PharmaPoint: Hemophilia A and B – Global Drug Forecast and Market Analysis to 2026
All the vital news, analysis, and commentary curated by our industry experts.
Hemophilia A and B are rare recessive X-linked genetic deficiencies in the blood clotting factors VIII and IX, respectively, and are characterized by the failure of blood to form normal clots after damage to veins and tissue. In the 7MM, patients with severe forms of the disease are increasingly treated on a prophylactic basis rather than on demand after bleeds. The frequent prophylactic infusions of rFVIII or rFIX begin from the first one or two years of life, often continuing through adulthood. However, the burden on patients and their families of maintaining the prophylactic treatment schedule is high.
Traditionally, the hemophilia market has been dominated by short-acting rFVIII or FIX concentrates. However, in recent years the hemophilia market has become increasingly competitive due to the approval of extended half-life products that can reduce the burden associated with prophylaxis. These improvements have mainly benefited hemophilia B patients, while a substantial unmet need remains for hemophilia A patients and patients with inhibitors who do not respond to standard treatments.
The hemophilia pipeline activity is strong and includes two candidates, Roche’s emicizumab (ACE-910) and Alnylam’s fitusiran, which do not exploit a replacement strategy but target different effectors of the coagulation cascade and are expected to cause a paradigm shift in the treatment of the disease.
Key Questions Answered
What are the key drivers behind the increasing uptake and market penetration of the long-acting factor concentrates in hemophilia A and B, since their approval in 2014, and what is the trend in the use of long-acting factors in the forecast period?
The hemophilia market is characterized by a number of unmet needs. What are the main unmet needs in this market? Will the pipeline drugs under development fulfil these unmet needs?
What impact will the approval of Roche’s emicizumab have on the hemophilia market? What will the drug’s peak sales be, and why?
Overview of hemophilia, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
Annualized hemophilia therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in nine patient segments and two treatment strategies (episodic and prophylactic treatment), forecast from 2016 to 2026.
Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications for the hemophilia therapeutics market.
Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for hemophilia. The most promising candidate in Phase III development is profiled.
Analysis of the current and future market competition in the global hemophilia 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 market.
Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global hemophilia 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.
Table of Contents
Table of Contents
1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Executive Summary
2.1 Sales for Hemophilia A and B Recombinant Therapies by Region, 2016–2026
2.2 Long-Acting rFVIIIs Have Not Lived Up to Expectations
2.3 Inhibitor Segment to Devalue Post-Alternative Coagulation Promoter Launch
2.4 Roche’s Emicizumab to Radically Impact Hemophilia A and Inhibitor Segments
2.5 Treatment Cost to Remain a Major Unmet Need
2.6 New Comers to Take Over Market Space From Established Companies
2.7 What Do Physicians Think?
3.2 Related Reports
3.3 Upcoming Related Reports
4 Disease Overview
4.1 Etiology and Pathophysiology
4.3.1 Hemophilia A and B
4.4 Prognosis and Quality of Life
5.1 Disease Background
5.2 Risk Factors and Comorbidities
5.3 Global and Historical Trends
5.4 Forecast Methodology
5.4.2 Forecast Assumptions and Methods
5.4.3 Diagnosed Prevalent Cases
5.4.4 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity
5.4.5 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Inhibitors
5.5 Epidemiological Forecast for Hemophilia A (2016–2026)
5.5.1 Diagnosed Prevalent Cases of Hemophilia A
5.5.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia A
5.5.3 Sex-Specific Diagnosed Prevalent Cases of Hemophilia A
5.5.4 Diagnosed Prevalent Cases of Hemophilia A by Severity
5.5.5 Diagnosed Prevalent Cases of Hemophilia A with Inhibitors
5.6 Epidemiological Forecast for Hemophilia B (2016–2026)
5.6.1 Diagnosed Prevalent Cases of Hemophilia B
5.6.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia B
5.6.3 Sex -Specific Diagnosed Prevalent Cases of Hemophilia B
5.6.4 Diagnosed Prevalent Cases of Hemophilia B by Severity
5.6.5 Diagnosed Prevalent Cases of Hemophilia B with Inhibitors
5.7 Epidemiological Forecast for Hemophilia A and Hemophilia B (2016–2026)
5.7.1 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B
5.7.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B
5.7.3 Sex -Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B
5.7.4 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity
5.7.5 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors
5.8.1 Epidemiological Forecast Insight
5.8.2 Limitations of the Analysis
5.8.3 Strengths of the Analysis
6 Disease Management
6.1 Treatment Overview
6.1.1 Treatment Guidelines
6.1.2 Clinical Practice
6.2.1 Clinical Practice
6.3.1 Clinical Practice
6.4.1 Clinical Practice
6.5.1 Clinical Practice
6.6.1 Clinical Practice
6.7.1 Clinical Practice
6.8.1 Clinical Practice
7 Competitive Assessment
7.2 Product Profiles – Hemophilia A
7.2.1 Short Acting rFVIII
7.2.2 Long Acting rFVIII
7.2.3 Plasma-Derived FVIII Concentrates
7.2.4 Desmopressin Acetate
7.3 Product Profiles – Hemophilia B
7.3.1 Short Acting rFIX
7.3.2 Long Acting FIX
7.3.3 Plasma-Derived FIX Concentrates
7.4 Product Profiles – Hemophilia A and B with Inhibitors
7.4.1 NovoSeven RT (Eptacog Alfa)
7.4.2 Feiba NF (Activated Prothrombin Complex Concentrate)
8 Unmet Needs and Opportunities
8.2 Reduction in Risk of Inhibitor Development in Previously Untreated Patients
8.2.1 Unmet Need
8.2.2 Gap Analysis
8.3 More Effective Treatments for Patients with Inhibitors
8.3.1 Unmet Need
8.3.2 Gap Analysis
8.4 Longer-Lasting Agents and More Convenient Administration Routes
8.4.1 Unmet Need
8.4.2 Gap Analysis
8.5 Treatments to Cure the Disease
8.5.1 Unmet Need
8.5.2 Gap Analysis
8.6 Decreasing the Costs Associated with Prophylaxis
8.6.1 Unmet Need
8.6.2 Gap Analysis
8.7 Individualized Prophylaxis
8.7.1 Unmet Need
8.7.2 Gap Analysis
9 Pipeline Assessment
9.2 Late-Stage Development Candidates – Hemophilia A
9.2.1 BAY 94-9027 (Damoctocog Alfa Pegol)
9.2.2 N8-GP (Turoctocog Alfa Pegol)
9.2.3 Emicizumab (ACE-910, RG-6013)
9.2.4 Fitusiran (ALN-AT3, SAR-439774)
9.3 Late-Stage Development Candidates – Hemophilia B
9.4 Late-Stage Development Candidates – Hemophilia A and B Patients with Inhibitors
9.4.1 LR-769 (rhFVIIa)
9.4.2 Obizur (Susoctocog Alfa)
9.4.3 CSL-689 (rVIIa-FP)
9.4.4 BAX-817 (rFVIIa BI)
9.5 Promising Approaches in Early-Stage Development
9.5.1 Alternative Coagulation Promoters – Tissue Factor Pathway Inhibitors
9.5.2 Gene Therapies
9.5.3 Replacement Strategies
9.5.4 Strategies to Prevent and Manage Inhibitors
9.5.5 Other Drugs in Development
10 Current and Future Players
10.2 Trends in Corporate Strategy
10.3 Company Profiles
10.3.3 CSL Behring
10.3.4 Novo Nordisk
11 Market Outlook
11.1 Global Markets
11.1.2 Drivers and Barriers – Global Market
11.2.2 Key Events
11.2.3 Drivers and Barriers
11.3.2 Key Events
11.3.3 Drivers and Barriers – 5EU
11.3.4 Drivers and Barriers – France
11.3.5 Drivers and Barriers – Germany
11.3.6 Drivers and Barriers – Italy
11.3.7 Drivers and Barriers – Spain
11.3.8 Drivers and Barriers – UK
11.4.2 Key Events
11.4.3 Drivers and Barriers
12.4 Forecasting Methodology
12.4.1 Diagnosed Hemophilia Patients
12.4.2 Percent Drug-Treated Patients
12.4.3 Drugs Included in Each Therapeutic Class
12.4.4 Launch and Patent Expiry Dates
12.4.5 General Pricing Assumptions
12.4.6 Individual Drug Assumptions
12.4.7 Pricing of Pipeline Agents
12.5 Primary Research – KOLs Interviewed for this Report
12.6 Primary Research – Prescriber Survey
12.7 Real-World Data
12.8 About the Authors
12.8.3 Therapy Area Director
12.8.6 Global Director of Therapy Analysis and Epidemiology
12.8.7 Global Head and EVP of Healthcare Operations and Strategy
12.9 About GlobalData
12.10 Contact Us
Frequently asked questions
Tel +44 20 7947 2745
Every customer’s requirement is unique. With over 220,000 construction projects tracked, we can create a tailored dataset for you based on the types of projects you are looking for. Please get in touch with your specific requirements and we can send you a quote.
Business Intelligence & Marketing Manager, SAL Heavy Lift
“The GlobalData platform is our go-to tool for intelligence services. GlobalData provides an easy way to access comprehensive intelligence data around multiple sectors, which essentially makes it a one-for-all intelligence platform, for tendering and approaching customers.
GlobalData is very customer orientated, with a high degree of personalised services, which benefits everyday use. The highly detailed project intelligence and forecast reports can be utilised across multiple departments and workflow scopes, from operational to strategic level, and often support strategic decisions. GlobalData Analytics and visualisation solutions has contributed positively when preparing management presentations and strategic papers.”
Market Analyst & Management, Liebherr-Werk
“COVID-19 has caused significant interference to our business and the COVID-19 intelligence from GlobalData has helped us reach better decisions around strategy. These two highlights have helped enormously to understand the projections into the future concerning our business units, we also utilise the project database to source new projects for Liebherr-Werk to use as an additional source to pitch for new business.”
Head of Key Accounts, Saab AB
Your daily news has saved me a lot of time and keeps me up-to-date with what is happening in the market, I like that you almost always have a link to the source origin. We also use your market data in our Strategic Business Process to support our business decisions. By having everything in one place on the Intelligence Center it has saved me a lot of time versus looking on different sources, the alert function also helps with this.
Marketing Intelligence Manager, Portugal Foods
Having used several other market research companies, I find that GlobalData manages to provide that ‘difficult-to-get’ market data that others can’t, as well as very diverse and complete consumer surveys.
Head of Customer Insight and Research, Standard Chartered
Our experience with GlobalData has been very good, from the platform itself to the people. I find that the analysts and the account team have a high level of customer focus and responsiveness and therefore I can always rely on. The platform is more holistic than other providers. It is convenient and almost like a one stop shop. The pricing suite is highly competitive and value for our organisation.
I like reports that inform new segments such as the analysis on generation Z, millennials, the impact of COVID 19 to our banking customers and their new channel habits. Secondly the specialist insight on affluent sector significantly increases our understanding about this group of customers. The combination of those give us depth and breadth of the evolving market.
I’m in the business of answering and helping people make decisions so with the intelligence center I can do that, effectively and efficiently. I can share quickly key insights that answer and satisfy our country stakeholders by giving them many quality studies and primary research about competitive landscape beyond the outlook of our bank. It helps me be seen as an advisory partner and that makes a big difference. A big benefit of our subscription is that no one holds the whole data and because it allows so many people, so many different parts of our organisation have access, it enables all teams to have the same level of knowledge and decision support.
Head of Consumer Sensory Insights, Givaudan
“I know that I can always rely on Globaldata’s work when I’m searching for the right consumer and market insights. I use Globaldata insights to understand the changing market & consumer landscape and help create better taste & wellbeing solutions for our customers in food, beverage and healthcare industries.
Globaldata has the right data and the reports are of very high quality compared to your competitors. Globaldata not only has overall market sizes & consumer insights on food & beverages but also provides insights at the ingredient & flavour level. That is key for B2B companies like Givaudan. This way we understand our customers’ business and also gain insight to our unique industry”
Senior Account Manager, TSYS
GlobalData provides a great range of information and reports on various sectors that is highly relevant, timely, easy to access and utilise. The reports and data dashboards help engagement with clients; they provide valuable industry and market insights that can enrich client conversations and can help in the shaping of value propositions. Moreover, using GlobalData products has helped increase my knowledge of the finance sector, the players within it, and the general threats and opportunities.
I find the consumer surveys that are carried out to be extremely beneficial and not something I have seen anywhere else. They provided an insightful view of why and which consumers take (or don’t) particular financial products. This can help shape conversations with clients to ensure they make the right strategic decisions for their business.
One of the challenges I have found is that data in the payments space is often piecemeal. With GD all of the data I need is in one place, but it also comes with additional market reports that provide useful extra context and information. Having the ability to set-up alerts on relevant movements in the industry, be it competitors or customers, and have them emailed directly to me, ensures I get early sight of industry activity and don’t have to search for news.