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Hemophilia A - Market Insights, Epidemiology, and Market Forecast - 2030

  • ID: 4911959
  • Report
  • January 2021
  • Region: Global
  • 276 pages
  • DelveInsight

FEATURED COMPANIES

  • Biomarin Pharmaceutical
  • Catalyst Biosciences
  • Novo Nordisk
  • Pfizer
  • Pfizer/ Sangamo
  • Roche/Spark therapeutics
  • MORE
This ‘Hemophilia A - Market Insights, Epidemiology, and Market Forecast - 2030’ report delivers an in-depth understanding of the Hemophilia A, historical and forecasted epidemiology as well as the Hemophilia A market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The Hemophilia A market report provides current treatment practices, emerging drugs, Hemophilia A market share of the individual therapies, current and forecasted Hemophilia A market size from 2018 to 2030 segmented by seven major markets. The Report also covers current Hemophilia A treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered
  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan
Study Period: 2018-2030

Hemophilia A Disease Understanding and Treatment Algorithm

Hemophilia A Overview

Hemophilia is a general term for a group of rare bleeding disorders caused by a congenital deficiency of certain clotting factors. The main form of hemophilia is hemophilia A; it is different from hemophilia B. The main difference between them is that hemophilia B is associated with a deficiency in factor IX (9) while hemophilia A is associated with a deficiency of factor VIII (8). Around 80% of people with hemophilia have hemophilia A. The mainstay treatment option has long been FVIII replacement therapy. Initially, FVIII replacement was accomplished by donated whole blood, subsequently by plasma, and currently by recombinant human FVIII (rFVIII) replacement therapies, which revolutionized the treatment of Hemophilia A. About 30% of severe hemophilia A patients develop neutralizing anti-FVIII alloantibodies (inhibitors), which render the FVIII replacement ineffective. The standard of care therapy for patients with inhibitors is to induce immune tolerance with high-dose, high-frequency FVIII, and treatment with bypassing agents (e.g., recombinant activated factor VII such as NovoSeven, FEIBA).

Hemophilia A Diagnosis

According to the CDC (Centers for Disease Control and Prevention), screening tests and clotting factor tests are involved in the diagnosis. Blood tests that show if the blood is clotting properly are screening tests. In order to diagnose a bleeding disorder, clotting factor tests, also called factor assays, are required. The type of hemophilia and the severity are shown by this blood test.

Hemophilia A Treatment

The current market for Hemophilia A includes Recombinant factor VIII (short and long-acting), Plasma-derived products, bypassing agents, bi-specific antibody, and a few others. The majority of the products in these classes were approved long back ago. Even after the launch of approved therapies now and then by regulatory authorities in the major markets, patients were stuck with treatments that had to be taken frequently to manage this disorder. However, the treatment landscape of hemophilia A have started to evolve over the period, as longer-acting hemophilia treatment options, and bispecific antibody have entered the hemophilia A market space. Besides this, gene therapy development is also reported for this disorder. Hence, the therapeutic market of Hemophilia A shall significantly rise in the coming years, owing to the expected launch of upcoming novel therapies, which will offer a curative option to the patients.

Hemophilia A Epidemiology

Key Findings

The overall prevalent cases of Hemophilia A in 2020 are projected to be around 43,243 in the 7MM that are expected to grow during the study period, i.e., 2018-2030.

The disease epidemiology covered in the report provides historical as well as forecasted Hemophilia A epidemiology [segmented as Total diagnosed Prevalent Pool of Hemophilia A, Severity-specific diagnosed Prevalent Pool of Hemophilia A, Inhibitor-specific Prevalent Pool of Hemophilia A and Total Treated Cases of Hemophilia A] scenario of Hemophilia A in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2018 to 2030.

Country Wise- Hemophilia A Epidemiology
  • In the United States, the total number of prevalent cases of Hemophilia A was 14,630 cases in the year 2020 which are expected to grow during the study period, i.e., 2018-2030.
  • In the year 2020, the total prevalent cases of Hemophilia A were 23,284 cases in EU-5 which are expected to grow during the study period, i.e., 2018-2030.
  • In Japan, the total number of prevalent cases of Hemophilia A was 5,329 cases in the year 2020 which are expected to grow during the study period, i.e., 2018-2030.
  • Severe Hemophilia A cases are more prominent in comparison to mild and moderate. Additionally, moderate and severe accounts for 75% of the Hemophilia A patient pool.
  • In the 7MM, approximately 30% of severe hemophilia A cases develop inhibitors, i.e., 6,422 patients with inhibitors in 2020. In addition to this, 70% of the Hemophilia A cases were observed without inhibitors. The number of cases for both categories will increase during the study period.
Hemophilia A Drug Chapters

Drug chapter segment of the Hemophilia A report encloses the detailed analysis of Hemophilia A developmental stage pipeline drugs. It also helps to understand the Hemophilia A clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Hemophilia A Marketed Drugs

Hemlibra (Emicizumab-kxwh): Roche (Chugai/ Genentech)

Hemlibra is a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors. In October 2018, Hemlibra was approved by the US Food and Drug Administration (FDA) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors (FVIII). However, this therapy, which was co-developed by Genentech, Chugai, and Roche, was already FDA approved in 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors. At present this candidate is being evaluated for mild to moderate hemophilia A, patients without FVIII inhibitors. Also, Chugai has started domestic phase III study for Hemlibra for the treatment of acquired hemophilia A.

Sevenfact [coagulation factor VIIa (recombinant)-jncw]: HEMA Biologics/LFB Pharmaceuticals

SEVENFACT from Hema Biologics is a bypassing agent that recently got an approval from the US FDA. It is also known by its other investigational names such as Novo7, rFVIIa, and LR769. The active ingredient of SEVENFACT is a recombinant analog of activated human coagulation Factor (F) VII (FVIIa). SEVENFACT is indicated for the treatment and control of bleeding episodes in adult and adolescent (12 years to < 18 years) hemophilia A and B patients with inhibitors to Factors VIII and IX. It is worth mentioning that it is not indicated for the treatment of congenital factor VII deficiency.

Esperoct (N8-GP; Turoctocog alfa pegol): Novo Nordisk

Esperoct (Turoctocog alfa pegol, also known as N8-GP), is an extended half-life glycopegylated factor VIII molecule indicated for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment, and control of bleeding episodes and perioperative management of bleeding. In February 2019, the US FDA approved the Biologics License Application (BLA) for Novo Nordisk’s long-acting factor VIII replacement treatment for adults and children with Hemophilia A. The approval was based on data from the largest pre-registration clinical program conducted in Hemophilia A evaluating previously-treated patients with severe hemophilia A and more than 5-year of clinical exposure.

Jivi (formerly BAY94-9027): Bayer

Jivi (antihemophilic factor [recombinant] PEGylated-aucl) is a product of Bayer which is specifically used in previously treated adults and adolescents (12 years of age and older) with hemophilia A. In August 2018, the US FDA approved Jivi for the routine prophylactic treatment of hemophilia A in previously treated adults and adolescents 12 years of age or older. The approval of Jivi was based on the PROTECT VIII study.

Note: Full and detailed list of emerging therapies will be provided in the final report.

Hemophilia A Emerging Drugs

Valoctocogene Roxaparvovec (BMN 270): Biomarin Pharmaceutical

Valoctocogene Roxaparvovec is under development by Biomarin Pharmaceutical, which is an experimental gene therapy based on the use of adeno-associated virus (AAV) vectors. Currently, Phase III GENEr8-3 trial is going on which started in June 2020, including 20 participants. This candidate has a Breakthrough Therapy designation granted by the US FDA, and Orphan Drug designation from the FDA and the EMA. In addition to this, recognizing its potential, EMA also granted access to its Priority Medicines (PRIME) regulatory initiative. In August 2020, the US FDA issued a Complete Response Letter to the Company's BLA for valoctocogene roxaparvovec. The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.

Concizumab: Novo Nordisk

Concizumab, which is under development by Novo Nordisk is a high-affinity monoclonal antibody against Tissue Factor Pathway Inhibitor intended for bleeding prevention after subcutaneous administration. Currently, it is in Phase III clinical trial for Hemophilia A and B with and without Inhibitors. In August 2020, Novo Nordisk announced that clinical trials for their investigational subcutaneous therapy concizumab will be resumed after an approximate five-month pause, initiated in March 2020 in response to non-fatal thrombotic events in three enrolled patients

Fitusiran: Sanofi/Alnylam Pharmaceuticals

Fitusiran is a subcutaneously administered small interfering RNA (siRNA) technology to target antithrombin (AT). Currently, it is in Phase III clinical trial for the treatment of Severe Hemophilia A and B Patients with inhibitors and without inhibitors. It works by silencing the gene responsible for AT, which inhibits the protein’s anticoagulant function, which further compensates for the imbalance caused by deficiencies in other clotting proteins, such as factor VIII (hemophilia A) or factor IX (hemophilia B). This candidate is based on an Alnylam delivery technology that enables increased potency and durability with subcutaneous (under-the-skin) injection, according to the companies.

Giroctocogene fitelparvovec (SB-525/PF-07055480): Pfizer/Sangamo Therapeutics

Sangamo in collaboration with Pfizer is investigating Giroctocogene fitelparvovec which is a recombinant AAV serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. Currently, the company is conducting phase III trials to attain better and clear results about the efficacy of this candidate.

RG6357 (SPK-8011): Roche (Spark Therapeutics)

RG6357 (SPK-8011), which is under development by Roche’s subsidiary Spark Therapeutics, is an intravenously administered novel bio-engineered AAV vector utilizing the AAV-LK03 capsid, also referred to as Spark200. Currently, a Phase I/II clinical research study is being conducted by the company to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A. In addition to this, the Phase III run-in study is also ongoing, and dosing participants in Phase III is expected to occur in 2021.

Note: Full and detailed list of emerging therapies will be provided in the final report.

Hemophilia A Market Outlook

Key Findings

The Hemophilia A market size in the 7MM is expected to change during the study period 2018-2030, at a CAGR of 10.2%. According to the estimates, the highest market size of Hemophilia A is accessed in the United States followed by the United Kingdom, Japan, and France in 2020.

The United States Market Outlook

In 2020, the total market size of Hemophilia A therapies is expected to increase at a CAGR of 11.0% during the study period (2018-2030).

EU-5 Countries: Market Outlook

In 2020, the total market size of Hemophilia A therapies is expected to increase ar a CAGR of 9.6% during the study period (2018-2030).

Japan Market Outlook

The total market size of Hemophilia A therapies in Japan is estimated to rise at a CAGR of 9.2%, during the study period (2018-2030).

Hemophilia A Pipeline Development Activities

The drugs which are in pipeline include:

1. Valrox (Valoctocogene Roxaparvovec): Biomarin Pharmaceutical

2. BIVV001 (rFVIIIFc-VWF-XTEN): Sanofi

3. RG6357 (SPK-8011): Roche/Spark therapeutics

4. Fitusiran: Genzyme, a Sanofi Company/ Alnylam Pharmaceuticals

5. Concizumab (NN7415): Novo Nordisk

6. Marstacimab (PF-06741086): Pfizer

7. Marzeptacog alfa: Catalyst Biosciences

8. Giroctocogene fitelparvovec (SB-525): Pfizer/ Sangamo

And others…….

Note: Detailed emerging therapies assessment will be provided in the final report.

Hemophilia A Drugs Uptake

Analysts Insight
Recombinant factors currently dominate the market in the non-inhibitors segment of hemophilia A. Hemlibra, however, is expected to disrupt the market of mostly in the recombinant FVIII class and is expected to have a more pronounced effect on short half-life products. Due to recently approved therapies such as Eloctate and Jivi, the effect would be less in the long-acting class because these candidates have a better half-life compared to old generation products. Hence, it is projected that the future of hemophilia care is going to trend toward prolonged half-life therapies as well as more innovative approaches like antibodies, and forthcoming siRNA, and gene therapies. If these treatments are eventually successfully commercialized, will change the current standard of care for hemophilia A patients.

Access and Reimbursement Scenario in Hemophilia A Therapies
Hemlibra with its strong clinical data and dosing convenience expected to become a blockbuster worldwide, and has gained valuable market access approval from NHS England in hemophilia A patients with inhibitors but will have to wait for broader use. However, as per the latest assessment by Institute for Quality and Efficiency in Health Care (IQWiG) in June 2019, there was no hint of an added benefit of Hemlibra in comparison with the ACT (Appropriate Comparator Therapy) in patients without inhibitors and have drawn similar conclusion previously (June 2018) for inhibitors patients as well.

KOL Views

To keep up with current market trends, we take KOLs and SME’s opinion working in the Hemophilia A domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Hemophilia A market trend. This will support the clients in potential upcoming novel treatment by identifying the over Hemophilia A scenario of the market and the unmet needs.

Competitive Intelligence Analysis

The publisher performs Competitive and Market Intelligence analysis of the Hemophilia A Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report
  • The report covers the descriptive overview of Hemophilia A, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies.
  • Comprehensive insight has been provided into the Hemophilia A epidemiology and treatment in the 7MM.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Hemophilia A is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of Hemophilia A market; historical and forecasted is included in the report, covering drug outreach in the 7MM.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Hemophilia A market.
Report Highlights
  • In the coming years, Hemophilia A market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia A R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Major players are involved in developing therapies for Hemophilia A. Launch of emerging therapies, will significantly impact the Hemophilia A market.
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Hemophilia A.
  • Our in-depth analysis of the pipeline assets across different stages of development different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the over Hemophilia A scenario of the research and development activities.
Hemophilia A Report Insights
  • Patient Population
  • Therapeutic Approaches
  • Hemophilia A Pipeline Analysis
  • Hemophilia A Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies
Hemophilia A Report Key Strengths
  • 10 Years Forecast
  • 7MM Coverage
  • Hemophilia A Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake
Hemophilia A Report Assessment
  • SWOT Analysis
  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Conjoint Analysis
  • Market Attractiveness
  • Market Drivers and Barriers
Key Questions Answered

Market Insights:
  • What was the Hemophilia A Market share (%) distribution in 2018 and how it would look like in 2030?
  • What would be the Hemophilia A total market size as well as market size by therapies across the 7MM during the study period (2018-2030)?
  • What are the key findings pertaining to the market across the 7MM and which country will have the largest Hemophilia A market size during the study period (2018-2030)?
  • At what CAGR, the Hemophilia A market is expected to grow in the 7MM during the study period (2018-2030)?
  • What would be the Hemophilia A market outlook across the 7MM during the study period (2018-2030)?
  • What would be the Hemophilia A market growth till 2030 and what will be the resultant market size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and a subsequent analysis of the associated trends?
  • Hemophilia A patient types/pool where unmet need is more and whether emerging therapies will be able to address the residual unmet need?
  • How emerging therapies are performing on the parameters like efficacy, safety, route of administration (RoA), treatment duration and frequencies on the basis of their clinical trial results?
  • Among the emerging therapies, what are the potential therapies which are expected to disrupt the Hemophilia A market?
Epidemiology Insights:
  • What is the disease risk, burden and unmet needs of the Hemophilia A?
  • What is the historical Hemophilia A patient pool in the seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
  • What would be the forecasted patient pool of Hemophilia A in the 7 major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
  • What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Hemophilia A?
  • Out of Hemophilia A the 7MM countries, which country would have the highest prevalent population of Hemophilia A during the study period (2018-2030)?
  • At what CAGR the population is expected to grow in the 7MM during the study period (2018-2030)?
  • What are the various recent and upcoming events which are expected to improve the diagnosis of Hemophilia A?
Current Treatment Scenario and Emerging Therapies:
  • What are the current options for the treatment of Hemophilia A?
  • What are the current treatment guidelines for the treatment of Hemophilia A in the US, Europe and Japan?
  • How many companies are developing therapies for the treatment of Hemophilia A?
  • How many therapies are developed by each company for the treatment of Hemophilia A?
  • How many emerging therapies are in mid stage, and late stage of development for the treatment of Hemophilia A?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hemophilia A therapies?
  • What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Hemophilia A and their status?
  • What are the key designations that have been granted for the emerging therapies for Hemophilia A?
  • What is the global historical and forecasted market of Hemophilia A?
Reasons to Buy
  • The report will help in developing business strategies by understanding trends shaping and driving the Hemophilia A market.
  • To understand the future market competition in the Hemophilia A market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Hemophilia A in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
  • Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for Hemophilia A market.
  • To understand the future market competition in the Hemophilia A market.
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Biomarin Pharmaceutical
  • Catalyst Biosciences
  • Novo Nordisk
  • Pfizer
  • Pfizer/ Sangamo
  • Roche/Spark therapeutics
  • MORE
1. Key Insights

2. Executive Summary

3. SWOT Analysis for Hemophilia A

4. Epidemiology and Market Forecast flow

5. Hemophilia A: Market Share of Hemophilia A at a Glance
5.1. Total Market Share (%) Distribution of Hemophilia A with Inhibitors in 2018
5.2. Total Market Share (%) Distribution of Hemophilia A with Inhibitors in 2030
5.3. Total Market Share (%) Distribution of Hemophilia A without Inhibitors in 2018
5.4. Total Market Share (%) Distribution of Hemophilia A without Inhibitors in 2030

6. Hemophilia A: Disease Background and Overview
6.1. Introduction
6.2. Sign and Symptoms
6.3. Inheritance Pattern
6.4. Molecular Pathogenesis
6.5. Pathophysiology
6.6. Risk Factors
6.7. Diagnosis of Hemophilia A
6.7.1. Establishing the Diagnosis
6.7.2. Molecular Genetic Testing
6.7.3. Screening Tests
6.7.4. Clotting Factor Tests
6.7.5. Inhibitor Testing

7. Epidemiology and Patient Population
7.1. Epidemiology Key Findings
7.2. Assumptions and Rationale: 7MM
7.3. Epidemiology Scenario: 7MM
7.3.1. Total Prevalence of Hemophilia A in the 7MM
7.3.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the 7MM
7.3.3. Severity- Specific Prevalence of Hemophilia A in the 7MM
7.3.4. Prevalence of Hemophilia A with or without Inhibitors the 7MM

8. United States Epidemiology
8.1. Total Prevalence of Hemophilia A in the United States
8.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the United States
8.3. Severity- Specific Prevalence of Hemophilia A in the United States
8.4. Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in the United States

9. EU-5 Country-wise Epidemiology
9.1. Germany Epidemiology
9.1.1. Total Prevalence of Hemophilia A in Germany
9.1.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Germany
9.1.3. Severity- Specific Prevalence of Hemophilia A in Germany
9.1.4. Prevalence of Hemophilia A with or without Inhibitors in Germany
9.2. France Epidemiology
9.2.1. Total Prevalence of Hemophilia A in France
9.2.2. Diagnosed and Treated Prevalent Population of Hemophilia A in France
9.2.3. Severity- Specific Prevalence of Hemophilia A in France
9.2.4. Prevalence of Hemophilia A with or without Inhibitors in France
9.3. Italy Epidemiology
9.3.1. Total Prevalence of Hemophilia A in Italy
9.3.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Italy
9.3.3. Severity-Specific Prevalence of Hemophilia A in Italy
9.3.4. Prevalence of Hemophilia A with or without Inhibitors in Italy
9.4. Spain Epidemiology
9.4.1. Total Prevalence of Hemophilia A in Spain
9.4.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Spain
9.4.3. Severity- Specific Prevalence of Hemophilia A in Spain
9.4.4. Prevalence of Hemophilia A with Inhibitors or Without-Inhibitors in Spain
9.5. United Kingdom Epidemiology
9.5.1. Total Prevalence of Hemophilia A in the United Kingdom
9.5.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the United Kingdom
9.5.3. Severity- Specific Prevalence of Hemophilia A in the United Kingdom
9.5.4. Prevalence of Hemophilia A with Inhibitors or Non-Inhibitors in the United Kingdom

10. Japan Epidemiology
10.1. Total Prevalence of Hemophilia A in Japan
10.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Japan
10.3. Severity- Specific Prevalence of Hemophilia A in Japan
10.4. Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in Japan

11. Treatment Algorithm, Current Treatment, and Medical Practices
11.1. Treatment and Management of Hemophilia A
11.1.1. Algorithm for treatment of Hemophilia A
11.2. Principles of care of Hemophilia A
11.3. Clotting Factors Concentrates
11.4. Prevention of Primary Manifestations
11.5. Prevention of Secondary Complications
11.6. Agents/Circumstances to Avoid
11.7. Inhibitors
11.8. Pregnancy Management

12. Patient Journey

13. Unmet Needs

14. Key Endpoints of Hemophilia A Treatment

15. Marketed Products
15.1. List of Marketed Products in the 7MM
15.2. Esperoct (N8-GP; Turoctocog alfa pegol): Novo Nordisk
15.2.1. Product Description
15.2.2. Regulatory Milestones
15.2.3. Other Developmental Activities
15.2.4. Pivotal Clinical Trials
15.3. Jivi (formerly BAY94-9027): Bayer
15.3.1. Drug Description
15.3.2. Regulatory Milestones
15.3.3. Other Developmental Activities
15.3.4. Pivotal Clinical Trial
15.4. Wilate: Octapharma
15.4.1. Product Description
15.4.2. Regulatory Milestones
15.4.3. Pivotal Clinical Trials
15.5. Adynovate (Adynovi; BAX 855): Takeda
15.5.1. Product Description
15.5.2. Regulatory Milestones
15.5.3. Other Developmental Activities
15.5.4. Pivotal Clinical Trials
15.6. Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein; Elocta (efmoroctocog alfa)]: Sanofi/Sobi
15.6.1. Product Description
15.6.2. Regulatory Milestones
15.6.3. Other Developmental Activities
15.6.4. Pivotal Clinical Trials
15.7. Afstyla (Lonoctocog alfa): CSL Behring
15.7.1. Product Description
15.7.2. Regulatory Milestones
15.7.3. Other Developmental Activities
15.7.4. Pivotal Clinical Trials
15.8. Kovaltry (BAY 81-8973): Bayer
15.8.1. Product Description
15.8.2. Regulatory Milestones
15.8.3. Other Developmental Activities
15.8.4. Pivotal Clinical Trials
15.9. Nuwiq (simoctocog alfa): Octapharma
15.9.1. Product Description
15.9.2. Regulatory Milestones
15.9.3. Other Developmental Activities
15.9.4. Pivotal Clinical Trials
15.10. NovoEight (Turoctocog alfa): Novo Nordisk
15.10.1. Product Description
15.10.2. Regulatory Milestones
15.10.3. Other Developmental Activities
15.10.4. Pivotal Clinical Trials
15.11. Obizur: Takeda
15.11.1. Product Description
15.11.2. Regulatory Milestones
15.11.3. Other Developmental Activities
15.11.4. Pivotal Clinical Trials
15.12. Kogenate FS (octocog alfa): Bayer
15.12.1. Product Description
15.12.2. Regulatory Milestones
15.12.3. Pivotal Clinical Trials
15.13. Xyntha (ReFacto AF): Pfizer
15.13.1. Product Description
15.13.2. Regulatory Milestones
15.13.3. Other Developmental Activities
15.13.4. Pivotal Clinical Trials
15.14. Feiba: Takeda
15.14.1. Product Description
15.14.2. Regulatory Milestones
15.14.3. Other Developmental Activities
15.14.4. Pivotal Clinical Trials
15.14.5. Ongoing Pipeline Activities
15.15. Hemlibra (Emicizumab-kxwh): Chugai/ Genentech/Roche
15.15.1. Product Description
15.15.2. Regulatory Milestones
15.15.3. Other Developmental Activities
15.15.4. Pivotal Clinical Trials
15.15.5. Ongoing Current Pipeline Activity
15.16. Sevenfact [coagulation factor VIIa (recombinant)-jncw]: HEMA Biologics/LFB Pharmaceuticals
15.16.1. Product Description
15.16.2. Regulatory Milestones
15.16.3. Other Developmental Activities
15.16.4. Pivotal Clinical Trials
15.16.5. Ongoing Current Pipeline Activity

16. Emerging Therapies
16.1. Key Cross
16.2. Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical
16.2.1. Product Description
16.2.2. Other Developmental Activities
16.2.3. Clinical Development
16.2.4. Safety and Efficacy
16.3. BIVV001 (rFVIIIFc-VWF-XTEN): Sanofi
16.3.1. Product Description
16.3.2. Other Developmental Activities
16.3.3. Clinical Development
16.3.4. Safety and Efficacy
16.4. OPK88005: OPKO Biologics
16.4.1. Product Description
16.4.2. Other Developmental Activities
16.4.3. Clinical Development
16.5. RG6357 (SPK-8011): Roche (Spark Therapeutics)
16.5.1. Product Description
16.5.2. Other Developmental Activities
16.5.3. Clinical Development
16.5.4. Safety and Efficacy
16.6. RG6358 (SPK-8016): Roche (Spark Therapeutics)
16.6.1. Product Description
16.6.2. Other Developmental Activities
16.6.3. Clinical Development
16.7. SerpinPC: ApcinteX
16.7.1. Product Description
16.7.2. Other Developmental Activities
16.7.3. Clinical Development
16.8. SIG-001 (Encapsulated Allogeneic FVIII Cell Therapy): Sigilon Therapeutics
16.8.1. Product Description
16.8.2. Other Developmental Activities
16.8.3. Clinical Development
16.9. Concizumab (NN7415): Novo Nordisk
16.9.1. Product Description
16.9.2. Other Developmental Activities
16.9.3. Clinical Development
16.9.4. Safety and Efficacy
16.10. Fitusiran (ALN-AT3, SAR-439774): Sanofi (Genzyme)/ Alnylam Pharmaceuticals
16.10.1. Product Description
16.10.2. Other Developmental Activities
16.10.3. Clinical Development
16.10.4. Safety and Efficacy
16.11. Marstacimab (PF-06741086): Pfizer
16.11.1. Product Description
16.11.2. Other Developmental Activities
16.11.3. Clinical Development
16.11.4. Safety and Efficacy
16.12. Marzeptacog alfa (Activated) (PF-05280602): Catalyst Biosciences
16.12.1. Product Description
16.12.2. Other Developmental Activities
16.12.3. Clinical Development
16.12.4. Safety and Efficacy
16.13. BAY2599023 (DTX201 AAV FVIII): Bayer/Ultragenyx Pharmaceutical
16.13.1. Product Description
16.13.2. Other Developmental Activities
16.13.3. Clinical Development
16.13.4. Safety and Efficacy
16.14. Giroctocogene fitelparvovec (SB-525 or PF-07055480): Pfizer/Sangamo Therapeutics
16.14.1. Product Description
16.14.2. Other Developmental Activities
16.14.3. Clinical Development
16.14.4. Safety and Efficacy
16.15. NNC0365-3769 A (Mim8): Novo Nordisk A/S
16.15.1. Product Description
16.15.2. Other Developmental Activities
16.15.3. Clinical Development
16.16. TAK-754 (BAX 888/SHP654): Takeda (Shire)
16.16.1. Product Description
16.16.2. Other Developmental Activities
16.16.3. Clinical Development

17. Hemophilia A: Seven Major Market Analysis
17.1. Key Findings
17.2. Market Outlook
17.3. 7MM Market Size
17.3.1. Market Size of Hemophilia A in 7MM
17.3.2. Market Size of Hemophilia A by Therapies in the 7MM
17.4. United States: Market Size
17.4.1. Total Market size of Hemophilia A in the United States
17.4.2. Market Size of Hemophilia A by Therapies in the US
17.5. EU-5 countries: Market Size
17.6. Germany Market Size
17.6.1. Total Market size of Hemophilia A in Germany
17.6.2. Market Size of Hemophilia A by therapies in Germany
17.7. France Market Size
17.7.1. Total Market size of Hemophilia A in France
17.7.2. Market Size of Hemophilia A by therapies in France
17.8. Italy Market Size
17.8.1. Total Market size of Hemophilia A in Italy
17.8.2. Market Size of Hemophilia A by therapies in Italy
17.9. Spain Market Size
17.9.1. Total Market size of Hemophilia A in Spain
17.9.2. Market Size of Hemophilia A by therapies in Spain
17.10. United Kingdom Market Size
17.10.1. Total Market size of Hemophilia A in the United Kingdom
17.10.2. Market Size of Hemophilia A by therapies in the United Kingdom
17.11. Japan Market Size
17.11.1. Total Market size of Hemophilia A in Japan
17.11.2. Market Size of Hemophilia A by therapies in Japan

18. Access and Reimbursement Overview of Hemophilia A
18.1. Access and Reimbursement Overview for Current and Future Therapies
18.1.1. Current Therapies: Hemlibra (Emicizumab)
18.2. HTA Assessment of Hemlibra by NHS and IQWiG
18.3. IQWIG assessment for some other therapies
18.4. Future Therapies: Gene Therapy

19. Market Drivers

20. Market Barriers

21. Appendix
21.1. Bibliography
21.2. Report Methodology

22. Publisher Capabilities

23. Disclaimer

24. About the Publisher

List of Tables
Table 1 Summary of Hemophilia A Market, Epidemiology, and Key Events (2018-2030)
Table 2 Approximate Frequency of Bleeding at Different Sites
Table 3 Sites of Bleeding In Hemophilia
Table 4 Levels of Factors in Hemophilia A
Table 5 Summary of sources explored for the epidemiology of hemophilia A
Table 6 Total Prevalence of Hemophilia A in the 7MM (2018-2030)
Table 7 Diagnosed and Treated Prevalent Population of Hemophilia A in the 7MM (2018-2030)
Table 8 Severity- Specific Prevalence of Hemophilia A in the 7MM (2018-2030)
Table 9 Prevalence of Hemophilia A with or without Inhibitors the 7MM (2018-2030)
Table 10 Total Prevalence of Hemophilia A in the United States (2018-2030)
Table 11 Diagnosed and Treated Prevalent Population of Hemophilia A in the United States (2018-2030)
Table 12 Severity- Specific Prevalence of Hemophilia A in the United States (2018-2030)
Table 13 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in the United States (2018-2030)
Table 14 Total Prevalence of Hemophilia A in Germany (2018-2030)
Table 15 Diagnosed and Treated Prevalent Population of Hemophilia A in Germany (2018-2030)
Table 16 Severity- Specific Prevalence of Hemophilia A in Germany (2018-2030)
Table 17 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in Germany (2018-2030)
Table 18 Total Prevalence of Hemophilia A in France (2018-2030)
Table 19 Diagnosed and Treated Prevalent Population of Hemophilia A in France (2018-2030)
Table 20 Severity- Specific Prevalence of Hemophilia A in France (2018-2030)
Table 21 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in France (2018-2030)
Table 22 Total Prevalence of Hemophilia A in Italy (2018-2030)
Table 23 Diagnosed and Treated Prevalent Population of Hemophilia A in Italy (2018-2030)
Table 24 Severity-Specific Diagnosed Prevalence of Hemophilia A in Italy (2018-2030)
Table 25 Prevalence of Hemophilia A with or Non-Inhibitors in Italy (2018-2030)
Table 26 Total Prevalence of Hemophilia A in Spain (2018-2030)
Table 27 Diagnosed and Treated Prevalent Population of Hemophilia A in Spain (2018-2030)
Table 28 Severity-Specific Prevalence of Hemophilia A in Spain (2018-2030)
Table 29 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in Spain (2018-2030)
Table 30 Total Prevalence of Hemophilia A and B in the United Kingdom (2018-2030)
Table 31 Diagnosed and Treated Prevalent Population of Hemophilia A in the United Kingdom (2018-2030)
Table 32 Severity-Specific Prevalence of Hemophilia A in the United Kingdom (2018-2030)
Table 33 Prevalence of Hemophilia A with or Non-Inhibitors in the United Kingdom (2018-2030)
Table 34 Total Prevalence of Hemophilia A in Japan (2018-2030)
Table 35 Diagnosed and Treated Prevalent Population of Hemophilia A in Japan (2018-2030)
Table 36 Severity- Specific Prevalence of Hemophilia A in Japan (2018-2030)
Table 37 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in Japan (2018-2030)
Table 38 Feiba; Clinical Trial Description, 2020
Table 39 Hemlibra (Emicizumab-kxwh); Clinical Trial Description, 2020
Table 40 Dosing for Treatment and Control of Bleeding
Table 41 Sevenfact; Clinical Trial Description
Table 42 Key Cross
Table 43 Valoctocogene Roxaparvovec (BMN 270); Clinical Trial Description
Table 44 BIVV001; Clinical Trial Description
Table 45 OPK88005 (MOD-1504); Clinical Trial Description
Table 46 RG6357 (SPK-8011); Clinical Trial Description
Table 47 RG6358 (SPK-8016); Clinical Trial Description
Table 48 SerpinPC; Clinical Trial Description
Table 49 SIG-001; Clinical Trial Description
Table 50 Concizumab; Clinical Trial Description
Table 51 Concizumab; Clinical Trial Description
Table 52 Marstacimab (PF-06741086); Clinical Trial Description
Table 53 BAY2599023 (DTX201 AAV FVIII); Clinical Trial Description
Table 54 Giroctocogene fitelparvovec (SB-525 or PF-07055480); Clinical Trial Description
Table 55 NNC0365-3769 A (Mim8); Clinical Trial Description
Table 56 TAK754 (SHP654 or BAX 888); Clinical Trial Description
Table 57 Market Size of Hemophilia A in the 7MM, in USD Million (2018-2030)
Table 58 The US Market Size of Hemophilia A by therapies, in USD Million (2018-2030)
Table 59 Market Size of Hemophilia A by therapies (Inhibitors), in the United States, in USD Million (2018-2030)
Table 60 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the United States, in USD Million (2018-2030)
Table 61 Germany Market Size of Hemophilia A by therapies, in USD Million (2018-2030)
Table 62 Market Size of Hemophilia A by therapies (Inhibitors) in Germany, in USD Million (2018-2030)
Table 63 Market Size of Hemophilia A by therapies (Non-Inhibitors) in France, in USD Million (2018-2030)
Table 64 France Market Size of Hemophilia A by therapies, in USD Million (2018-2030)
Table 65 Market Size of Hemophilia A by therapies (Inhibitors) in France, in USD Million (2018-2030)
Table 66 Market Size of Hemophilia A by therapies (Non-Inhibitors) in France, in USD Million (2018-2030)
Table 67 Italy Market Size of Hemophilia A by therapies, in USD Million (2018-2030)
Table 68 Market Size of Hemophilia A by therapies (Inhibitors) in Italy, in USD Million (2018-2030)
Table 69 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Italy, in USD Million (2018-2030)
Table 70 Spain Market Size of Hemophilia A by therapies, in USD Million (2018-2030)
Table 71 Market Size of Hemophilia A by therapies (Inhibitors) in Spain, in USD Million (2018-2030)
Table 72 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Spain, in USD Million (2018-2030)
Table 73 The UK Market Size of Hemophilia A by therapies, in USD Million (2018-2030)
Table 74 Market Size of Hemophilia A by therapies (Inhibitors) in the United Kingdom, in USD Million (2018-2030)
Table 75 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the United Kingdom, in USD Million (2018-2030)
Table 76 Japan Market Size of Hemophilia A by therapies, in USD Million (2018-2030)
Table 77 Market Size of Hemophilia A by therapies (Inhibitors) in Japan, in USD Million (2018-2030)
Table 78 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Japan, in USD Million (2018-2030)
Table 79 Issues of the Benefit Assessment of Emicizumab

List of Figures
Figure 1 SWOT Analysis for Hemophilia A
Figure 2 Coagulation Cascade and the Effects of Recombinant Activated Factor Vll (rFVlla)
Figure 3 Inheritance Pattern of Hemophilia A
Figure 4 Established and Proposed Risk Factors for Inhibitor Development
Figure 5 Screening Tests for Hemophilia A
Figure 6 Total Prevalence of Hemophilia A in the 7MM (2018-2030)
Figure 7 Diagnosed and Treated Prevalent Population of Hemophilia A in the 7MM (2018-2030)
Figure 8 Severity- Specific Prevalence of Hemophilia A in the 7MM (2018-2030)
Figure 9 Prevalence of Hemophilia A with or without Inhibitors in the 7MM (2018-2030)
Figure 10 Total Prevalent Population of Hemophilia A in the United States (2018-2030)
Figure 11 Diagnosed and Treated Prevalent Population of Hemophilia A in the United States (2018-2030)
Figure 12 Severity- Specific Prevalence of Hemophilia A in the United States (2018-2030)
Figure 13 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in the United States (2018-2030)
Figure 14 Total Prevalence of Hemophilia A in Germany (2018-2030)
Figure 15 Diagnosed and treated Prevalent Population of Hemophilia A in Germany (2018-2030)
Figure 16 Severity- Specific Prevalence of Hemophilia A in Germany (2018-2030)
Figure 17 Prevalence of Hemophilia A with or Non-Inhibitors in Germany (2018-2030)
Figure 18 Total Prevalence of Hemophilia A in France (2018-2030)
Figure 19 Diagnosed and Treated Prevalent Population of Hemophilia A in France (2018-2030)
Figure 20 Severity-Specific Prevalence of Hemophilia A in France (2018-2030)
Figure 21 Diagnosed Prevalence of Hemophilia A with or Non-Inhibitors in France (2018-2030)
Figure 22 Total Prevalence of Hemophilia A in Italy (2018-2030)
Figure 23 Diagnosed and Treated Prevalent Population of Hemophilia A in Italy (2018-2030)
Figure 24 Severity- Specific Prevalence of Hemophilia A in Italy (2018-2030)
Figure 25 . Prevalence of Hemophilia A with or Non-Inhibitors in Italy (2018-2030)
Figure 26 Total Prevalence of Hemophilia A and B in Spain (2018-2030)
Figure 27 Diagnosed and Treated Prevalent Population of Hemophilia A in Spain (2018-2030)
Figure 28 Severity- Specific Prevalence of Hemophilia A in Spain (2018-2030)
Figure 29 Prevalence of Hemophilia A with or Non-Inhibitors in Spain (2018-2030)
Figure 30 Total Prevalence of Hemophilia A and B in the United Kingdom (2018-2030)
Figure 31 Diagnosed and Treated Prevalent Population of Hemophilia A in the United Kingdom (2018-2030)
Figure 32 Severity- Specific Prevalence of Hemophilia A in the United Kingdom (2018-2030)
Figure 33 Prevalence of Hemophilia A with or Non-Inhibitors in the United Kingdom (2018-2030)
Figure 34 Total Prevalent Population of Hemophilia A in Japan (2018-2030)
Figure 35 Diagnosed and Treated Patient Population of Hemophilia A in Japan (2018-2030)
Figure 36 Severity- Specific Prevalence of Hemophilia A in Japan (2018-2030)
Figure 37 Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in Japan (2018-2030)
Figure 38 Treatment Algorithm for the patient with Hemophilia A
Figure 39 Management Algorithm for the patient with Inhibitors
Figure 40 Improvements in Hemophilia Therapy
Figure 41 Unmet Needs of Hemophilia A
Figure 42 Key Endpoints of Hemophilia A Treatment
Figure 43 Mechanism of SerpinPC inhibiting APC
Figure 44 Market Size of Hemophilia A in the 7MM, in USD Million (2018-2030)
Figure 45 Market Size of Hemophilia A by therapies (Inhibitors) in the 7MM, in USD Million (2018-2030)
Figure 46 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the 7MM, in USD Million (2018-2030)
Figure 47 Market Size of Hemophilia A in the US, USD Millions (2018-2030)
Figure 48 Market Size of Hemophilia A by therapies (Inhibitors) in the United States, in USD Million (2018-2030)
Figure 49 Market Size of Hemophilia A by therapies (Non-Inhibitors) the United States, in USD Million (2018-2030)
Figure 50 Market Size of Hemophilia A in Germany, USD Millions (2018-2030)
Figure 51 Market Size of Hemophilia A by therapies (Inhibitors) in Germany, in USD Million (2018-2030)
Figure 52 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Germany, in USD Million (2018-2030)
Figure 53 Market Size of Hemophilia A in France, USD Millions (2018-2030)
Figure 54 Market Size of Hemophilia A by therapies (Inhibitors) in France, in USD Million (2018-2030)
Figure 55 Market Size of Hemophilia A by therapies (Non-Inhibitors) in France, in USD Million (2018-2030)
Figure 56 Market Size of Hemophilia A in Italy, USD Millions (2018-2030)
Figure 57 Market Size of Hemophilia A by therapies (Inhibitors) in Italy, in USD Million (2018-2030)
Figure 58 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Italy, in USD Million (2018-2030)
Figure 59 Market Size of Hemophilia A in Spain, USD Millions (2018-2030)
Figure 60 Market Size of Hemophilia A by therapies (Inhibitors) in Spain, in USD Million (2018-2030)
Figure 61 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Spain, in USD Million (2018-2030)
Figure 62 Market Size of Hemophilia A in the United Kingdom, USD Million (2018-2030)
Figure 63 Market Size of Hemophilia A by therapies (Inhibitors) in the United Kingdom, in USD Million (2018-2030)
Figure 64 Market Size of Hemophilia A by therapies (Non-Inhibitors) in the United Kingdom, in USD Million (2018-2030)
Figure 65 Market Size of Hemophilia A in Japan, in USD Million (2018-2030)
Figure 66 Market Size of Hemophilia A by therapies (Inhibitors) in Japan, in USD Million (2018-2030)
Figure 67 Market Size of Hemophilia A by therapies (Non-Inhibitors) in Japan, in USD Million (2018-2030)
Figure 68 Factors affecting the Management of Hemophilia Patients
Figure 69 Market Drivers of Hemophilia A
Figure 70 Market Barriers
Note: Product cover images may vary from those shown
  • Biomarin Pharmaceutical
  • Sanofi
  • Roche/Spark therapeutics
  • Genzyme, a Sanofi Company/ Alnylam Pharmaceuticals
  • Novo Nordisk
  • Pfizer
  • Catalyst Biosciences
  • Pfizer/ Sangamo
Note: Product cover images may vary from those shown
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