Hemophilia Treatment Market

Market Overview

Hemophilia is a rare inherited bleeding illness affecting the blood's clot ability. It is usually treated with clotting factor replacement treatment, injecting clotting factor concentrates into the bloodstream to reduce bleeding. Hemophilia is classified into two types: A (clotting factor VIII deficiency) and B (clotting factor IX deficiency). The Hemophilia Treatment Market is predicted to expand rapidly due to the increasing prevalence of hemophilia or bleeding disorders. As a result, the rising prevalence of hemophilia will likely drive up demand for hemophilia therapy over the projection period.

Furthermore, there is an increasing demand for effective medicines for treating Factor Vill insufficiency in emerging economies like India, which is expected to boost the growth of the Hemophilia Treatment Market during the forecast period. The variety of therapy and medications drives up demand for hemophilia treatment. Some of the most frequent therapies and medicines for hemophilia include inhibitor therapy, home remedies, free factor replacement therapy, antifibrinolytic pharmaceuticals, gene therapy, and physical therapy. These reasons have significantly boosted the hemophilia treatment market growth.

Market Dynamics

Global Hemophilia Treatment Market Drivers

Increasing Incidence of Hemophilia

The increasing frequency of hemophilia worldwide adds to the growing demand for hemophilia therapies. As awareness grows and diagnostic capabilities improve, more cases are detected and treated. The World Federation of Hemophilia (WFH) estimates that one in every 5,000 males is born with hemophilia A, and one in every 30,000 is born with hemophilia B. According to the CDC, hemophilia is a hereditary bleeding disorder that affects about one in every 5,617 live male births. This suggests that there are around 30,000 to 33,000 guys in the United States who have hemophilia. Improved awareness, screening, and diagnosis have resulted in earlier detection of people with hemophilia.

Hemophilia is commonly underdiagnosed or misdiagnosed, which may result in an underestimation of its prevalence. Improved awareness and diagnostic methods have helped identify more cases. So, on April 17, 2023, the WFH commemorated World Hemophilia Day, which strives to increase awareness of hemophilia and other inherited bleeding disorders. "Access for All: Prevention of Bleeds as the Global Standard of Care" was the 2023 theme. In early 2023, the FDA authorized Altuviiio (efanesoctocog alfa) as a long-term FVIII replacement therapy for persons with hemophilia A. Altuviiio can treat bleeds both preventively and on demand. Therefore, the rising incidence of hemophilia, fueled by improved diagnosis, awareness campaigns, and the creation of specialist treatment facilities, contributes to the hemophilia treatment market trend.

Global Hemophilia Treatment Market Restraints

High Cost

Hemophilia treatment, particularly factor replacement therapies, can be expensive. The exorbitant expense of these medicines could put a tremendous financial strain on healthcare systems, individuals, and their families. The annual cost of treating hemophilia is more than USD 393,000 per patient. Gene treatments can cure hemophilia, but each dose can cost up to USD 3.5 million. These treatments are not usually curative. Replacement therapy in India costs between Rs. 1,95,000 and Rs. 5,52,500, depending on the severity of the illness. 

Additionally, factor VIII and IX concentrates constitute the cornerstone of hemophilia treatment, particularly in severe instances. These clotting factor concentrations are generated from human plasma or created using recombinant technology. The cost of these factor concentrates can be high, and people with hemophilia frequently require recurrent infusions throughout their lifetimes. According to Value in Health, the average wholesale price (AWP) per international unit (IU) for Factor VIII (FVIII) concentrates is from USD 1.35 to USD 2.56, and for Factor IX (FIX) consolidates, the range is USD 1.31 to USD 5.28. Besides, According to TIFAC, the US Pharmaceutical Manufacturers Association anticipates that recombinant DNA treatments will cost roughly USD 250 million over the next ten years. This imposes a tremendous financial strain on healthcare systems, insurers, and individuals. 

Global Hemophilia Treatment Market Opportunity

Gene Therapy Advancement

Gene therapy for hemophilia can cure patients by restoring continuous endogenous expression of factor VIII or factor IX (FIX). Bleeds have been significantly reduced, with some studies reporting a 91.5% decrease. Continued research and developments in gene editing technologies may result in more effective and long-lasting hemophilia treatments. Several clinical trials have been done to determine the safety and efficacy of gene therapy for hemophilia. In these trials, functional copies of the defective clotting factor gene are introduced into patients' cells to allow clotting factor production. Notable instances include trials for hemophilia A and B. 

The approval of gene therapy for hemophilia poses a tremendous opportunity. The acceptance of these medications by regulatory authorities such as the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) paves the way for commercialization and extensive patient access. In 2023, the FDA approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for people with severe hemophilia A. This one-time infusion employs a vector to deliver a functioning gene that replaces the patient's faulty gene. The end consequence is increased clotting factor activity, which protects against bleeding. Furthermore, successful gene treatments can change the hemophilia treatment landscape by offering long-term or curative options. While the initial expenses of gene therapy may be considerable, the long-term economic impact could be significant, given the reduced need for lifelong factor replacement treatments.

Regional Analysis

The global hemophilia treatment market analysis is conducted in North America, Europe, Asia-Pacific, the Middle East and Africa, and Latin America.

North America Dominates the Global Market

North America is the most significant global hemophilia treatment market shareholder and is estimated to grow at a CAGR of 8.5% over the forecast period. It is predicted to do the same over the forecast period, owing to increased hemophilia instances, increased awareness of technologically improved products, and favorable regional government policies.  Furthermore, a study published in Frontiers in Immunology in April 2021 found that in the United States, patients with genetic illnesses such as hemophilia B benefited from Adeno-associated virus (AAV)-mediated gene transfer by achieving long-term therapeutic transgene expression. Such findings strengthen gene treatments for hemophilia, accelerating market expansion. 

Furthermore, increased initiatives from key industry players will likely propel market expansion. For example, in February 2022, BioMarin Pharmaceutical Inc. announced positive results from a two-year Phase 3 GENEr8-1 study analysis and an overall safety update for valoctocogene roxaparvovec. The 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) featured an investigational gene therapy for treating people with severe hemophilia A. 

Asia-Pacific is anticipated to exhibit a CAGR of 8.8% over the forecast period due to an increase in the number of people who have hemophilia. Furthermore, an increase in healthcare expenditure represents a profitable prospect for market expansion. In mainland China, the overall prevalence of hemophilia is 3.6 per 100,000 people, with males accounting for 5.5 per 100,000. Over 65,000 people in China are thought to have hemophilia. Thus, the hemophilia treatment market in China is expected to proliferate throughout the forecast period.

Similarly, advances in genetic research, new product releases and approvals, rising public health awareness, and an increase in diagnostic screening are projected to drive up demand for hemophilia treatment in China. For example, in August 2021, Belief Biomed's Gene Therapy for Hemophilia B (BBM-H901) received IND approval from China's National Medical Products Administration (NMPA), and the company completed dosing the first subject in the registrational gene therapy clinical trial for hemophilia B in 2022.

Europe was the main contributor in 2023 and will likely stay so throughout the hemophilia treatment business projection period, according to the market insights. This was linked to the soaring research activity for hemophilia therapy in Europe, owing to well-established healthcare facilities, recent technical developments, and a spike in demand for early hemophilia diagnosis due to increased disease awareness. For example, in February 2023, the European Commission approved Hemgenix, a new gene therapy, to treat hemophilia B. UniQure Inc. manufactures the product, while CSL Behring develops and distributes it. This approval is expected to significantly shift the hemophilia B patient treatment paradigm, alleviating the burden of life-long infusions.

Segmental Analysis

The global hemophilia treatment market is segmented based on type, drug therapy, and region.

The type is further segmented into Hemophilia A and Hemophilia B.

Hemophilia A accounts for the largest share of the market. The Hemophilia A segment accounted for most of the Hemophilia Treatment Market revenues. Hemophilia A is a genetic bleeding condition defined by the lack or absence of clotting factor VIII. Factor VIII is an essential protein in the blood clotting cascade, and its absence causes poor blood coagulation. Individuals with hemophilia A may have prolonged bleeding after an injury or surgery, and spontaneous bleeding into joints and muscles is common. The intensity of symptoms varies, with severe examples resulting in random bleeding episodes, whereas milder variants may only show after trauma. Factor VIII replacement therapy is commonly used to restore normal blood clotting function by infusing the missing clotting factor. Advances in prolonged half-life factor concentrates and current gene therapy research provide prospective pathways for more effective and convenient treatments for people with hemophilia A.

Hemophilia B, often known as Christmas disease, is another kind of hemophilia caused by a lack or absence of clotting factor IX. It is an X-linked genetic condition that affects primarily men, similar to Hemophilia A. Individuals with Hemophilia B have comparable symptoms, such as prolonged bleeding after traumas or surgeries and the possibility of spontaneous bleeding. The severity of symptoms varies, and factor IX levels influence the degree of clotting impairment. Hemophilia B is commonly treated with factor IX replacement, provided by infusions to control bleeding episodes. As with Hemophilia A, developments in longer half-life factor concentrates and current gene therapy research seek to improve treatment options and quality of life for people with Hemophilia B.

The market can be further bifurcated by drug therapy into Recombinant Coagulation Factor Concentrates Therapy, Plasma Derived Coagulation Factor Concentrates Therapy, and Non-Factor Replacement Therapy.

Recombinant Coagulation Factor Concentrates Therapy generates the most revenue in the market. The recombinant coagulation factor concentrates therapy category contributed the most revenue to the hemophilia treatment market size throughout the projection period, owing to the increased number of recombinant technology products accessible for treatment. Recombinant coagulation factor concentrates therapy uses clotting factors created using genetic engineering techniques. In this method, human genes are put into cells, usually bacteria or mammalian cells, which make the required clotting factor. Recombinant factor concentrates provide a pure and controlled source of clotting factors, lowering the risk of infections caused by plasma-derived products. This medication is often used in both Hemophilia A and Hemophilia B to replace a defective clotting factor. Advances in recombinant technology aim to increase these medicines' purity, safety, and efficacy, giving more dependable and convenient treatment alternatives for hemophilia patients.

Plasma-derived coagulation factor concentrate therapy extracts clotting factors from donated human blood plasma. These concentrates contain a variety of clotting factors, including factor VIII or IX, depending on the deficit being treated. Plasma-derived products have long been employed in hemophilia treatment, providing a consistent source of clotting components. However, developments in recombinant technology have resulted in growing acceptance due to the perceived safety benefits of preventing dangerous blood-borne diseases. Nonetheless, plasma-derived products remain a vital therapy option, especially in areas where recombinant medicines may be difficult to obtain.

Recent Developments

• December 2023- The FDA and EMA accepted Marstacimab regulatory submissions for treating Hemophilia A and B.
• December 2023- Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the primary analysis of the Phase III HAVEN 7 study confirmed the efficacy and safety of Hemlibra® (emicizumab) in previously untreated or partially treated babies with severe hemophilia A without factor VIII inhibitors. Hemlibra demonstrated significant bleed control in babies up to 12 months of age and was well tolerated.