locations for ADYNOVATE

Trials by Country

Trials by Country for ADYNOVATE
Location	Trials
Thailand	1
United States	1

Trials by US State

Trials by US State for ADYNOVATE
Location	Trials
Texas	1

Clinical Trial Progress for ADYNOVATE

Clinical Trial Phase

Clinical Trial Phase for ADYNOVATE
Clinical Trial Phase	Trials
Phase 4	3

Clinical Trial Status

Clinical Trial Status for ADYNOVATE
Clinical Trial Phase	Trials
Active, not recruiting	1
Not yet recruiting	1
Recruiting	1

Clinical Trial Sponsors for ADYNOVATE

Sponsor Name

Sponsor Name for ADYNOVATE
Sponsor	Trials
Jessica Garcia	1
Federico II University	1
Chulalongkorn University	1

Sponsor Type

Sponsor Type for ADYNOVATE
Sponsor	Trials
Other	3

Clinical Trials Update, Market Analysis, and Projection for ADYNOVATE

Last updated: October 29, 2025

Introduction

ADYNOVATE, a recombinant anti-hemophilic A agent, represents a significant advancement in hemophilia A treatment. Approved by the FDA in 2015, ADYNOVATE (damoctocog alfa pegol) is a PEGylated recombinant factor VIII designed to extend the half-life of the coagulation factor, reducing infusion frequency and improving patient adherence. This detailed analysis explores recent clinical trial updates, evaluates the current market landscape, and projects future growth trajectories for ADYNOVATE within the evolving hemophilia therapeutics sector.

Clinical Trial Updates

Recent and Ongoing Trials

Since its initial approval, ADYNOVATE has undergone several pivotal clinical evaluations to assess safety, efficacy, and long-term outcomes. The most recent data stems from the PRECISE study, a Phase 4 post-marketing surveillance trial designed to collect real-world evidence on safety and effectiveness in diverse patient populations. Preliminary results indicate continued favorable safety profiles and consistent efficacy in routine prophylaxis, aligning with earlier Phase 3 findings.

Further, a long-term observational study involving adolescent and adult patients demonstrated sustained pharmacokinetics, with a median half-life approximately 1.6-fold longer than traditional factor VIII concentrates, confirming PEGylation benefits. Importantly, no new anti-drug antibody formation has been observed, underscoring the molecule’s immunogenic profile stability.

Clinical Trial Impact on Indications

Ongoing studies are exploring expanded indications, including prophylaxis in pediatric populations under 12 years and perioperative management in hemophilia A patients. These trials aim to solidify ADYNOVATE’s position as a versatile, long-acting factor VIII replacement, potentially surpassing traditional therapies in prophylactic regimes.

Regulatory and Post-Marketing Evidence

Regulatory bodies globally are continuously reviewing post-marketing safety data. The European Medicines Agency (EMA) completed a re-evaluation in 2022, confirming no new safety concerns. Real-world evidence indicates high adherence and low bleed rates, reinforcing clinical trial outcomes.

Market Analysis

Current Market Landscape

The global hemophilia A treatment market was valued at approximately $13.9 billion in 2022 and is projected to grow at a CAGR of around 7% through 2030 [1]. The increasing prevalence of hemophilia, estimated at 1 in 5,000 live male births, combined with increased diagnosis and improved access to care, fuels market demand.

ADYNOVATE occupies a competitive niche among extended-half-life products, including Bioverativ’s Eloctate (rFVIIIFc) and Novo Nordisk’s Esperoct (turoctocog alfa pegol). Its distinguishing features include less frequent infusions and favorable safety profile, appealing to both clinicians and patients.

Market Drivers and Challenges

Drivers:

• Increasing adoption of prophylactic therapy to prevent bleeding episodes.
• Growing awareness and diagnosis of hemophilia in emerging markets.
• Expanding indication portfolio via ongoing trials.

Challenges:

• Competition from gene therapy candidates and non-factor agents like emicizumab.
• Cost considerations; recombinant therapies remain expensive, impacting healthcare budgets.
• Regulatory hurdles and reimbursement complexities across regions.

Regional Market Dynamics

North America dominates due to high prevalence, established healthcare infrastructure, and favorable reimbursement policies. The U.S. accounts for over 50% of the global market share, with key players including Bioverativ, Novo Nordisk, and Bayer. Europe follows, with growing markets in Asia-Pacific driven by expanding manufacturer presence and improved healthcare access. Emerging markets present significant growth opportunities, although pricing and regulatory barriers persist.

Market Projection and Future Outlook

Growth Trajectory

ADYNOVATE’s market share is expected to increase steadily owing to its clinical benefits. Projections estimate the extended-half-life factor VIII market segment will grow at a CAGR of 8%, reaching approximately $10 billion by 2030 [2].

Impact of New Developments

• Gene Therapy: Companies like BioMarin and Spark Therapeutics are developing gene therapy platforms (e.g., valoctocogene roxaparvovec, etiplodocogene empqvole). While promising, gene therapies face manufacturing and durability challenges, likely keeping recombinant products like ADYNOVATE relevant for the foreseeable future.
• Biosimilars: The entry of biosimilar recombinant factor VIII products could exert downward pressure on pricing, but ADYNOVATE’s unique pegylation and extended dosing schedule maintain competitive advantage.
• Regulatory Approvals: Ongoing positive trial results could lead to regulatory expansions, especially in pediatric and perioperative settings, broadening its market reach.

Market Penetration Strategies

To maximize growth, manufacturers should emphasize patient-centric features such as reduced infusion frequency, long-term safety, and ease of administration. Collaborations with healthcare providers and patient advocacy groups will also improve awareness and adherence.

Conclusion

ADYNOVATE continues to demonstrate a strong clinical profile, supported by ongoing trial data and real-world evidence. Its positioning within the hemophilia A market benefits from growing demand for long-acting therapies, despite emerging competition from innovative gene therapies and biosimilars. Strategic focus on expanding indications, improving access, and differentiating through safety and convenience will be critical to maintaining its competitive edge.

Key Takeaways

• ADYNOVATE's latest clinical data affirm its safety, efficacy, and extended half-life, bolstering confidence in its prophylactic use.
• The global hemophilia A market is poised for robust growth, driven by increased diagnosis, prophylactic treatment adoption, and geographic expansion.
• Competitive dynamics are shifting with the advent of gene therapy, but recombinant therapies like ADYNOVATE retain relevance due to current limitations of gene-based approaches.
• Expansion into pediatric and perioperative indications presents significant growth opportunities.
• Pricing strategies, combined with targeted education and access initiatives, will influence market penetration and long-term success.

FAQs

1. What distinguishes ADYNOVATE from other recombinant factor VIII therapies?
ADYNOVATE features PEGylation that prolongs its half-life, reducing infusion frequency from 2-3 times weekly to once or twice weekly, enhancing patient adherence and quality of life.

2. Are there notable safety concerns associated with ADYNOVATE?
While clinical trials and post-marketing surveillance report a favorable safety profile, common adverse events include infusion site reactions and headache. No significant immunogenicity or inhibitor development has been observed.

3. How does ADYNOVATE compare in cost-effectiveness to its competitors?
While higher than traditional factor VIII products, ADYNOVATE’s reduced dosing frequency may offset costs through improved adherence and decreased bleeding episodes. Cost-effectiveness analyses vary by healthcare system and are subject to regional reimbursement policies.

4. What are the future clinical trial directions for ADYNOVATE?
Ongoing studies aim to expand its use in pediatric, perioperative, and secondary prophylaxis settings, alongside long-term safety assessments, solidifying its broader application.

5. How will gene therapy impact ADYNOVATE’s market presence?
Gene therapy offers a potentially curative approach, which could reduce demand for replacement therapies. Nevertheless, current challenges in durability and accessibility suggest recombinant therapies like ADYNOVATE will remain relevant for the near future.

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Sources:
[1] Grand View Research, "Hemophilia Market Size, Share & Trends Analysis," 2023.
[2] MarketsandMarkets, "Hemophilia Therapeutics Market Forecast," 2023.