市場調查報告書
商品編碼
1576098
血友病的全球市場的評估:各類型,不同治療,各類藥物,各治療法,各流通管道,各地區,機會,預測(2017年~2031年)Hemophilia Market Assessment, By Type, By Treatment, By Drug Class, By Therapy, By Distribution Channel, By Region, Opportunities and Forecast, 2017-2031F |
全球血友病市場規模預計將從 2023 年的 131.1 億美元增至 2031 年的 201.2 億美元,預計 2024 年至 2031 年的複合年增長率為 5.50%。由於盛行率的增加以及投資者和市場參與者興趣的增加,預計血友病市場需求將在預測期內呈指數級增長。
血友病是一種極為罕見的遺傳性疾病,由於體內缺乏凝血因子,血液凝固受到抑制。這種疾病是一種X連鎖隱性遺傳疾病,因此較常見於男性。此外,血友病治療市場的推動因素包括用於治療血友病的療法領域的發展、向標靶治療和聯合治療的範式轉變,以及由於認識的提高而提高的診斷率,預計該市場將以比預測更快的速度成長。此外,政府為消除血友病做出了重大努力,批准了用於治療血友病的產品,無論是藥物還是治療方法,從而補充了這種增長。 2024年4月,輝瑞宣布BEQVEZ(fidanacogene elaparvovec-dzkt)獲得美國食品藥物管理局(FDA)核准用於治療成人中度重度B型血友病。這包括目前正在使用 FIX 預防性治療的患者、曾發生危及生命的出血的患者、曾多次發生嚴重自發性出血的患者,以及在 FDA 批准的測試中顯示具有 AAVRh74var 衣殼中和抗體的患者。 BEQVEZ 是一種一次性治療,有可能讓 B 型血友病患者自行修復。
本報告提供全球血友病市場相關調查分析,提供市場規模與預測,市場動態,主要企業的形勢等資訊。
Global hemophilia market is projected to witness a CAGR of 5.50% during the forecast period 2024-2031, growing from USD 13.11 billion in 2023 to USD 20.12 billion in 2031. The market demand for hemophilia is anticipated to thrive drastically in the forecast years due to rising prevalence and the growing interest of investors and market players.
Hemophilia is a very rare genetic disorder where blood clotting gets impeded due to the lack of blood clotting factors present in the body. The foremost incidence of this disease takes place among males because this is an X-linked recessive disorder. Further catalyzed by developments in the therapy areas used for the treatment of hemophilia, a paradigm shift towards targeted and combination therapy, and a higher diagnosis rate on account of rising awareness, the market for hemophilia therapeutics will probably boom at a faster rate during the forecast period, with the growing disease burden of this disease. Further, this growth is complemented by significant initiatives taken by the government to eradicate hemophilia by approving the products used in treating hemophilia, whether it be pharmaceuticals or therapies. In April 2024, Pfizer Inc. stated that it received approval from the U.S. Food and Drug Administration for BEQVEZ (fidanacogene elaparvovec-dzkt) to treat adults with moderate to severe hemophilia B. It includes those who currently use FIX prophylaxis therapy, have had life-threatening hemorrhages, repeated, serious spontaneous bleeding episodes, or do not have neutralizing antibodies to AAVRh74var capsid as indicated by an FDA-approved test. BEQVEZ is a one-time treatment that might enable people with hemophilia B to make FIX themselves, as opposed to now, receiving regular intravenous infusions of FIX several times a week or month.
High Disease Burden of Hemophilia to Drive Market Growth
The global disease burden is relatively high and rising. Inheritance patterns, consanguinity, and an increasing diagnosis rate contribute to the high prevalence. Usually, a high disease burden and high demand for therapeutic products lead to higher diagnosis prevalence. As the disease burden of hemophilia increases, the demand for the hemophilia market is expected to rise. Awareness and diagnosis increase with the rise in prevalence, giving rise to the development of technologies for treatment, genetic engineering, and recombinant factor concentrates. These innovations improve patient outcomes and fuel market growth as they find widespread acceptance. Global initiatives, including the World Federation of Hemophilia and the World Health Organization (WHO), are ongoing to reduce the disease burden and create adequate awareness. For instance, if one looks at the results of the Annual Global Survey by the World Federation of Hemophilia published in October 2023, the total number of identified patients with hemophilia globally was 257,146 compared to 241,535 in 2020, thus increasing 6.46% over two years.
Advancements in Gene Therapy to Lead to Market Growth
Advancements in gene therapy are significantly driving market growth in the hemophilia market. Recent clinical trials utilizing adeno-associated viral (AAV) vectors have demonstrated stable and long-term expression of these factors, leading to significant reductions in spontaneous bleeding and the need for regular infusions. The market is further bolstered by biopharmaceutical companies' ongoing research and development efforts, which are focused on optimizing gene delivery methods and improving treatment efficacy. In June 2023, BioMarin Pharmaceutical Inc. announced that the United States Food and Drug Administration (FDA) approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A. This type of hemophilia is characterized by congenital factor VIII (FVIII) deficiency with FVIII activity less than 1 IU/dL and without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the United States. Also, ROCTAVIAN was first approved by the European Medicines Agency in August 2022.
Hemophilia A Segment to Dominate the Hemophilia Market Share
The dominance of Hemophilia A in the hemophilia treatment market can be attributed to several key factors. Hemophilia A accounts for approximately 74.16% of all hemophilia cases globally, making it the most prevalent type of genetic disorder. This significant patient population drives the demand for targeted therapies and treatments, ensuring that Hemophilia A remains the focal point for pharmaceutical companies and healthcare providers. As per the Annual Global Survey conducted by the World Federation of Hemophilia, published in October 2023, out of 257,146 identified hemophilia patients worldwide, 208,957 were suffering from hemophilia A while 42,203 were suffering from hemophilia B and the rest were suffering from unknown type. It indicates that hemophilia A is 4.9 times more prevalent than hemophilia B. Thus, the combination of high prevalence, continuous innovation in treatment, and substantial financial implications positions Hemophilia A as the leading segment in the hemophilia market.
North America to Dominate the Hemophilia Market Share
North America leads the hemophilia market due to several interrelated factors that improve treatment accessibility and innovation. The presence of established healthcare infrastructure, specialized hemophilia treatment centers, and significant financial support for research and development further bolsters market growth. Substantial government initiatives and funding have been directed toward gene therapy and other innovative treatments, improving patient outcomes and expanding treatment options. According to the Canadian Hemophilia Society, about 3,900 Canadians are affected by hemophilia A, and about 800 Canadians suffer from hemophilia B. Similarly, the data of the National Bleeding Disorders Foundation quotes that hemophilia occurs in approximately 1 in 5,617 live male births, resulting in 30,000-33,000 males with hemophilia in the United States.
Future Market Scenario (2024 - 2031F)
Favorable reimbursement and health insurance coverage for hemophilia treatment are shaping the future market growth of the hemophilia market. The growing popularity of health insurance, especially in developing countries, enables more patients to afford expensive hemophilia treatments. Reimbursement policies are expanding to include newer therapies, such as gene therapy for hemophilia treatment. In November 2023, BioMarin Pharmaceutical Inc. reached an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) regarding the reimbursement amount for ROCTAVIAN (valoctocogene roxaparvovec-rvox) for individuals with severe hemophilia A. It marks the first gene therapy for hemophilia to establish a set federal price in Germany. ROCTAVIAN provides value to patients and the German healthcare system as a one-time, single-dose therapy administered through an intravenous infusion lasting 3-4 hours, priced at USD 31,274.25 per vial.
Key Players Landscape and Outlook
The hemophilia market is primarily dominated by players such as Novo Nordisk A/S and Baxter International Inc. Market activity reported in recent years includes business agreements, collaborations, and regulatory approvals of products. The market fosters several smaller players as well, which operate through partnerships with other players to cater to a larger market.
In June 2024, 2seventy bio, Inc. announced the completion of an asset purchase agreement by Novo Nordisk A/S. Under the terms of the agreement, Novo Nordisk acquired the Hemophilia A program and the rights to 2seventy's in vivo gene editing technology outside of oncology and gene editing for autologous or allogeneic cell therapies to treat autoimmune disease. The 2seventy bio team currently involved in the program will join Novo Nordisk and continue to advance the technology.
In March 2024, ReciBioPharm AB entered into a collaboration agreement with GeneVentiv Therapeutics, Inc., a preclinical gene therapy company, to extend the development of a universal gene therapy for hemophilia based on adeno-associated virus (AAV). This therapy is reportedly the first of its kind designed to treat hemophilia patients with inhibitors. GeneVentiv's GENV-HEM (AAV8.FVa) is a single infusion, universal AAV-based gene therapy for all types of hemophilia. ReciBioPharm intends to expedite the development by leveraging its AAV manufacturing platform at its Watertown facility in Massachusetts.
All segments will be provided for all regions and countries covered
Companies mentioned above DO NOT hold any order as per market share and can be changed as per information available during research work.