Haemophilia Treatment Market: By Type (Haemophilia A, B & C) By Therapy (On-demand & Prophylaxis) By Treatment (Replacement Therapy, ITI Therapy, and Gene Therapy) Regional Analysis & Forecast - 2026

Global Haemophilia treatment market was valued at $10.84bn in 2017 and is forecast to grow at a modest 5.12% CAGR between 2017 and 2024, culminating in 2024 global sales of $16.16bn. The increased Research and Development expenditure and exploration of new geographic regions in order to develop longer acting alternatives are the key drivers

Scope:

• Provides comprehensive understanding about pipeline Haemophilia therapeutics


• Includes all-inclusive pipeline product coverage with segregation by various stages of pipeline from Early development to In Approval.


• Provides a comparative analysis of key marketed products and pipeline drugs


• Provides key information on players involved in Haemophilia drugs.


• Provides in-depth coverage of key news related to Haemophilia therapeutics, including major mergers and acquisitions and product development updates such as clinical trial progression updates and regulatory updates

Haemophilia is a rare inherited bleeding disorder whereby blood does not clot normally. Predominantly, Haemophilia is classified as hemophilia A and hemophilia B. Hemophilia A occurs when the body is unable to produce adequate amounts of factor VIII (FVIII), while patients with hemophilia B are unable to produce adequate amounts of factor IX (FIX).  
Based on patient numbers and market opportunity, hemophilia A is the largest market, followed by hemophilia A patients that need bypassing agents to treat their disease. Hemophilia B is the third largest segment. Rare bleeding disorders, such as Von Willebrand disease, make up the remainder of the total hemophilia treatment market.  
 
“Treatment Algorithm”
The current treatment paradigm for most hemophilia patients remains predominantly first generation recombinant factor replacement therapies. Recombinant products now represent two third of the market. Prior to the development of recombinant technologies, plasma derived therapies remained the standard of care. Some patients have elected to switch to extended half-life factor replacement products which have recently entered the market and are expected to gain strong adoption over the next 5 years. Shire’s Adynovate and Biogen’s Eloctate are currently approved extended half-life products.  There is also a small subset of patients, ~10% of the market who have elected to remain on plasma derived therapies.  
 
Consultants view lower inhibitor formation and less frequent infusions as the biggest unmet needs in hemophilia and are optimistic that longer-acting products that require less frequent dosing could represent an improvement.  
 
"Shire Baxalta, Baxter, Biogen, Novo Nordisk, Pfizer and CSL are some of the key players in hemophilia treatment market"
 
In June 2016, Shire merged with Baxalta, giving Shire an attractive hemophilia product portfolio. Shires’ hemophilia franchise includes a strong breadth of products such as Advate, Feiba, Rixubis, Obizur, and plasma derived factors, such as Hemofil.
 
Shire’s Advate, acquired from Baxalta reported worldwide sales of ~$2bn in sales in 2015. Shire is expected to hold market leading position for the treatment of hemophilia A over the next 5 years supported by breadth of its hemophilia product franchise.
 
Novo Nordisk holds formidable position in the hemophilia market represented by its inhibitor treatment NovoSeven followed by Pfizer’s Benefix and Xyntha/Refacto, Bayer’s Kogenate, Biogen’s Eloctate and Alprolix, and CSL’s Helixate.
 
“Reimbursement coverage and Payer Insights”
Given that recombinant factor replacement therapies to treat hemophilia command a high price (well over $100K/year for an annual cost of prophylaxis treatment) and have favorable reimbursement, these therapies take modest price increases per year. The extended half-life products that have entered the market have come in at a premium to older factor replacement therapies on an annual cost of therapy basis, despite the less frequent dosing requirements.
 
“New Product Innovations”
 
The Haemophilia market has grown at a steady pace over the last 5 years and is expected to grow for next several years driven by greater prophylaxis treatment in the adult and pediatric populations, and new extended half-life factor replacement therapies entering the market.  
 
Bayer has filed its extended half-life FVIII product BAY94-9027 in Sep 2017 and Novo Nordisk has received FDA approval for N8-GP in feb 2019. 
 
Completely new approaches to the treatment of hemophilia, such as Roche/Chugai’s bispecific FIXa/X antibody ACE910 administered subcutaneously, could ultimately change the landscape for hemophilia treatment, but more data is needed in Phase III studies to ensure optimal dosing, long-term durability of therapy, and long-term safety with this approach can be achieved.

Sangamo Therapeutics has completed preclinical studies and entered into phase-I trials of its hemophilia SB-525 candidate. UniQure N.V. also started clinical trials on their therapeutic candidate AMT-061, AMT-180. Once approved, these products can gradually gain more share of this market over the next decade.