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


Dublin, Jan. 27, 2020 (GLOBE NEWSWIRE) -- The "Hemophilia A - Market Insights, Epidemiology, and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report deliver an in-depth understanding of the disease, historical and forecasted epidemiology as well as the market trends of Hemophilia A in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

Study Period: 2017-2030

Hemophilia A Treatment Algorithm

The treatment of Hemophilia A is mainly focused on the prophylaxis as there is no cure for hemophilia A. Treatment consists of replacing the missing clotting protein (factor VIII) and preventing the complications associated with the disorder. Replacement of this protein may be obtained through recombinant factor VIII, which is artificially created in a lab.

Several recombinant forms of factor VII are also approved for the treatment of hemophilia A. In some cases, subjects with hemophilia A may develop inhibitors' against the replacement factor VIII. Inhibitors are antibodies, which are specialized proteins created by the body's immune system to combat foreign or invading substances such as toxins or bacteria. The immune system may recognize replacement factor VIII as foreign' and create these antibodies (inhibitors), which target and destroy the replacement factor. For inhibitors treatment is mainly dependent on bypassing agents and immne tolerance therapy (ITI).

Hemophilia A Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology [segmented by Total Prevalence of Hemophilia A, Diagnosed and Treated Prevalent Population of Hemophilia A, Severity-Specific Prevalence of Hemophilia A, and Prevalence of Hemophilia A with Inhibitors and Without Inhibitors] scenario of Hemophilia A in the 7MM Countries covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Analysts have assessed that the total prevalent population of Hemophilia A in the 7MM was 42,458 in 2017. In addition to this, it was accessed that the total diagnosed and treated prevalent population of Hemophilia A in the 7MM was assessed to be 38,212 in 2017.

Hemophilia A Drug Chapters

Hemophilia A can be characterized by immediate or delayed bleeding or prolonged oozing after injuries, tooth extractions, or surgery or renewed bleeding after initial bleeding has stopped. Prolonged or delayed bleeding or wound hematoma formation after surgery is common. After circumcision, males with hemophilia A of any severity may have prolonged oozing, or they may heal normally without treatment. In severe hemophilia A, spontaneous joint bleeding is the most frequent symptom.

The treatment of hemophilia A is based upon the severity of disease if the disease is mild and moderate then on-demand (episodic) treatment will be given, and if the disease is severe, then continuous prophylaxis treatment is given. Some individuals with severe hemophilia A may receive periodic factor VIII infusions at regular intervals to prevent bleeding episodes and associated complications, such as joint damage. This is referred to as prophylactic therapy.

The treatment landscape of Hemophilia A comprises of recombinant or plasma derived Factor VIII (long and short acting) as well as antibody for non-inhibitors patients and for inhibitors Hemophilia A patients the treatment is mainly comprises of by passing agents and ITI.

The US Food and Drug Administration (FDA) has approved several recombinant forms of factor VIII for the treatment of hemophilia A, including HelixateFS (CSL Behring); Recombinate (Baxter); Kogenate FS (Bayer HealthCare); Advate (Baxter); ReFacto (Pfizer); Eloctate (Biogen-Idec), and Xyntha (Pfizer). Human plasma-derived preparations include Monarc-M (Baxter), Monoclate-P (CSL Behring), Hemofil M (Baxter), Koate-DVI (Kedrion), Nuwiq (Octapharma), Adynovate (Baxalta), and Jivi (Bayer). Individuals with mild or moderate hemophilia A may be treated with replacement therapy as needed (i.e., to treat specific bleeding episodes). This is referred to as on-demand' therapy.

Another breakthrough treatment for both Inhibitors and non-inhibitors patients is Hemlibra (Roche) which was approved by US FDA in 2017. It is a bispecific monoclonal antibody for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A.

Hemophilia A Market Outlook

The market size of Hemophilia A in seven major markets (7MM) is estimated to be USD 6,266 million in 2017. The United States accounts for the highest market size of Hemophilia A, i.e., USD 3,178 million, in comparison to the other major markets i.e., EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan. Among the EU5 countries, the United Kingdom had the highest market size in 2017, while Spain had the lowest market size of Hemophilia A in 2017.

Hemophilia A Drugs Uptake

With the launch of emerging therapies like Valoctocogene Roxaparvovec (BMN 270, a product of Biomarin Pharmaceutical), which is a gene therapy based on the use of adeno-associated virus (AAV) vectors, Concizumab (NN7415, a product of Novo Nordisk), Fitusiran (Sanofi Genzyme/ Alnylam Pharmaceuticals), BIVV001 (Sanofi), Marstacimab (Pfizer), SPK-8011 (Spark Therapeutics) and others, the market of Hemophilia A is anticipated to change during the forecast period (2019-2030).

Products that are anticipated to be launched during the forecast period are in late clinical stages of development, while others are in ongoing late clinical development stages. Some of the above-mentioned drug candidates have shown very promising results and it has been anticipated by Analysts that completion of clinical development and launch of these products in the market, might increase the market share of these companies, and besides this, patients of Hemophilia A will have better management practices.

According to our assessment, potential emerging candidates shall launch in the upcoming years of the forecast period [2019-2030], and with their anticipated launch, the market size of Hemophilia A will also experience significant growth.

Key Topics Covered

1. Key Insights

2. Executive Summary

3. Hemophilia A: Market Overview at a Glance
3.1. Total Market Share (%) Distribution of Hemophilia A in 2017
3.2. Total Market Share (%) Distribution of Hemophilia A in 2030

4. Hemophilia A: Disease Background and Overview
4.1. Introduction
4.1.1. Sign and Symptoms of Hemophilia A
4.1.2. Inheritance Pattern of Hemophilia A
4.1.3. Molecular Pathogenesis of Hemophilia A
4.1.4. Pathophysiology of Hemophilia A
4.1.5. Risk Factors of Hemophilia A
4.2. Diagnosis of Hemophilia A
4.2.1. Establishing the Diagnosis
4.2.2. Molecular Genetic Testing
4.2.3. Screening Tests
4.2.4. Clotting Factor Tests
4.2.5. Inhibitor Testing

5. Epidemiology and Patient Population
5.1. Epidemiology Key Findings
5.2. Assumptions and Rationale: 7MM
5.3. Epidemiology Scenario: 7MM
5.3.1. Total Prevalence of Hemophilia A in the 7MM
5.3.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the 7MM
5.3.3. Severity-Specific Prevalence of Hemophilia A in the 7MM
5.3.4. Prevalence of Hemophilia A with or without Inhibitors the 7MM

6. United States Epidemiology
6.1. Total Prevalence of Hemophilia A in the United States
6.2. Diagnosed and Treated Prevalent Population of Hemophilia A in the United States
6.3. Severity- Specific Prevalence of Hemophilia A in the United States
6.4. Prevalence of Hemophilia A with Inhibitors and Without Inhibitors in the United States

7. EU-5 Country-wise Epidemiology
7.1. Germany Epidemiology
7.1.1. Total Prevalence of Hemophilia A in Germany
7.1.2. Diagnosed and Treated Prevalent Population of Hemophilia A in Germany
7.1.3. Severity-Specific Prevalence of Hemophilia A in Germany
7.1.4. Prevalence of Hemophilia A with or without Inhibitors in Germany
7.2. France Epidemiology
7.3. Italy Epidemiology
7.4. Spain Epidemiology
7.5. United Kingdom Epidemiology

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

9. Treatment Algorithm, Current Treatment, and Medical Practices
9.1. Treatment and Management of Hemophilia A
9.1.1. Algorithm for treatment of Hemophilia A
9.2. Principles of care of Hemophilia A
9.3. Clotting Factors Concentrates
9.4. Prevention of Primary Manifestations
9.5. Prevention of Secondary Complications
9.6. Agents/Circumstances to Avoid
9.7. Inhibitors
9.8. Pregnancy Management

10. Unmet Needs

11. Marketed Products
11.1. List of Marketed Products in the 7MM
11.2. Esperoct (N8-GP; Turoctocog alfa pegol): Novo Nordisk
11.2.1. Product Description
11.2.2. Regulatory Milestones
11.2.3. Other Developmental Activities
11.2.4. Pivotal Clinical Trials
11.3. Jivi (formerly BAY94-9027): Bayer
11.4. Hemlibra (Emicizumab-kxwh): Chugai/ Genentech/Roche
11.5. Wilate: Octapharma
11.6. Adynovate (Adynovi; BAX 855): Takeda
11.7. Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein; Elocta (efmoroctocog alfa)]: Sanofi/Sobi
11.8. Afstyla (Lonoctocog alfa): CSL Behring
11.9. Kovaltry (BAY 81-8973): Bayer
11.10. Nuwiq (simoctocog alfa): Octapharma
11.11. NovoEight (Turoctocog alfa): Novo Nordisk
11.12. Obizur: Takeda
11.13. Kogenate FS (octocog alfa): Bayer
11.14. Xyntha (ReFacto AF): Pfizer
11.15. Feiba: Takeda

12. Emerging Therapies
12.1. Key Cross
12.2. Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical
12.2.1. Product Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.4. Safety and Efficacy
12.3. LR769: HEMA Biologics/LFB Biotechnologies
12.4. Concizumab (NN7415): Novo Nordisk
12.5. Fitusiran (ALN-AT3, SAR-439774): Sanofi Genzyme/ Alnylam Pharmaceuticals
12.6. BIVV001 (rFVIIIFc-VWF-XTEN): Sanofi
12.7. Marstacimab (PF-06741086): Pfizer
12.8. Marzeptacog alfa (Activated): Catalyst Biosciences
12.9. OPK88005: OPKO Biologics
12.10. SPK-8011: Spark Therapeutics

13. Hemophilia A: Seven Major Market Analysis
13.1. Key Findings
13.2. Market Size of Hemophilia A in 7MM
13.3. Market Size of Hemophilia A by Therapies in the 7MM

14. 7MM: Market Outlook

15. United States: Market Size
15.1. Total Market size of Hemophilia A in the United States
15.2. Market Size of Hemophilia A by Therapies in the US

16. EU-5 countries: Market Size and Outlook
16.1. Germany Market Size
16.1.1. Total Market size of Hemophilia A in Germany
16.1.2. Market Size of Hemophilia A by therapies in Germany
16.2. France Market Size
16.3. Italy Market Size
16.4. Spain Market Size
16.5. United Kingdom Market Size

17. Japan Market Outlook
17.1. Japan Market Size
17.1.1. Total Market size of Hemophilia A in Japan
17.1.2. Market Size of Hemophilia A by therapies in Japan

18. Market Drivers

19. Market Barriers

Companies Mentioned

  • Alnylam Pharmaceuticals
  • APCI
  • Bayer
  • Biomarin Pharmaceutical
  • Catalyst Biosciences
  • HEMA Biologics
  • LFB Biotechnologies
  • Novo Nordisk
  • Opko Biologics
  • Pfizer
  • Roche
  • Sangamo Therapeutics
  • Sanofi
  • Shire
  • Spark Therapeutics

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/a5rs9

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