PharmaPoint: Hemophilia A and B Recombinant Factor Replacement Therapy - Global Drug Forecast and Market Analysis to 2024
Congenital hemophilia A and B are bleeding disorders that are caused by genetic X-linked deficiencies in the blood clotting Factors VIII and IX, respectively. Hemophilia manifests as mild, moderate, or severe inability to form clots, depending upon the endogenous levels of circulating factor in an individual patient. The mainstay of treatment for hemophilia is to replace the missing FVIII or FIX, produced using plasma-derived or recombinant methods. Some patients develop inhibitors to FVIII or FIX treatment and need a bypassing agent, such as FVIIa, to resolve bleeds. Patients of hemophilia A or B often require lifelong treatment to prevent and control bleeding episodes, and this therefore represents a lucrative sales opportunity.
GlobalData estimates the 2014 sales for recombinant hemophilia A and B markets at approximately $5.38 billion across the 7MM+A covered in this report. The market is expected to reach $6.25 billion in 2024 at a CAGR of 1.52%. This growth will be mainly driven by an increasing use of routine prophylaxis and a greater use of recombinant products in place of plasma-derived therapies.
The hemophilia recombinant treatment pipeline is strong, and crowded with multiple long-acting recombinant clotting factors; however, GlobalData expects a lack of initiative for hemophilia A patients to switch to long-acting rFVIII, and a limited scope for premium pricing for new agents, restraining the market growth. Overall, Baxalta is expected to remain as the market leader in the hemophilia space throughout the forecast period, dominating the market with its current gold standard therapy for hemophilia A, Advate, supported by the launch its of pegylated rFVIII, Adynovate.
Key Questions Answered
- What will be the impact of the new long-acting rFVIII and rFIX products in the hemophilia market sales? Led by Biogens Eloctate and Alprolix, moderate and high rates of adoption of the long-acting rFVIII and rFIX therapies, respectively, are expected.
- With the growing importance of long-acting Factors, what are the strategies of the companies that have short-acting assets to remain competitive? What are the other approaches that new entrants employ?
- Prevention of inhibitor formation remains as a major unmet need in the hemophilia space. What are the other unmet needs and opportunities in the hemophilia market?
- One of the main drivers of the expansion of the hemophilia recombinant market will be the increasing use of routine prophylaxis in pediatric and adult hemophilia A and B patients. Prophylactic regimens have demonstrated improvements in the prevention of bleeding episodes and joint damage. In the 7MM, routine prophylaxis is the standard of care for severe hemophilia patients.
- The high cost of rFVIII and rFIX therapies is a significant barrier to market growth. Scope for premium pricing for new entrants in the hemophilia A and B market is limited. GlobalData believes that Biogen has set an upper limit to the price of long-acting rFVIII and rFIX products with its competitive pricing strategy for Eloctate and Alprolix.
- Companies are focusing on the development of therapies that can further reduce the dosing frequency required for prophylaxis. Strategies to prevent inhibitor formation are also sought. Early-stage pipeline products that employ monoclonal antibodies, RNA interference, and gene therapies appear to be highly promising, and represent a huge threat, if they are approved, for the existing treatment options.
- Overview of hemophilia, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
- Annualized hemophilia recombinant therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in six patient segments (including on-demand, prophylactic, and inhibitor), forecast from 2014 to 2024.
- Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the hemophilia recombinant therapeutics market.
- Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for different lines of therapy. The most promising candidate in Phase III development is profiled.
- Analysis of the current and future market competition in the global hemophilia recombinant therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.
Reasons to buy
The report will enable you to -
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the global hemophilia recombinant therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global hemophilia recombinant therapeutics market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.