Hemophilia is a rare inherited bleeding disorder in which the blood does not clot properly which leads to spontaneous bleeding as well as bleeding following injuries or surgery. Blood contains proteins called clotting factors that help to stop bleeding. People with hemophilia have low levels of either factor VIII or factor IX. The severity of hemophilia that a person has is determined by the amount of factor in the blood. Lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems. The treatment of hemophilia may involve prophylaxis, management of bleeding episodes, treatment of factor VIII inhibitors, and treatment & rehabilitation of hemophilia synovitis. Use of factor replacement products and other medications, including pain medications, is typically required.
The global hemophilia treatment market is mainly driven by rising prevalence of hemophilia, increasing investment for R&D activities to launch more effective therapies, and growing government initiatives. Rising adoption for prophylaxis treatment is also playing a pivotal role in contributing to the growth of the market globally. However, high cost of treatment is likely to hinder the market growth to a certain extent.
The global hemophilia treatment market has been analyzed from four perspectives: Disorder Type, Treatment, Therapy Type and Region.
Based on disorder type, the hemophilia treatment market has been segmented into hemophilia A, hemophilia B and others (hemophilia C, Von Willebrand Disease (VWD), etc.). The hemophilia A segment accounted for the largest share in the market owing to its higher prevalence as compared to other bleeding disorders. Among all hemophilia cases globally around 80-85% are hemophilia A, 4% are hemophilia B, and the remainder are various other clotting abnormalities.
Based on treatment, the hemophilia treatment market has been segmented into on-demand and prophylaxis. The prophylaxis segment is expected to be the fastest growing segment during the forecast period. Prophylaxis is the regular infusion of clotting factor concentrates in order to prevent bleeding in patients with moderate or mild hemophilia (who have clotting factor levels of 1% or more). Doctors believe that the risk of bleeding can be reduced if the minimum factor levels are kept around 1% by regular infusions of clotting factor concentrates. Studies have shown that children who receive prophylaxis have fewer bleeds.
Based on therapy type, the hemophilia treatment market has been segmented into immune tolerance induction (ITI) therapy, replacement therapy and gene therapy. The replacement therapy segment accounted for the largest share in the market. Hemophilia can be treated by replacing missing blood clotting factors. This is called clotting factor replacement therapy. Clotting factors are replaced by injecting (infusing) a clotting factor concentrate into the vein. Infusions of clotting factors help blood to clot normally. Rising adoption of the therapy owing to its benefits and growing R&D investment for replacement therapy can be attributed to its growth.
Based on region, the global hemophilia treatment market has been segmented into North America, Europe, Asia Pacific, and the Rest of the World (ROW). North America dominated the hemophilia treatment market, followed by Europe. The large share of North America in the global market can largely be attributed to the rising prevalence of hemophilia A & B, advanced healthcare infrastructure, presence of prominent players, adoption of better diagnostic technology, and rising investments in the R&D of hemophilia treatment.
The hemophilia treatment market is marked by presence of prominent players such Takeda Pharmaceutical Company Limited, Sanofi, Bayer AG, Pfizer Inc., Baxter, Octapharma AG, Kedrion S.p.A, Grifols, S.A., CSL and Novo Nordisk A/S. Other players operating in the market are Swedish Orphan Biovitrum AB, Genentech, Inc., BioMarin, among others.
• In Mar 2021, BioMarin Pharmaceutical Inc. announced that U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A
• In Jun 2020, CSL acquired the exclusive global license rights from uniQure to commercialize a novel late-stage gene therapy candidate for Hemophilia B patients
• In Mar 2020, Enzyre, entered into a strategic partnership with Takeda Pharmaceutical Company Limited (Takeda) to develop assays for the diagnosis and monitoring of congenital bleeding disorders
• In Oct 2018, Roche announced that the US Food and Drug Administration (FDA) has approved Hemlibra® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors
• In Aug 2018, Bayer announced that the U.S. Food and Drug Administration (FDA) approved Jivi® (BAY94-9027, antihemophilic factor [recombinant] PEGylated-aucl) for the routine prophylactic treatment of hemophilia A in previously treated adults and adolescents 12 years of age or older
• Quantitative (market size and forecasts) and qualitative insights of the market across years - historical years, current year and forecasted years
• Deep-rooted insights on headwinds, tailwinds and opportunities prevailing in the market
• Quantitative (market size and forecasts) and qualitative insights at segment and sub-segment level
• Comprehensive quantitative (market size and forecasts) and qualitative insights on geographic, regional and country level markets
• Granular insights on the competitive landscape prevailing in the market
• Competitive mapping of strategic developments
|Market Size Period||2018-2031|
|Market Segments||Asessment of Hemophilia Treatment Market By:
• Disorder Type
• Therapy Type
|Geographic Scope||• North America
◦ Rest of Europe (RoE)
• Asia Pacific
◦ Rest of Asia Pacific (RoAPAC)
• Rest of the World
◦ Latin America
◦ Middle East & Africa
|Companies Covered||Takeda Pharmaceutical Company Limited, Sanofi, Bayer AG, Pfizer Inc., Baxter, Octapharma AG, Kedrion S.p.A, Grifols, S.A., CSL and Novo Nordisk A/S, Swedish Orphan Biovitrum AB, Genentech, Inc., BioMarin, among others.|
1 RESEARCH ENVELOPE
1.1 Market Classification
1.2 Scope of the Study
1.2.1 Timeline Considered for Market Study
· Historic Years – 2018 & 2019
· Base Year – 2020
· Projected Years – 2021 to 2031
1.3 Currency Used in the Report
2 RESEARCH METHODOLOGY
2.1 Research Approach
2.2 Data Collection Methodology
2.3 Data Sources
2.3.1 Secondary Sources
2.3.2 Primary Sources
2.4 Market Crackdown Methodology
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Data Forecasting Model
2.6 Limitations and Assumptions for Study
3 HEADWINDS & TAILWINDS ASSESSMENT
4 MARKET OPPORTUNITIES ASSESSMENT
5 PREMIUM INSIGHTS
5.1 Key Market Trends
5.2 Key Players & Their Competitive Positioning (2020)
5.3 COVID-19 Impact Assessment
5.4 Pipeline Assessment
6 GLOBAL HEMOPHILIA TREATMENT MARKET - ANALYSIS & FORECAST, BY DISORDER TYPE
6.1 Hemophilia A
6.2 Hemophilia B
6.3 Others (Hemophilia C, Von Willebrand disease (VWD), Etc.)
7 GLOBAL HEMOPHILIA TREATMENT MARKET - ANALYSIS & FORECAST, BY TREATMENT
8 GLOBAL HEMOPHILIA TREATMENT MARKET - ANALYSIS & FORECAST, BY THERAPY TYPE
8.1 Immune Tolerance Induction (ITI) Therapy
8.2 Replacement Therapy
8.3 Gene Therapy
9 GLOBAL HEMOPHILIA TREATMENT MARKET - ANALYSIS & FORECAST, BY REGION
9.1 North America Hemophilia Treatment Market
9.1.1 North America Hemophilia Treatment Market, By Country
9.1.2 North America Hemophilia Treatment Market, By Disorder Type
9.1.3 North America Hemophilia Treatment Market, By Treatment
9.1.4 North America Hemophilia Treatment Market, By Therapy Type
9.2 Europe Hemophilia Treatment Market
9.2.1 Europe Hemophilia Treatment Market, By Country/Region
184.108.40.206 Rest of Europe (ROE)
9.2.2 Europe Hemophilia Treatment Market, By Disorder Type
9.2.3 Europe Hemophilia Treatment Market, By Treatment
9.2.4 Europe Hemophilia Treatment Market, By Therapy Type
9.3 Asia Pacific Hemophilia Treatment Market
9.3.1 Asia Pacific Hemophilia Treatment Market, By Country/Region
220.127.116.11 Rest of Asia Pacific (RoAPAC)
9.3.2 Asia Pacific Hemophilia Treatment Market, By Disorder Type
9.3.3 Asia Pacific Hemophilia Treatment Market, By Treatment
9.3.4 Asia Pacific Hemophilia Treatment Market, By Therapy Type
9.4 Rest of the World (RoW) Hemophilia Treatment Market
9.4.1 Rest of the World Hemophilia Treatment Market, By Region
18.104.22.168 Latin America
22.214.171.124 Middle East & Africa
9.4.2 Rest of The World Hemophilia Treatment Market, By Disorder Type
9.4.3 Rest of The World Hemophilia Treatment Market, By Treatment
9.4.4 Rest of The World Hemophilia Treatment Market, By Therapy Type
10 COMPANY PROFILES
10.1 Takeda Pharmaceutical Company Limited
10.1.1 Business Overview
10.1.2 Financial Metrics*
10.1.3 Product Portfolio
10.1.4 Key Developments
10.3 Bayer AG
10.4 Pfizer Inc.
10.6 Octapharma AG
10.7 Kedrion S.p.A
10.8 Grifols, S.A.
10.10 Novo Nordisk A/S
*Insights on financial metrics is subject to availability of information in the public domain
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