HEMGENIX Market Summary

Key Factors Driving HEMGENIX Growth

1. Market Share Gains and New Patient Starts

• HEMGENIX continues to advance adoption as the first and only FDA- and EMA-authorized gene therapy for adults with severe and moderately severe hemophilia B, offering a potential one-time durable treatment compared with chronic factor replacement or prophylactic therapies.
• New patient starts are increasing as treatment centers in Europe and North America begin administering HEMGENIX in real-world settings following regulatory approvals and reimbursement negotiations.
• Early commercial use in markets like Germany and France shows growing clinician confidence and patient interest, especially for eligible patients seeking reduced treatment burden.

 

2. Expansion Across Key Indications

• Severe and Moderately Severe Hemophilia B: HEMGENIX is indicated for adults with congenital Factor IX deficiency who currently use Factor IX prophylaxis or have experienced repeated serious bleeding episodes.
• The therapy delivers a functional gene enabling patients’ liver cells to produce Factor IX, reducing the risk of bleeding and potentially eliminating the need for routine infusions.
• Unlike traditional factor replacement therapies administered regularly, HEMGENIX’s one-time infusion creates a durable treatment effect, which is particularly compelling for patients aiming to minimize treatment burden.
• No additional indications beyond hemophilia B have been approved yet, though long-term gene therapy research and real-world evidence could support broader applications within rare bleeding disorders.

 

3. Geographic Expansion

• United States: FDA approval in 2022 enabled early commercial use, and the first US patient has been treated with HEMGENIX in clinical practice.
• European Union: The European Commission granted conditional marketing authorization (CMA) in 2023, and multiple EU countries such as France, Germany, Denmark, Spain, and the UK have seen patients treated with HEMGENIX.
• Switzerland and others: Swissmedic authorization followed in January 2024, expanding available markets further.
• Continued expansion into additional reimbursement markets is progressing as local healthcare systems establish outcome-based models to facilitate access.

 

4. New Indication Approvals

• Regulatory agencies including the FDA (US), European Commission (EU/EEA), Swissmedic (Switzerland), Health Canada and the UK MHRA have authorized HEMGENIX for adults with severe or moderately severe hemophilia B without a history of Factor IX inhibitors.
• These approvals significantly expand the treatment landscape by enabling real-world use beyond clinical trials; there are no additional labeled indications yet, but interest in pediatric or broader gene therapy applications remains under evaluation by researchers.

 

5. Strong Volume Momentum

• Volume growth is currently driven by expanding treatment center capacity, rising clinician familiarity, and increasing patient eligibility identification rather than traditional prescription volume.
• Real-world data through four years post-infusion shows sustained Factor IX activity, safety, and reductions in bleeding rates, supporting continued adoption and clinical confidence.
• Outcome-based reimbursement models and expanded access agreements in markets like Germany and Denmark are helping accelerate patient starts.

 

6. Competitive Differentiation and Market Trends

• One-time gene therapy advantage: HEMGENIX’s single intravenous infusion distinguishes it from ongoing prophylactic infusions or newer non-factor agents by offering durable endogenous Factor IX production.
• HEMGENIX leverages the AAV5 vector with the high-activity Factor IX Padua variant, which may support higher and more sustained Factor IX expression compared with older strategies.
• Broader trends in personalized medicine and gene therapy adoption coupled with payer interest in long-term cost offsets — are reinforcing HEMGENIX’s value proposition relative to chronic care models.
• Outcome-based agreements and innovative reimbursement approaches are emerging to support access for high-cost gene therapies.

 

HEMGENIX Recent Developments

• First Real-World Treatments in Europe (2024–2025): CSL Behring announced that patients in France, Denmark and Germany were treated with HEMGENIX in real-world clinical settings following European Commission approval, marking key milestones in commercial adoption.
• Swissmedic Authorization (Jan 2024): Swissmedic authorized HEMGENIX as the first gene therapy for adults with severe/moderately severe hemophilia B in Switzerland, expanding approved geographic reach.
• Long-Term Efficacy Data (EAHAD 2025): Four-year post-infusion data presented at the European Association for Haemophilia and Allied Disorders congress demonstrated sustained Factor IX activity, durable bleed protection, and a favorable safety profile in treated adults.

 

“HEMGENIX Sales Forecast, and Market Size Analysis – 2034” report provides comprehensive insights of HEMGENIX for approved indication like Haemophilia B in the 7MM. A detailed picture of HEMGENIX’s existing usage in anticipated entry and performance in approved indications in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2020 –2034 is provided in this report along with a detailed description of the HEMGENIX for approved indications. The HEMGENIX market report provides insights about HEMGENIX’s sales forecast, mechanism of action (MoA), dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of historical and current HEMGENIX performance, future market assessments inclusive of the HEMGENIX market forecast analysis for approved indications in the 7MM, SWOT, analysts’ views, comprehensive overview of market competitors, and brief about other emerging therapies in respective indications. It also provides analysis of HEMGENIX sales forecasts, along with factors driving its market.

 

HEMGENIX Drug Summary

HEMGENIX (Etranacogene dezaparvovec) is an adeno-associated virus serotype 5 (AAV5) vector-based gene therapy approved for the treatment of adults with hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or repeated serious spontaneous bleeding episodes, and who have preexisting neutralizing anti-AAV5 antibody titers below specified limits (e.g., <1:700). It delivers a modified, highly active factor IX-Padua (FIX-Padua) transgene variant under a liver-specific promoter via a one-time intravenous infusion, enabling hepatocytes to produce and secrete functional factor IX into the bloodstream, thereby sustaining elevated FIX activity levels (typically 25-40% of normal over years) to reduce bleeding rates by ~90%, eliminate the need for routine FIX prophylaxis in most patients, and provide long-term bleed protection as evidenced by four-year data showing stable mean FIX levels around 37%. The report provides HEMGENIX’s sales, growth barriers and drivers, post usage and approvals in multiple indications.

 

Scope of the HEMGENIX Market Report

The report provides insights into:

• A comprehensive product overview including the HEMGENIX MoA, description, dosage and administration, research and development activities in approved indication like Haemophilia B.
• Elaborated details on HEMGENIX regulatory milestones and other development activities have been provided in HEMGENIX market report.
• The report also highlights HEMGENIX‘s cost estimates and regional variations, reported and estimated sales performance, research and development activities in approved indications across the United States, Europe, and Japan.
• The HEMGENIX market report also covers the patents information, generic entry and impact on cost cut.
• The HEMGENIX market report contains current and forecasted HEMGENIX sales for approved indications till 2034.
• Comprehensive coverage of the late-stage emerging therapies for respective indications.
• The HEMGENIX market report also features the SWOT analysis with analyst views for HEMGENIX in approved indications.

Methodology

The HEMGENIX market report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight’s team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.

 

HEMGENIX Analytical Perspective by DelveInsight

In-depth HEMGENIX Market Assessment

This HEMGENIX sales market forecast report provides a detailed market assessment of HEMGENIX for approved indication like Haemophilia B in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides current and forecasted HEMGENIX sales data uptil 2034.

 

HEMGENIX Clinical Assessment

The HEMGENIX market report provides the clinical trials information of HEMGENIX for approved indications covering trial interventions, trial conditions, trial status, start and completion dates.

HEMGENIX Competitive Landscape

The report provides Insights on competitors and marketed products within the domain, along with a summary of emerging products and their respective launch dates, posing significant competition in the market.

 

HEMGENIX Market Potential & Revenue Forecast

• Projected market size for the HEMGENIX and its key indications
• Estimated HEMGENIX sales potential (HEMGENIX peak sales forecasts)
• HEMGENIX Pricing strategies and reimbursement landscape

 

HEMGENIX Competitive Intelligence

• Number of competing drugs in development (pipeline analysis)
• HEMGENIX Market positioning compared to existing treatments
• HEMGENIX Strengths & weaknesses relative to competitors

 

HEMGENIX Regulatory & Commercial Milestones

• HEMGENIX Key regulatory approvals & expected launch timelines
• Commercial partnerships, licensing deals, and M&A activity

 

HEMGENIX Clinical Differentiation

• HEMGENIX Efficacy & safety advantages over existing drugs
• HEMGENIX Unique selling points

HEMGENIX Market Report Highlights

• In the coming years, the HEMGENIX market scenario is set to change due to strong adoption, increased prescriptions and broader uptake in multiple immunological indications; which would expand the size of the market.
• The HEMGENIX companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence HEMGENIX’s dominance.
• Other emerging products for Haemophilia B are expected to give tough market competition to HEMGENIX and launch of late-stage emerging therapies in the near future will significantly impact the market.
• A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of HEMGENIX in approved indications.
• Analyse HEMGENIX cost, pricing trends and market positioning to support strategic decision-making in the immunology landscape.
• Our in-depth analysis of the forecasted HEMGENIX sales data uptil 2034 will support the clients in decision-making process regarding their therapeutic portfolio by identifying the overall scenario of HEMGENIX in approved indications.

 

Key Questions Answered In The HEMGENIX Market Report:

• What is the class of therapy, route of administration and mechanism of action of HEMGENIX? How strong is HEMGENIX’s clinical and commercial performance?
• What is HEMGENIX’s clinical trial status in each individual indications such as Haemophilia B and study completion date?
• What are the key collaborations, mergers and acquisitions, licensing and other activities related to the HEMGENIX Manufacturers?
• What are the key designations that have been granted to HEMGENIX for approved indications? How are they going to impact HEMGENIX’s penetration in various geographies?
• What is the current and forecasted HEMGENIX market scenario for approved indications? What are the key assumptions behind the forecast?
• What are the current and forecasted sales of HEMGENIX in the seven major countries, including the United States, Europe (Germany, France, Italy, Spain) and the United Kingdom, and Japan? 
• What are the other emerging products available and how are these giving competition to HEMGENIX for approved indications?
• Which are the late-stage emerging therapies under development for the treatment of approved indications?
• How cost-effective is HEMGENIX? What is the duration of therapy and what are the geographical variations in cost per patient?