17:31 ET Hemophilia A Market to Exhibit Growth at a CAGR of 2.9% During the Forecast Period (2025-2034) Owing to the Rising Hemophilia Prevalence, Strong Uptake of New Drugs, and Emergence of High-cost Gene Therapies | DelveInsight

The dynamics of the hemophilia A market are expected to change mainly due to the increase in prevalence and the launch of upcoming therapies such as Mim8 (Novo Nordisk), DTX201 (Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]), giroctocogene fitelparvovec (SB-525 [Sangamo Therapeutics]), and others during the forecast period. , /PRNewswire/ -- DelveInsight's Hemophilia A Market Insights report includes a comprehensive understanding of current treatment practices, hemophilia A emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Hemophilia A Market Summary The market size for hemophilia A was found to be USD 12.2 billion in the leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] in 2024. The United States accounted for the largest hemophilia A treatment market size, approximately 48% of the total market size in the 7MM in 2024, compared to other major markets, including the EU4 countries, the United Kingdom, and Japan. HEMLIBRA's growth curve (US Q1 2025 year-over-year sales) in the United States was flat, indicating increased competition from newer therapies. The total number of diagnosed prevalent cases of Hemophilia A in the 7MM was around 49,500 in 2024. Leading hemophilia A companies, such as Novo Nordisk, Ultragenyx Pharmaceutical, Sangamo Therapeutics, ASC Therapeutics, Roche, Chugai, and others, are developing new hemophilia A treatment drugs that can be available in the hemophilia A market in the coming years. The promising hemophilia A therapies in clinical trials include Mim8, DTX201 (Peboctocogene camaparvovec, BAY2599023), Giroctocogene fitelparvovec (SB-525), ASC618, NXT007/RG6512, and others. Discover the new hemophilia A treatment @ Hemophilia A Treatment Market Key Factors Driving the Growth of the Hemophilia A Market Rising Hemophilia A Target Patient Pool The diagnosed prevalence of Hemophilia A is expected to rise from 49K in 2024 to 50K by 2034. Improved diagnostic capabilities and increased disease awareness mainly drive this increase in cases. Advancements in Gene Therapy Gene therapy has emerged as a transformative approach in hemophilia A treatment. Notably, BioMarin's ROCTAVIAN has been approved in the U.S. and EU, offering a one-time infusion that provides long-term correction of the factor VIII deficiency. Pfizer's gene therapy giroctocogene fitelparvovec for hemophilia A has also demonstrated promising results in late-stage trials, significantly reducing bleeding episodes and outperforming traditional treatments. Regulatory Approvals and Hemophilia A Market Expansion Recent regulatory approvals have bolstered the availability of hemophilia A treatments. For instance, the U.S. FDA approved Pfizer's once-weekly injection, HYMPAVZI (in October 2024), for hemophilia A patients aged 12 and above, aiming to prevent or reduce bleeding episodes. Similarly, Sanofi's QFITLIA (in March 2025), a subcutaneous treatment administered every two months, was approved for patients with Hemophilia A or B aged 12 and older. Emergence of Non-Factor Hemophilia A Therapies Expanding beyond traditional therapies, hemophilia A care now includes two cutting-edge non-factor approaches: anti-TFPI therapies (Pfizer's HYMPAVZI and Novo Nordisk's ALHEMO) and siRNA therapies (Sanofi's QFITLIA). Robust Hemophilia A Clinical Trial Activity Several hemophilia A companies are actively developing emerging therapies, including Novo Nordisk (Mim8, ALHEMO), Sangamo Therapeutics (Giroctocogene fitelparvovec), Roche/Chugai (NXT007), ASC Therapeutics (ASC-618), Ultragenyx (DTX201), and others. Hemophilia A Market Analysis The primary goal of current treatment strategies for hemophilia A is to minimize complications caused by bleeding into joints, tissues, or organs. With ongoing technological advancements and improved medical understanding, patients now have access to a range of therapeutic options. Treatment is typically administered either "on-demand" or as "prophylaxis", with prophylactic therapy increasingly becoming the preferred approach. The U.S. FDA has approved several recombinant Factor VIII products for hemophilia A management, including HELIXATE FS, RECOMBINATE, KOGENATE FS, ADVATE, REFACTO, ELOCTATE, NUWIQ, ADYNOVATE, KOVALTRY, JIVI, and XYNTHA. Additionally, plasma-derived Factor VIII products, such as MONARC-M, MONOCLATE-P, HEMOFIL M, and KOATE-DVI, remain available. The latest addition, QFITLIA (fitusiran), a siRNA-based therapy approved in 2025, works by lowering antithrombin levels to enhance clot formation. It offers the convenience of only six injections per year and is effective for both inhibitor and non-inhibitor patients. HEMLIBRA has emerged as a first-line prophylactic therapy for Hemophilia A patients with inhibitors, although immune tolerance induction (ITI) therapy continues to be the gold standard. For those who encounter difficulties with ITI, HEMLIBRA provides an alternative, requiring less frequent FVIII dosing during ITI. Looking ahead, hemophilia management is shifting toward extended half-life factor therapies and cutting-edge modalities, including siRNA agents, bispecific antibodies, and gene therapy. The competitive landscape is expected to expand with the introduction of new long-acting factor products and next-generation therapeutic technologies. Hemophilia A Competitive Landscape The hemophilia A clinical trial landscape features several drugs in mid- and late-stage development that are expected to be approved in the near future. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including Mim8 (Novo Nordisk), DTX201 (Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]), giroctocogene fitelparvovec (SB-525 [Sangamo Therapeutics]), and others. Novo Nordisk's Mim8 is an advanced FVIIIa-mimicking bispecific antibody designed to provide sustained hemostatic control with convenient once-weekly or once-monthly prophylactic dosing for individuals with Hemophilia A, regardless of inhibitor status. Delivered subcutaneously, Mim8 functions by bridging activated Factors IXa and X (FIXa/FX) upon activation, effectively compensating for the absence of FVIII. This restores normal thrombin generation and promotes effective blood clotting. Ultragenyx Pharmaceutical's DTX201 represents the first clinical-stage gene therapy for Hemophilia A utilizing an Adeno-associated Virus (AAV) vector derived from the AAVhu37 serotype. It is a non-replicating AAV vector carrying a single-stranded DNA genome that encodes a B-domain–deleted FVIII, driven by a liver-specific promoter and enhancer sequence optimized for strong transgene expression. The AAVhu37 capsid, part of the hepatotropic clade E family, was chosen for its demonstrated ability in preclinical studies to enable efficient liver-targeted FVIII gene delivery, optimal biodistribution, and long-lasting FVIII expression. The anticipated launch of these emerging therapies are poised to transform the hemophilia A market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the hemophilia A market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about the FDA approved gene therapy hemophilia A @ FDA Approved AAV Gene Therapy Hemophilia A Recent Developments in the Hemophilia A Market In July 2025, Novo Nordisk announced that the FDA has approved ALHEMO injection for once-daily prophylactic use to prevent or reduce bleeding episodes in adults and children aged 12 years and older with hemophilia A or B (HA/HB) without inhibitors. This approval broadens the drug's indication beyond its initial December 2024 approval for patients with HA/HB with inhibitors. In June 2025, Pfizer Inc. reported positive topline findings from its Phase 3 BASIS trial (NCT03938792), which evaluated HYMPAVZI (marstacimab) in adults and adolescents with hemophilia A or B who have inhibitors. In May 2025, the FDA approved Antihemophilic factor (recombinant), PEGylated-aucl (Jivi; Bayer) for treating pediatric patients aged 7 and older with hemophilia A. The approval was supported by results from the Alfa-PROTECT and PROTECT Kids trials. In March 2025, Alnylam Pharmaceuticals announced the FDA's approval of Qfitlia™ (fitusiran), the sixth Alnylam-discovered RNAi therapeutic approved in the U.S. It is the first and only treatment to lower antithrombin (AT), aiming to promote thrombin generation, rebalance hemostasis, and prevent bleeds. What is Hemophilia A? Hemophilia A is an inherited bleeding disorder caused by a deficiency or low levels of clotting factor VIII. For many years, the standard treatment has been replacement therapy with FVIII. This began with FVIII obtained from donated whole blood, later advanced to plasma-derived FVIII, and is now primarily managed with recombinant human FVIII (rFVIII) products that have transformed hemophilia care. While the condition is typically diagnosed at birth, it can also be acquired later in life when the immune system develops antibodies that neutralize clotting factors, a rare condition known as acquired hemophilia. Hemophilia A Epidemiology Segmentation The hemophilia A epidemiology forecast section provides insights into the historical and current hemophilia A patient pool and forecasted trends for the leading markets. In the 7MM, the highest diagnosed prevalent cases of Hemophilia A were in the United States, accounting for nearly 14,900 cases in 2024. These cases are anticipated to increase in the upcoming years. The hemophilia A treatment market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets, segmented into: Total Diagnosed Prevalent Cases of Hemophilia A Diagnosed Prevalent Cases of Hemophilia A with Inhibitors or without Inhibitors Age-Specific Diagnosed Prevalent Cases of Hemophilia A Severity-specific Diagnosed Prevalent Cases of Hemophilia A Hemophilia A Market Report Metrics Details Study Period 2020–2034 Coverage 7MM [The United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Hemophilia A Market CAGR 2.9 % Hemophilia A Market Size in 2024 USD 12.2 Billion Key Hemophilia A Companies Novo Nordisk, Ultragenyx Pharmaceutical, Sangamo Therapeutics, ASC Therapeutics, Roche, Chugai, Sanofi, Alnylam Pharmaceuticals, Pfizer, Genentech, BioMarin Pharmaceutical, HEMA Biologics, LFB Pharmaceuticals, Octapharma, CSL Behring, Octapharma, Bayer, Takeda, and others Key Hemophilia A Therapies Mim8, DTX201 (Peboctocogene camaparvovec, BAY2599023), Giroctocogene fitelparvovec (SB-525), ASC618, NXT007/RG6512, QFITLIA, HYMPAVZI, ALHEMO, HEMLIBRA, ALTUVIIIO, ROCTAVIAN, SEVENFACT/CEVENFACTA, ESPEROCT, WILATE, JIVI, ADYNOVATE, AFSTYLA, NUWIQ, FEIBA, KOVALTRY, OBIZUR, ELOCTATE/Elocta, XYNTHA, and others Scope of the Hemophilia A Market Report Therapeutic Assessment: Hemophilia A current marketed and emerging therapies Hemophilia A Market Dynamics: Key Market Forecast Assumptions of Emerging Hemophilia A Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Hemophilia A Market Access and Reimbursement Download the report to evaluate the hemophilia A treatment company HEMLIBRA on hemophilia in women @ Royal Hemophilia A or B Table of Contents 1 Hemophilia A Market Key Insights 2 Hemophilia A Market Report Introduction 3 Executive Summary 4 Key Events 5 Epidemiology and Market Forecast Methodology 6 Hemophilia A Market Overview at a Glance 6.1 Market Share (%) Distribution of Hemophilia A by Therapy in 2020 6.2 Market Share (%) Distribution of Hemophilia A by Therapy in 2034 6.3 Market Share (%) Distribution of Hemophilia A by Inhibitor in 2020 6.4 Market Share (%) Distribution of Hemophilia A by Inhibitor in 2034 6.5 Market Share (%) Distribution of Hemophilia A by Noninhibitor in 2020 6.6 Market Share (%) Distribution of Hemophilia A by Noninhibitor in 2034 7 Hemophilia A: Disease Background and Overview 7.1 Introduction 7.2 Hemophilia A Signs and Symptoms 7.3 Hemophilia A Inheritance Pattern 7.4 Hemophilia A Molecular Pathogenesis 7.5 Hemophilia A Pathophysiology 7.6 Hemophilia A Risk Factors 7.7 Hemophilia A Diagnosis of Hemophilia A 8 Hemophilia A Treatment Algorithm, Current Treatment, and Medical Practices 9 Epidemiology and Patient Population 9.1 Key Findings 9.2 Assumptions and Rationale: 7MM 9.3 Total Diagnosed Prevalent Cases of Hemophilia A in the 7MM 9.4 United States 9.4.1 Total Diagnosed Prevalent Cases of Hemophilia A in the United States 9.4.2 Diagnosed Prevalent Cases of Hemophilia A With Inhibitors and Without Inhibitors in the United States 9.4.3 Age-Specific Diagnosed Prevalent Cases of Hemophilia A in the United States 9.4.4 Severity-specific Prevalent Cases of Hemophilia A in the United States 9.5 EU4 and the UK 9.6 Japan Epidemiology 10 Hemophilia A Patient Journey 11 Marketed Hemophilia A Therapies 11.1 Key Cross of Marketed Therapies 11.2 QFITLIA (fitusiran): Sanofi/Alnylam Pharmaceuticals 11.2.1 Product Description 11.2.2 Regulatory Milestones 11.2.3 Other Developmental Activities 11.2.4 Clinical Development 11.2.4.1 Clinical Trials Information 11.2.5 Safety and Efficacy 11.3 HYMPAVZI (marstacimab-hncq): Pfizer 11.4 ALHEMO (concizumab): Novo Nordisk 11.5 HEMLIBRA (emicizumab-kxwh): Chugai/Genentech/Roche 11.6 ALTUVIIIO (antihemophilic factor [recombinant], Fc-VWF-XTEN fusion protein-ehtl): Sanofi/Sobi 11.7 ROCTAVIAN (valoctocogene roxaparvovec): BioMarin Pharmaceutical 11.8 SEVENFACT/CEVENFACTA [coagulation factor VIIa (recombinant)-jncw]: HEMA Biologics/LFB Pharmaceuticals 11.9 ESPEROCT (N8-GP; Turoctocog alfa pegol): Novo Nordisk 11.10 WILATE: Octapharma 11.11 JIVI (BAY94-9027): Bayer 11.12 ADYNOVATE (Adynovi; BAX 855): Takeda Pharmaceutical 11.13 AFSTYLA (Lonoctocog alfa): CSL Behring 11.14 NUWIQ (simoctocog alfa): Octapharma 11.15 FEIBA: Takeda 11.16 KOVALTRY (BAY 81-8973): Bayer 11.17 OBIZUR (susoctocog alfa): Takeda 11.18 ELOCTATE/Elocta (efmoroctocog alfa): Sanofi/Sobi 11.19 XYNTHA (ReFacto AF): Pfizer 12 Emerging Hemophilia A Drugs 12.1 Key Cross Competition 12.2 Mim8: Novo Nordisk 12.2.1 Product Description 12.2.2 Other Development Activities 12.2.3 Clinical Development 12.2.3.1 Clinical Trial Information 12.2.4 Safety and Efficacy 12.2.5 Analyst View 12.3 DTX201 (Peboctocogene camaparvovec, BAY2599023): Ultragenyx Pharmaceutical 12.4 Giroctocogene fitelparvovec (SB-525): Sangamo Therapeutics 12.5 ASC618: ASC Therapeutics 12.6 NXT007/RG6512: Roche/Chugai 13 Hemophilia A Market: 7MM Analysis 13.1 Key Findings 13.2 Hemophilia A Market Outlook 13.3 Key Hemophilia A Market Forecast Assumptions 13.4 Conjoint Analysis 13.5 Total Market Size of Hemophilia A by Country in the 7MM 13.6 United States Hemophilia A Market Size 13.6.1 Total Market Size of Hemophilia A in the United States 13.6.2 Market Size of Hemophilia A by Therapies in the United States 13.7 EU4 and the UK Hemophilia A Market Size 13.8 Japan Hemophilia A Market Size 14 Hemophilia A Market Unmet Needs 15 Hemophilia A Market SWOT Analysis 16 KOL Views on Hemophilia A 17 Hemophilia A Market Access and Reimbursement 17.1 United States 17.2 EU4 and the UK 17.3 Japan 17.4 Market Access and Reimbursement of Hemophilia A 18 Bibliography 19 Hemophilia A Market Report Methodology Related Reports Hemophilia A Clinical Trial Analysis Hemophilia A Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Hemophilia A companies, including Jiangsu Gensciences Inc., Takeda, Gritgen Therapeutics Co., Ltd., Suzhou Alphamab Co., Ltd., Novo Nordisk, Pfizer, SK Plasma Co., Ltd., Hoffmann-La Roche, Shanghai Belief-Delivery BioMed Co., Ltd, ASC Therapeutics, Biocad, Expression Therapeutics, LLC, GCBiopharma, Amarna Therapeutics, Cabaletta Bio, among others. Acquired Hemophilia A Market Acquired Hemophilia A Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key acquired hemophilia A companies, including Pfizer, Novo Nordisk, Hema Biologics, Hoffman-La-Roche, Takeda, among others. Hemophilia Market Hemophilia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia companies, including Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk, among others. Hemophilia B Market Hemophilia B Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, UniQure Biopharma B.V., ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact Us Shruti Thakur [email protected] +14699457679 www.delveinsight.com Logo - https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg SOURCE DelveInsight Business Research, LLP