CLINICAL TRIALS PROFILE FOR BENEFIX

All Clinical Trials for BENEFIX

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00037557 ↗	Study Evaluating rFIX; BeneFIX in Severe Hemophilia B	Completed	Wyeth is now a wholly owned subsidiary of Pfizer	Phase 3	2002-09-01	To characterize the safety and efficacy of rFIX in children less than 6 years of age with severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery. This study will provide an opportunity for systematic observation of treatment with rFIX in children less than 6 years of age regardless of prior FIX treatment. Younger patients exhibit a different pharmacokinetic profile and therefore may respond differently to rFIX infusions when compared with older children and adults. This evaluation will provide data from which recommendations can be made regarding rFIX dosing and treatment of these patients. Surveillance for certain observations that have been made in patients treated with rFIX in the clinical and postmarketing setting will be performed, including inhibitor development, thrombogenicity, FIX recovery/lack of effect, allergic-type manifestations, and RBC agglutination. Comparisons will be derived from published reports and communications describing experience with other FIX products and protein therapeutics in general.
NCT00093171 ↗	Study Evaluating rFIX; BeneFIX® in Hemophilia B	Completed	Wyeth is now a wholly owned subsidiary of Pfizer	Phase 3	1969-12-31	The primary objective of this clinical research study is to assess the safety and efficacy of rFIX for a minimum of 6 months in previously treated patients (PTPs) with hemophilia B (FIX:C ≤2%) during standard-of-care treatment (on-demand, prophylaxis, and through major and minor surgical procedures).
NCT00093210 ↗	Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B	Completed	Wyeth is now a wholly owned subsidiary of Pfizer	Phase 3	1969-12-31	The primary objective of this clinical research study is to establish the bioequivalence of 2 treatments, rFIX and rFIX-R, when given as a 10-minute intravenous bolus infusion.
NCT00244114 ↗	Study Evaluating Allergic Reactions To Benefix In Hemophilia B Patients	Completed	Wyeth is now a wholly owned subsidiary of Pfizer		2006-02-01	Based on the finding that anaphylactic reactions to Benefix ("FIX") are associated with a specific IgE, a Basophil histamine release assay was selected to evaluate and demonstrate subject sensitization to FIX.
NCT00364182 ↗	Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B	Completed	Pfizer	Phase 3	2007-05-01	This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate in 4 study periods. The first period is a 16-week on-demand treatment, during which subjects will utilize BeneFIX to treat bleeding events episodically only as they occur (i.e., on-demand treatment). At the end of this period, subjects will be randomly assigned to 1 of 2 BeneFIX prophylaxis regimens: either 100 IU/kg once weekly or 50 IU/kg twice weekly, and followed for 16 weeks. At the end of this period, subjects will use on-demand treatment for 8 weeks. The purpose of this 8-week period is to mitigate the potential carry-over effect of the prior prophylaxis regimen on bleeding frequency. Following this 8-week on-demand treatment, subjects will cross-over and receive the alternate study prophylactic regimen for 16 weeks. The primary endpoint is annualized number of bleeding episodes, compared between the first on-demand period and each of the prophylaxis regimens. A comparison of annualized bleeding episodes between the two prophylaxis regimens will also be performed. Subject-reported outcomes will also be collected using a patient diary. At 24 and 48 hours following the onset of each joint bleeding episode, information on pain, sleep, and work will be collected. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period. Information on safety will also be collected. Each subject will participate in this study for approximately 59 weeks (15 months), including a screening period of up to 3 weeks, an initial 16-week on-demand period, two prophylaxis treatment periods of 16 weeks each, separated by an 8-week on-demand treatment period. A modified FIX recovery study will be performed once during each prophylaxis period. The study diary will also be used by subjects to collect secondary endpoints. These endpoints, which are subject-reported outcomes, will be recorded in the diary at 24 and 48 hours following the onset of each joint bleeding episode. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period. Patients will be recruited in the United States, Canada, Europe and Russia.
NCT00484185 ↗	Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B	Completed	Pfizer		2007-08-01	To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including: 1) Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses. 3) Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug

Clinical Trial Conditions for BENEFIX

Condition Name

Condition Name for BENEFIX
Intervention	Trials
Hemophilia B	11
Severe Hemophilia B	1

Condition MeSH

Condition MeSH for BENEFIX
Intervention	Trials
Hemophilia B	12
Hemophilia A	10
Hypersensitivity	1

Clinical Trial Locations for BENEFIX

Trials by Country

Trials by Country for BENEFIX
Location	Trials
United States	25
China	16
Canada	6
Germany	5
India	3

Trials by US State

Trials by US State for BENEFIX
Location	Trials
Texas	3
New Jersey	3
Michigan	3
Colorado	3
Illinois	2

Clinical Trial Progress for BENEFIX

Clinical Trial Phase

Clinical Trial Phase for BENEFIX
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	5
Phase 1	1

Clinical Trial Status

Clinical Trial Status for BENEFIX
Clinical Trial Phase	Trials
Completed	12

Clinical Trial Sponsors for BENEFIX

Sponsor Name

Sponsor Name for BENEFIX
Sponsor	Trials
Pfizer	6
Wyeth is now a wholly owned subsidiary of Pfizer	5
Swedish Orphan Biovitrum	1

Sponsor Type

Sponsor Type for BENEFIX
Sponsor	Trials
Industry	14

BENEFIX (Coagulation Factor IX, Recombinant) Clinical Trials Update, Market Analysis, and Projections

Last updated: May 4, 2026

What is BENEFIX and what product line does it represent?

BENEFIX (nonacog alfa, recombinant coagulation factor IX) is used for hemophilia B (congenital factor IX deficiency) for:

• On-demand treatment of bleeding episodes
• Perioperative management of bleeding
• Routine prophylaxis to reduce bleeding frequency in eligible patients

Regulatory positioning (core label): Factor IX replacement for hemophilia B across dosing strategies (on-demand and prophylaxis). BENEFIX is a marketed recombinant factor IX product, distinct from FIX concentrates derived from plasma.

What is the clinical trials update for BENEFIX?

No current, discrete “BENEFIX-only” late-stage development program (Phase 3 or registrational expansion) is established in the publicly indexed literature within the provided knowledge window. Most contemporary “clinical update” activity in hemophilia B has shifted to:

• Extended half-life factor IX molecules and
• Gene therapy programs for hemophilia B

Given the absence of a confirmed active registrational trial set for BENEFIX in the available record set, the practical clinical “update” for BENEFIX is best treated as post-approval use and regimen optimization, with market behavior shaped by competition from newer FIX assets and gene therapy pipelines rather than new BENEFIX pivotal studies.

What is the competitive landscape affecting BENEFIX?

The hemophilia B market has concentrated around two substitution vectors:

1. Next-generation FIX products (extended half-life)

• Extended interval dosing reduces infusion frequency and can drive payer preference.
• Competitive products can change switching behavior even when clinical outcomes are similar.

2. Hemophilia B gene therapy

• One-time administration models can displace long-term FIX utilization for some patients.
• Commercial impact is strongest where gene therapy uptake is supported by eligibility, payer coverage, and center adoption patterns.

This competitive structure is the main determinant of BENEFIX net sales trajectory after the initial product lifecycle maturity.

How big is the FIX replacement market and where does BENEFIX sit?

The hemophilia B treatment market is a component of the larger hemophilia therapeutics market (hemophilia A and B). BENEFIX participates in a niche defined by:

• US/EU hemophilia B patient population
• Dosing intensity (prophylaxis vs on-demand)
• Per-patient annual factor consumption
• Switch rates to extended half-life products
• Gene therapy uptake among eligible severe patients

Because BENEFIX is not the newest extended half-life asset, it faces pricing pressure and volume erosion risk versus current “preferred” FIX concentrates in many formularies.

What is the pricing and contracting reality for FIX concentrates?

Within hemophilia markets, pricing is shaped by:

• Brand-to-brand payer contracting
• Step therapy and formulary tiering
• Patient-level preference based on dosing interval
• Provider inventory and infusion center protocols

For BENEFIX, the key market constraint is that extended half-life competitors typically offer a lower infusion frequency value proposition, which can translate into payer willingness to favor them even when clinical efficacy is adequate with conventional half-life dosing.

Market analysis: drivers and headwinds for BENEFIX

Demand drivers

• Ongoing need for lifelong replacement in hemophilia B where prophylaxis is required
• Continued use for patients who:
  • Do not qualify for gene therapy
  • Prefer existing factor plans
  • Are stable on current dosing schedules
• Availability and established clinical experience for physicians and treatment centers

Demand headwinds

• Switching pressure toward extended half-life factor IX products
• Gene therapy displacement in selected markets and treatment centers
• Margin compression from price competition and payer renegotiations
• Patient share loss where dosing interval is used as an economic decision lever

Sales and market share outlook: what is the likely trajectory?

On balance, BENEFIX is expected to follow a late-lifecycle profile typical of mature FIX concentrates:

• Stable-to-declining unit demand driven by switching and gene therapy
• Net sales resilience only where:
  • Payer formularies keep coverage
  • Patients remain on established prophylaxis
  • Competitive substitution is limited by contracts

Projections: BASE/UPSIDE/DOWNSIDE framework

The projections below are structured for business planning rather than pinpoint forecasting. They reflect known market mechanics in hemophilia: substitution by extended half-life FIX and gene therapy.

Scenario	Primary assumption for BENEFIX	Expected volume trend	Expected revenue trend
Downside	Higher-than-market switch to extended half-life; faster gene therapy uptake in major centers	Declines faster than hemophilia B cohort growth	Declines faster than volume due to price pressure
Base	Continued gradual switch; gene therapy uptake offsets but does not dominate	Moderate decline with cohort stability	Mild-to-moderate decline, depends on contracting
Upside	Favorable contracts; lower switching; limited uptake in some geographies	Stable or slow decline	Flatter revenue profile despite unit softness

What endpoints matter for payer and provider decisions?

For FIX concentrates, purchasing decisions typically weight:

• Bleeding control (annualized bleeding rate outcomes)
• Dosing interval practicality (prophylaxis convenience)
• Safety profile and immunogenicity considerations
• Overall patient adherence and infusion center workflow fit
• Net cost per treated patient episode under payer contracts

BENEFIX remains clinically effective within its label domain, but the market tends to bias toward therapies that reduce infusion burden unless contracts preserve share.

Clinical trial strategy implications for investors and sponsors

No active registrational BENEFIX program is identified in the provided record set. For stakeholders, the R&D implication is direct: the growth engine is not BENEFIX incremental trials, it is:

• Competing FIX lifecycle products (especially extended half-life)
• Hemophilia B gene therapy development and commercialization
• Any new generation of FIX molecules that target durability, dosing interval, and reduced treatment burden

Key regulatory and manufacturing context that influences commercial resilience

For mature biologics, commercial resilience depends on:

• Supply continuity
• Manufacturing scale consistency
• Pharmacovigilance record
• Contracting and hospital formularies
• Label stability (no major post-approval retrenchment)

BENEFIX’s long market presence supports a stable clinical use base, but substitution dynamics still dominate volume.

Key Takeaways

• BENEFIX is a recombinant factor IX product for hemophilia B with established on-demand and prophylaxis use.
• The clinical “update” for BENEFIX is not driven by newly identified registrational trials in the available record set; hemophilia B development momentum has shifted to extended half-life FIX and gene therapy.
• Market outlook is shaped by switching pressure and gene therapy displacement, creating a late-lifecycle pattern of stable-to-declining volume with price/contract-driven revenue variability.
• Business planning should use scenario-based forecasting tied to formularies, switching rates, and gene therapy uptake in major treatment centers.

FAQs

1. Is BENEFIX still used for prophylaxis in hemophilia B?
   Yes. BENEFIX is indicated for routine prophylaxis in eligible hemophilia B patients to reduce bleeding frequency.

2. What is the biggest competitive threat to BENEFIX?
   Extended half-life FIX products and hemophilia B gene therapy, which can reduce infusion frequency and/or eliminate long-term factor use for some patients.

3. Are there major new Phase 3 registrational trials for BENEFIX currently driving growth?
   No discrete current registrational expansion program for BENEFIX is supported by the provided record set within the available window.

4. How do payers choose between conventional FIX and extended half-life products?
   Typically through contract terms and value assessments that include dosing interval convenience and net cost per treated patient.

5. What determines BENEFIX revenue in the near-to-medium term?
   Patient share after switching, pricing/contracting outcomes, and the rate of gene therapy uptake by severe hemophilia B patients.

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References (APA)

[1] FDA. (2019). BENEFIX (nonacog alfa) prescribing information. U.S. Food and Drug Administration.
[2] EMA. (n.d.). BENEFIX summary of product characteristics (SmPC). European Medicines Agency.