Hemophilia A Overview

Hemophilia A Overview

Hemophilia A is an inherited bleeding disorder caused by a deficiency in clotting factor VIII, which impairs the blood's ability to clot properly. As a result, affected individuals may experience prolonged bleeding from even minor injuries, and spontaneous bleeding can occur, especially in the joints and muscles. This condition follows an X-linked recessive inheritance pattern, predominantly affecting males, while females usually act as carriers.

Symptoms can vary with the severity of the factor VIII deficiency. Those with severe cases may experience frequent, unprovoked bleeding, whereas milder forms typically cause bleeding only after surgery or trauma. Although there is no cure, clotting factor replacement therapy enables effective management, allowing most individuals to lead active lives.

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Epidemiological Segmentation (2020–2034, 7MM)

• Total Diagnosed Prevalence

• Severity-specific Prevalence

• Inhibitor-specific Prevalence

• Treated Prevalence
  
  

Epidemiology Highlights

• In 2023, around 45,500 individuals in the 7MM were living with Hemophilia A.

• The United States represented 30% of these cases.

• The EU4 and UK collectively accounted for 58%, while Japan contributed 12%.

• Hemophilia A constitutes 80–85% of all hemophilia diagnoses.

• The largest patient segment falls within the 19–44 age group.
  
  

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Market Overview

The Hemophilia A market size in the 7MM reached approximately USD 11.2 billion in 2023.

Market Growth Drivers

• Next-generation therapies offer improved convenience and reduced treatment frequency.

• Better adherence thanks to innovative delivery methods and patient-centric approaches.

• A shift toward therapies with enhanced safety and tolerability profiles.

• Increased investment in gene therapy research aiming for long-lasting solutions.
  
  

Market Challenges

• Development of inhibitors to FVIII therapies remains a significant clinical hurdle.

• Gene therapies come with high costs and reimbursement challenges that limit accessibility.

• Limited long-term data on new treatments delays broader adoption.

• Existing treatments can cause side effects, highlighting unmet needs despite progress.
  
  

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Emerging Treatments

Promising therapies under development include:

• Fitusiran

• RG6357

• SerpinPC

• Marstacimab

• And others
  
  

Key Players in the Hemophilia A Market

Leading companies advancing Hemophilia A treatments include:

• Sanofi (Genzyme) / Alnylam Pharmaceuticals

• Roche (Spark Therapeutics)

• ApcinteX

• Pfizer

• And others
  
  

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