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

DelveInsight’s ‘Hemophilia A—Market Insights, Epidemiology, and Market Forecast—2030’ 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.
Geography Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan
Study Period: 2017–2030
Hemophilia A—Disease Understanding and Treatment Algorithm
Hemophilia A is an X chromosome-linked genetic disorder caused by the mutations in the genes for factor VIII (FVIII). This clotting factor is a part of the intrinsic pathway of blood coagulation. Without enough factor VIII, the blood cannot clot properly to control bleeding. It is mainly a defect in the gene on chromosome X, females have two copies of the X chromosome. So, if the factor VIII gene on one chromosome does not work, the gene on the other chromosome can do the job of making enough factor VIII. On the other hand, males have only one X chromosome; if the factor VIII gene is missing on the male X chromosome, he will have hemophilia A that is why the most people with Hemophilia A are male.
The symptoms of Hemophilia A can vary greatly from one person to another, it ranges from mild to moderate to severe. The age of onset and frequency of bleeding episodes depend upon the amount of factor VIII protein and overall clotting ability of the blood. In most individuals, regardless of severity, bleeding episodes tend to be more frequent in childhood and adolescence than in adulthood.
Additionally, the diagnosis of Hemophilia A depends on the identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation, and a variety of specialized laboratory tests. The identification of a hemizygous F8 pathogenic variant on molecular genetic testing in a male proband confirms the diagnosis.
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.
DelveInsight’s 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 DelveInsight’s 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.
Hemophilia A Report Insights
• Total Prevalence of Hemophilia A
• Diagnosed and Treated Prevalent Population of Hemophilia A
• Severity-Specific Prevalence of Hemophilia A
• Prevalence of Hemophilia A with Inhibitors and without Inhibitors
• Therapeutic Approaches
• Pipeline Analysis
• Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies
Hemophilia A Report Key Strengths
• 12–Year Plus Forecast
• 7MM Coverage
• Epidemiology Segmentation
• Drugs Uptake
• Highly Analyzed Market
• Key Cross Competition
Hemophilia A Report Assessment
• Current Treatment Practices
• Unmet Needs
• Detailed Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers
Key Benefits
• This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving Hemophilia A market
• 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.