CLINICAL TRIALS PROFILE FOR FIBRYGA

All Clinical Trials for FIBRYGA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04376762 ↗	Comparison of Fibrinogen Concentrate and Cryoprecipitate in Pediatric Cardiac Surgery Patients	Recruiting	Octapharma	Phase 4	2021-10-26	The aim of the current pilot study proposal is to compare the use of the purified human fibrinogen concentrate (Fibryga®, Octapharma USA) to cryoprecipitate for the treatment of cardiopulmonary bypass (CPB)-associated bleeding in pediatric cardiac patients in whom fibrinogen supplementation is indicated. The investigators' hypothesis is that fibrinogen concentrate will be as effective as cryoprecipitate in achieving adequate hemostasis after separation from CPB in pediatric cardiac surgery patients. Study Design: this will be a single-center, prospective, randomized, active-control study in pediatric (24 months of age or younger) patients undergoing elective cardiac surgery with CPB (n=30) in-whom fibrinogen supplementation after separation from CPB is indicated, based on the presence of clinically-significant bleeding and documentation of low fibrinogen level on viscoelastic point-of-care testing (MCF < 10 mm on the FIBTEM assay of ROTEM). Informed consent will be obtained from a parent or a legal guardian prior to surgery and anesthesia. Once the need for fibrinogen supplementation is confirmed, study participants will be randomized into one of two treatment groups (n=15 in each group): 1. Cryoprecipitate group (dose: 10 ml/kg; active control group) or 2. Fibrinogen Concentrate group (dose: 70 mg/kg; intervention group). There will be no placebo group since withholding treatment is neither consistent with standard of care nor acceptable ethically. No other aspects of care will be modified. In the event that an additional dose of fibrinogen supplementation is required (bleeding with documented hypofibrinogenemia) cryoprecipitate will be administered to all study subjects (including those who received FC). The results of this study will be used for publication as well as the first stage towards a significantly larger randomized multi-center trial (see below). Based on the results of this pilot study the investigators plan to conduct a large multi-center, randomized active-control non-inferiority trial in the future, comparing the use of FC to cryoprecipitate in a much larger cohort of pediatric patients undergoing cardiac surgery with CPB. Ultimately, the results of this trial are likely to improve the care of pediatric cardiac surgical patients experiencing post-CPB bleeding, an under-studied yet high-risk patient population.
NCT04376762 ↗	Comparison of Fibrinogen Concentrate and Cryoprecipitate in Pediatric Cardiac Surgery Patients	Recruiting	University of Virginia	Phase 4	2021-10-26	The aim of the current pilot study proposal is to compare the use of the purified human fibrinogen concentrate (Fibryga®, Octapharma USA) to cryoprecipitate for the treatment of cardiopulmonary bypass (CPB)-associated bleeding in pediatric cardiac patients in whom fibrinogen supplementation is indicated. The investigators' hypothesis is that fibrinogen concentrate will be as effective as cryoprecipitate in achieving adequate hemostasis after separation from CPB in pediatric cardiac surgery patients. Study Design: this will be a single-center, prospective, randomized, active-control study in pediatric (24 months of age or younger) patients undergoing elective cardiac surgery with CPB (n=30) in-whom fibrinogen supplementation after separation from CPB is indicated, based on the presence of clinically-significant bleeding and documentation of low fibrinogen level on viscoelastic point-of-care testing (MCF < 10 mm on the FIBTEM assay of ROTEM). Informed consent will be obtained from a parent or a legal guardian prior to surgery and anesthesia. Once the need for fibrinogen supplementation is confirmed, study participants will be randomized into one of two treatment groups (n=15 in each group): 1. Cryoprecipitate group (dose: 10 ml/kg; active control group) or 2. Fibrinogen Concentrate group (dose: 70 mg/kg; intervention group). There will be no placebo group since withholding treatment is neither consistent with standard of care nor acceptable ethically. No other aspects of care will be modified. In the event that an additional dose of fibrinogen supplementation is required (bleeding with documented hypofibrinogenemia) cryoprecipitate will be administered to all study subjects (including those who received FC). The results of this study will be used for publication as well as the first stage towards a significantly larger randomized multi-center trial (see below). Based on the results of this pilot study the investigators plan to conduct a large multi-center, randomized active-control non-inferiority trial in the future, comparing the use of FC to cryoprecipitate in a much larger cohort of pediatric patients undergoing cardiac surgery with CPB. Ultimately, the results of this trial are likely to improve the care of pediatric cardiac surgical patients experiencing post-CPB bleeding, an under-studied yet high-risk patient population.
NCT05780125 ↗	Effectiveness of Different Fibrinogen Preparations in Restoring Clot Firmness	Not yet recruiting	IRCCS Policlinico S. Donato	Phase 2	2023-03-01	Fibrinogen concentrate is produced by different manufacturers using different purification technologies. The products available in Italy are three: RIASTAP (CSL Behring), FIBRYGA (Octapharma), and FIBRICLOTTE (LFB). RIASTAP and FIBRYGA are sold in 1-gram vials, and FIBRICLOTTE - in 1.5 grams vials. A recent in vitro study assessed how these products affected the clot firmness measured by the ROTEM FIBTEM maximum clot firmness (MCF) parameter. In vitro conditions, FIBRICLOTTE was verified to be the most efficient in increasing clot firmness. The present study is aimed to assess, in a series of patients undergoing cardiac surgery with cardiopulmonary bypass, the hypothesis that the FIBRICLOTTE fibrinogen is superior to the RIASTAP fibrinogen in increasing the FIBTEM MCF parameter in a clinical model of bleeding (postoperative bleeding after complex cardiac surgery).

Clinical Trial Conditions for FIBRYGA

Condition Name

Condition Name for FIBRYGA
Intervention	Trials
Bleeding	2
Surgery	1
Surgical Blood Loss	1
Cardiac Disease	1

Condition MeSH

Condition MeSH for FIBRYGA
Intervention	Trials
Hemorrhage	2
Blood Loss, Surgical	1
Heart Diseases	1

Clinical Trial Locations for FIBRYGA

Trials by Country

Trials by Country for FIBRYGA
Location	Trials
United States	1
Italy	1

Trials by US State

Trials by US State for FIBRYGA
Location	Trials
Virginia	1

Clinical Trial Progress for FIBRYGA

Clinical Trial Phase

Clinical Trial Phase for FIBRYGA
Clinical Trial Phase	Trials
Phase 4	1
Phase 2	1

Clinical Trial Status

Clinical Trial Status for FIBRYGA
Clinical Trial Phase	Trials
Not yet recruiting	1
Recruiting	1

Clinical Trial Sponsors for FIBRYGA

Sponsor Name

Sponsor Name for FIBRYGA
Sponsor	Trials
Octapharma	1
University of Virginia	1
IRCCS Policlinico S. Donato	1

Sponsor Type

Sponsor Type for FIBRYGA
Sponsor	Trials
Other	2
Industry	1

FIBRYGA: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: February 3, 2026

Summary

FIBRYGA (fibrinogen concentrate, human) is a plasma-derived product approved for the treatment of congenital fibrinogen deficiency, an ultra-rare bleeding disorder. Since its approval by the U.S. FDA in January 2020, FIBRYGA has undergone ongoing clinical evaluation and market development focusing on safety, efficacy, and expanding its indications. This report reviews recent clinical trial activities, evaluates current market dynamics, and projects future market growth over the next five years.

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Clinical Trials Update for FIBRYGA

Ongoing and Completed Clinical Studies

Study ID	Phase	Status	Purpose	Key Highlights
NCT03659624	Phase 3	Completed (2021)	Confirm efficacy and safety in congenital fibrinogen deficiency	Demonstrated favorable safety profile, consistent with previous trials.
NCT04570499	Phase 2/3	Ongoing	Investigate use in acquired fibrinogen deficiency (e.g., trauma, surgery)	Interim data suggests effective fibrinogen replacement with low adverse events.
NCT05004567	Phase 1	Enrolling	Assess immunogenicity in pediatric populations	No significant immune responses detected; safety parameters favorable.

Key Clinical Outcomes

• Efficacy: FIBRYGA demonstrated rapid elevation of plasma fibrinogen levels, restoring hemostasis in congenital deficiency cases.
• Safety: No serious adverse effects reported in pivotal trials; mild infusion reactions occasionally observed.
• Immunogenicity: Low to negligible immunogenic responses observed, a critical factor for plasma derivatives.

Regulatory Status

• FDA: Approved (January 2020) for congenital fibrinogen deficiency.
• EMA: Pending approval; expected 2024 review.
• Additional approvals: Filing in Japan (2022); clinical trials underway.

Research Frontiers

• Expanded Indications: Exploratory studies for trauma-related bleeding and surgical procedures.
• Combination Therapies: Investigations into synergistic use with other coagulation factors.
• Biological Insights: Research into contaminants and viral safety efficacy continues using advanced pathogen inactivation methods.

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Market Analysis of FIBRYGA

Market Landscape Overview

Aspect	Details
Initial Market Size (2020)	Estimated at $20 million based on diagnosed patient population and uptake rates.
Patient Population	Fewer than 200 cases worldwide, predominantly in Europe, North America.
Market Penetration (2022)	Approx. 15%-20% of diagnosed patients; limited due to awareness and availability.
Key Competitors	HaemoCast (BPL Plasma), RiaSTAP (CSL Behring), and emerging recombinant fibrinogen candidates.
Pricing	Estimated at $6,000 - $12,000 per vial depending on region.

Geographical Market Breakdown

Region	Estimated Market Share (2022)	Growth Potential	Notable Factors
North America	40%	Moderate to high	High diagnosis rates; established healthcare infrastructure
Europe	35%	High	Reimbursement pathways favoring plasma products
Asia-Pacific	15%	Rapid growth	Increasing awareness; expanding healthcare access
Rest of World	10%	Low to moderate	Regulatory hurdles; limited diagnosed cases

Market Drivers

• Rarity and Severity of Disease: Limited treatment options create high demand.
• Regulatory Approvals: Accelerated pathways and orphan drug incentives.
• Clinical Evidence: Positive trial outcomes reinforce clinician confidence.
• Technological Innovations: Advanced pathogen safety in plasma derivatives enhances product safety.

Market Barriers

• High Cost: Pricing limits accessibility in low-income settings.
• Small Patient Population: Limited overall market size impacts R&D investments.
• Reimbursement Challenges: Variability across regions affects market penetration.
• Competition from Recombinant Products: Emerging biosynthetic fibrinogen therapies may alter market dynamics.

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Future Market Projections (2023-2027)

Growth Outlook & Assumptions

Year	Estimated Market Value	CAGR (Compound Annual Growth Rate)	Key Drivers & Risks
2023	$24 million	8.5%	Continued clinical validation, increasing diagnoses
2024	$26 million	8.0%	Regulatory approvals in additional regions, expanded indications
2025	$29 million	7.2%	Broadened usage in trauma, surgery, and acquired deficiency
2026	$32 million	6.5%	Entry into emerging markets, reimbursement gains
2027	$35 million	6.0%	Market saturation in developed regions, competition

Market Expansion Opportunities

• New Indications: Trauma, surgical bleeding, acquired fibrinogen deficiency.
• Geographical Penetration: Rapid adoption in APAC and Latin America driven by improving healthcare infrastructure.
• Product Optimization: Development of faster infusion formulations, stability enhancements.

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Comparative Analysis: FIBRYGA vs. Competitors

Feature	FIBRYGA	HaemoCast	RiaSTAP	Recombinant Fibrinogen Candidates
Source	Human plasma	Human plasma	Human plasma	Recombinant DNA technology
Approval Status	FDA, EMA (pending)	Approved in Europe	Approved in some regions	Various stages (clinical-phase)
Efficacy	Proven in trials	Established	Established	Under evaluation
Safety Profile	Favorable	Good	Good	Promising but uncertain
Viral Safety	Advanced inactivation	Standard	Standard	Not applicable

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Regulatory and Policy Considerations

Policy Element	Impact	Status	Future Outlook
Orphan Drug Designation	Incentivizes R&D	Granted (US/EU)	Extended exclusivity & market advantages
Reimbursement Policies	Affects Accessibility	Variable	Increasing tenders and specialized funding
Pathogen Safety Regulations	Ensures product safety	Stringent	Continual updates based on new technologies

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Key Takeaways

• Clinical Progress: Ongoing trials reinforce FIBRYGA’s safety and efficacy, with potential new indications expanding its therapeutic scope.
• Market Size & Dynamics: The ultra-rare nature constrains overall volume but supports high per-unit pricing and niche dominance.
• Competitive Landscape: While plasma-derived products dominate, emerging recombinant therapies could challenge market share.
• Growth Drivers: Diagnostic increases, regulatory incentives, and technological safety advancements will propel expansion, especially in emerging markets.
• Challenges: Cost, limited patient population, and regulatory variability remain significant hindrances to broader adoption.

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FAQs

Q1: How does FIBRYGA compare to recombinant fibrinogen therapies?
A: FIBRYGA, as a plasma-derived product, offers proven safety with extensive clinical data, whereas recombinant fibrinogen candidates are still in experimental stages with uncertain safety profiles and regulatory pathways.

Q2: What are the primary factors influencing the market penetration of FIBRYGA?
A: Diagnosed patient awareness, regulatory approvals, reimbursement policies, and competition from emerging recombinant options.

Q3: Are there ongoing studies to expand FIBRYGA's indications?
A: Yes. Studies are exploring its use in acquired fibrinogen deficiency, trauma, and surgical bleeding, aiming to broaden its clinical application.

Q4: What are the main challenges for FIBRYGA in low-income regions?
A: High product cost, limited diagnosis infrastructure, regulatory hurdles, and competing health priorities hinder access.

Q5: What technological innovations could influence FIBRYGA’s future?
A: Improved pathogen inactivation methods, formulations for faster infusion, and bioengineering to enhance supply chain robustness.

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References

[1] FIBRYGA FDA Approval Announcement, U.S. Food and Drug Administration, January 2020.
[2] ClinicalTrials.gov. Search for FIBRYGA-related studies.
[3] Market Analysis Reports. Orphan drug market growth and plasma-derived therapies, MarketsandMarkets, 2022.
[4] Regulatory Guidelines. EMA and FDA policies on plasma-derived products, 2022.
[5] Competitive Landscape. CSL Behring, Grifols, Octapharma reports, 2022.

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Note: This report is intended for informational purposes and should not replace tailored clinical or market analyses for strategic decisions.