The hemophilia market is expected to experience significant growth due to advancements in gene therapy, innovative treatments, and an increasing focus on personalized medicine. Rising awareness and early diagnosis, along with expanding healthcare infrastructure in emerging markets, are further driving market demand. Additionally, the growing prevalence of hemophilia, particularly in developing regions, is contributing to the market’s expansion.
New York, USA, Feb. 18, 2025 (GLOBE NEWSWIRE) — Hemophilia Market Insights: Key Trends and Developments Across Different Types of Hemophilia | DelveInsight
The hemophilia market is expected to experience significant growth due to advancements in gene therapy, innovative treatments, and an increasing focus on personalized medicine. Rising awareness and early diagnosis, along with expanding healthcare infrastructure in emerging markets, are further driving market demand. Additionally, the growing prevalence of hemophilia, particularly in developing regions, is contributing to the market’s expansion.
Hemophilia is a rare, inherited bleeding disorder in which the blood does not clot properly, leading to excessive bleeding or spontaneous bleeding episodes. It primarily affects males, though females can be carriers of the defective gene. The severity of hemophilia can vary depending on the level of clotting factor in the blood. The condition is caused by mutations in the genes responsible for producing clotting factors, particularly factor VIII (hemophilia A) or factor IX (hemophilia B). Without enough of these clotting factors, the blood cannot form a stable clot to stop bleeding.
The most common symptoms of hemophilia include frequent and prolonged bleeding episodes, particularly after injury or surgery. People with hemophilia may also experience spontaneous internal bleeding, especially into joints and muscles, which can lead to pain, swelling, and long-term joint damage. Bruising is often more extensive than in individuals without the condition, and excessive bleeding can occur even from minor cuts or dental work. In severe cases, bleeding can occur in vital organs, leading to life-threatening complications.
The primary cause of hemophilia is a genetic mutation that is inherited in an X-linked recessive pattern, meaning that it is carried on the X chromosome. Because males have only one X chromosome, they are more likely to develop the condition if they inherit the defective gene. Females, having two X chromosomes, are typically carriers unless both X chromosomes carry the mutated gene, which is much rarer. Genetic testing can identify carriers and diagnose hemophilia in newborns.
Treatment for hemophilia focuses on replacing the missing clotting factor through factor replacement therapy. This is typically done through intravenous infusion of clotting factors, and it may need to be administered regularly to prevent bleeding episodes or on-demand to control bleeding when it occurs. Advances in gene therapy hold promise as a potential long-term solution, aiming to provide a more permanent fix by introducing a functional gene for clotting factor production. Additionally, newer treatments, such as clotting factor bypassing agents, can help manage bleeding episodes, particularly in those with inhibitors to replacement factors. With proper treatment and care, many people with hemophilia can lead relatively normal lives, though they must take precautions to avoid injury.
Discover more about the hemophilia market in detail @ Hemophilia Market Report
DelveInsight has recently released epidemiology-based market reports focusing on hemophilia including Hemophilia A and Hemophilia B. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].
Additionally, the reports feature an examination of prominent companies working with their lead candidates in different stages of clinical development. Let’s deep dive into the assessment of these hemophilia markets individually.
Hemophilia A Market
Hemophilia A is characterized by bleeding episodes, although the initial ones in mild and moderate cases tend to be less severe, often leading patients to overlook early diagnosis. Additionally, many patients remain undiagnosed in the early stages due to limited diagnostic facilities.
The total treated prevalent population of Hemophilia A in the 7MM was assessed to be around 45,000 cases in 2023, and are projected to increase during the study period, as per DelveInsight’s analysis. Among the hemophilia A patients, i.e., with or without/non-inhibitors, the latter accounts for a higher number of hemophilia A cases.
Current treatments for hemophilia A mainly focus on replacing the missing clotting protein, FVIII, to enable proper clot formation and reduce or prevent bleeding. Historically, FVIII replacement was achieved through whole blood donations, plasma, and, with technological advances, recombinant human FVIII (rFVIII) therapies. These advancements have significantly improved the management of hemophilia A, offering more efficient and targeted treatment options. Recently, new therapies aimed at preventing bleeding through novel mechanisms are diversifying the available treatments for hemophilia A.
Several drugs approved for hemophilia A include ALTUVIIIO (Sanofi/Sobi), ROCTAVIAN (BioMarin Pharmaceutical), OBIZUR (Takeda), ALHEMO (Novo Nordisk), HEMLIBRA (Chugai/Genentech/Roche), ESPEROCT (Novo Nordisk), JIVI (Bayer), WILATE (Octapharma), ADYNOVATE (Takeda), among others. ROCTAVIAN gene therapy is a groundbreaking treatment, offering a one-time solution by delivering functional FVIII genes to cells, restoring normal blood clotting, and potentially replacing frequent injections. Priced at USD 2.9 million (wholesale acquisition cost), BioMarin has successfully navigated reimbursement processes in Europe and treated a patient in Germany in the second quarter of 2023 at a reduced cost of around USD 900,000.
Emerging therapies such as SPK-8011 (Roche [Spark Therapeutics]), fitusiran (Sanofi), marstacimab (Pfizer), SerpinPC (Centessa Pharmaceuticals), Mim8 (Novo Nordisk), and others are being developed to provide safe and effective treatment options. Companies like GeneVentiv Therapeutics are also working on advancements like Adeno-associated Virus (AAV)-based universal gene therapy for hemophilia (GENV-HEM [AAV8.Fva]).
Fitusiran is a small interfering RNA therapy administered subcutaneously, currently being developed for the preventive treatment of individuals with hemophilia A or B, regardless of inhibitor presence. It aims to reduce antithrombin, a protein that inhibits blood clotting, thereby promoting thrombin generation, rebalancing hemostasis, and preventing bleeding.
In January 2018, Sanofi and Alnylam Pharmaceuticals reorganized their RNAi therapeutics partnership to streamline and enhance the development and commercialization of select products aimed at treating rare genetic disorders. As part of this arrangement, Sanofi acquired global rights for the development and commercialization of fitusiran.
Marstacimab (PF-06741086) is a human monoclonal IgG1 antibody targeting the Kunitz 2 domain of Tissue Factor Pathway Inhibitor (TFPI). It is being developed as a preventive treatment to reduce or prevent bleeding episodes in individuals with severe hemophilia A or B (factor VIII or IX activity <1%), with or without inhibitors.
In September 2019, the FDA granted Fast Track Designation to marstacimab for use with inhibitors as a potential treatment for hemophilia A and B. In December 2023, Pfizer announced that the FDA accepted its Biologics License Application for marstacimab, based on results from the Phase III BASIS trial for patients with hemophilia A or B without inhibitors to Factor VIII (FVIII) or Factor IX (FIX).
BBM-H803 is Belief BioMed’s first gene therapy for hemophilia A and their second product to receive Investigational New Drug approval from the National Medical Products Administration (NMPA). In December 2022, BBM-H803 was granted Orphan Drug Designation by the US FDA.
Overall, it is expected that the hemophilia A treatment landscape will undergo significant changes from 2024 to 2034, driven by the launch of new therapies currently in clinical development. As per DelveInsight, the hemophilia A market is expected to grow across the 7MM from USD 11.1 billion in 2023 at a significant CAGR by 2034. However, market growth could be hindered by challenges such as delayed diagnosis, lack of awareness, and the high cost of treatment.
Dive deeper for rich insights into the Hemophilia A Market Trends and Analysis
Hemophilia B Market
Hemophilia B, also known as Christmas disease, is a rare inherited bleeding disorder where individuals have low levels of a blood protein called factor IX. Factor IX is a clotting factor, which is a specialized protein essential for blood clotting, a process that helps stop bleeding and promote healing after an injury. The severity of hemophilia B in a person depends on the amount of factor IX (FIX) present in their blood.
Among the 7MM, the US accounted for the highest diagnosed prevalent cases of hemophilia B in 2023, with approximately 4000 cases; these cases are expected to increase during the forecast period, as per DelveInsight. Based on severity, the majority of cases in the United States were classified as mild, i.e. nearly 40%.
The primary treatment for hemophilia B is replacement therapy. Recombinant factor IX products available in the US include BeneFIX, RIXUBIS, IXINITY, ALPROLIX, IDELVION, and REBINYN. In addition, bypassing agents like Feiba, Novoseven, and SEVENFACT, as well as human plasma-derived coagulation factor IX products such as AlphaNine SD and Mononine, have also been approved by the FDA.
Gene therapies offer the potential to cure hemophilia B. HEMGENIX, a gene therapy approved in November 2022, is priced at an extraordinary USD 3.5 million per dose, making it the most expensive drug globally and the first gene therapy approved for this rare disease.
The latest advancement in hemophilia B treatment is Pfizer’s BEQVEZ, which received approval in April 2024. Pfizer becomes the second company in the space, following CSL and uniQure’s HEMGENIX. Despite the possibility of offering a lower price, Pfizer has chosen to set the price of BEQVEZ at the same USD 3.5 million.
In the past two decades, significant progress has been made in hemophilia treatment, empowering patients to manage their condition more effectively. However, the development of inhibitors remains the biggest obstacle in treatment, complicating the control of bleeding episodes and making surgical procedures challenging. The absence of a curative treatment option severely impacts patients’ quality of life and daily activities. Gene therapy offers the potential for a lasting cure with a single drug administration, unlike the current range of treatments for hemophilia, a genetic bleeding disorder, which are still undergoing clinical trials.
The pipeline is brimming with promising therapies at different stages of development. Drugs like fitusiran, Marstacimab, SerpinPC, AskBio009/BAX 335, and others in clinical trials offer great potential as they include disease-modifying agents, symptomatic treatments, and therapies targeting specific pathways, addressing the unmet needs in hemophilia B treatment.
SerpinPC is a new APC inhibitor administered via subcutaneous injection, being developed as a potential treatment for hemophilia, irrespective of its severity or inhibitor status. It may also be explored for preventing bleeding in other bleeding disorders. Centessa Pharmaceuticals is leading the regulatory program for SerpinPC in hemophilia B. In May 2023, the US FDA awarded SerpinPC Fast Track Designation (FTD) for the treatment of hemophilia B patients.
AskBio009/BAX 335 is a gene therapy currently in the early stages of development for treating hemophilia B. It is an AAV8-based therapy that uses the clotting factor IX (FIX) Padua (R338L) gene to address hemophilia B. Takeda is presently conducting a Phase I/II clinical trial for AskBio009.
In a nutshell, the Hemophilia B market is driven by increasing awareness, advancements in gene therapies, and the growing adoption of recombinant factor IX therapies. As per DelveInsight, the total hemophilia B market size in the US was estimated to be approximately USD 2 billion in 2023, which is expected to grow during the forecast period (2024–2034).
Innovations in treatment options, including extended half-life therapies, are expanding market potential, while the rising prevalence of hemophilia B globally and the focus on improving patient quality of life contribute to market growth. Despite high treatment costs, ongoing research into novel therapies and government initiatives are expected to shape the market dynamics in the coming years.
For a comprehensive view of the hemophilia B market, check out the Hemophilia B Market Assessment
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