Hemophilia A and B are rare, genetic, X-linked deficiencies in the blood clotting factors VIII (FVIII) and IX (FIX), respectively. Patients can be categorized as mild, moderate or severe depending on the percentage of circulating FVIII or FIX in their blood. Patients can be treated either on-demand or prophylactically using plasma-derived or recombinant replacement factors, the current standard of care.
Although these therapies are effective, they are associated with a great treatment burden. There is a significant unmet need for new therapies that can reduce the great treatment burden associated with intravenous administration, the frequency of prophylactic infusions, and the risk of developing neutralizing antibodies, or inhibitors, against replacement factors. Overall, the late-stage pipeline indicates an increase in the development of alternative coagulation promoters (ACPs) and gene therapies to further target these areas of significant unmet need. During the forecast window, Roche entered the market with the first monoclonal antibody targeting hemophilia A patients both with and without inhibitors, which addresses the unmet needs of frequency and route of administration. Sanofi are also to exploring a non-replacement approach and will enter the market with the ribonucleic acid (RNA) therapeutic, Fitusiran (previously Alnylam's), further ensuring cross-segment efficacy in patients both with and without inhibitors. The approval of these new products has begun to have a drastic effect on the hemophilia A and B treatment landscapes which is expected to continue through the forecast window. During this forecast window, several pharmaceutical companies, including BioMarin, uniQure and Pfizer/Roche (Spark Therapeutics), will also be launching new gene therapies that aim to cure, or at least reduce the severity of, hemophilia A and B and diminish inhibitors. If approved, gene therapies will pose a threat to existing hemophilia therapies.
GlobalData projects the global hemophilia A and B marketplace to experience a significant growth during the forecast period. GlobalData valued the hemophilia A and B market at $6.9B in 2018 in the 8MM and expects the market to increase to $9.3B in 2028 at a Compound Annual Growth Rate (CAGR) of 2.9%. This growth will be driven primarily by the increasing prophylactic treatment rates among patients, the continued use of recombinant products and the uptake of non-replacement therapies in the 8MM. However, cost constraints in the 8MM are expected to limit the premium pricing opportunities for new products such as late-to-market long-acting factor concentrates and new alternative coagulation promoters, thereby limiting the size of the hemophilia market.
This model covers the market forecast for marketed and late-stage pipeline hemophilia A and B therapeutics. The base year of this model is 2018, and the forecast period is 2018-2028.
Key Questions Answered
- Which pipeline agents - replacement factors, alternative coagulation promoters and gene therapies - are the most promising and expected to launch in the 8MM?
- What are the forecasted sales of these agents and what will be their impact in the hemophilia A and B market?
- What are the main unmet needs in hemophilia A and B, and which pipeline drugs will fulfil these needs, and to what extent?
- What are the current research and development (R&D) strategies being explored and how can developers incorporate these methods into their business strategy?
- Key Opinions Leader (KOL) and payer insights across the 8MM, relating to the current treatment options and opinions on the late and early stage pipeline agents.
- The hemophilia A & B market is expected to grow at a CAGR of 2.9% until 2028, reaching a global value of $9.3B.
- GlobalData forecasts a marginal increase in prevalence rates across 8MM with AGR of 0.1% due to increased in prevalence rates across the US, Spain and UK.
- Gene therapies will emerge as novel treatments for hemophilia A & B during the forecast period. Particularly, BioMarin's first-to-market gene therapy, ValRox is likely to perform exceptionally well. Additionally, Roche's Hemlibra is anticipated to achieve blockbuster status by the end of the forecast period.
- Rising life expectancy for hemophilia patients leads to an increase in the size of the treated population.
- Expected launches of expensive new drugs such as gene therapies, continued preference for prophylactic regimens, continued patient evolution from plasma-based to recombinant therapies are other significant drivers of market.
- Limited scope for price premiums of late-to-market replacement products, high uptake of lower-priced drugs, rise in cost-consciousness and imposing market access barriers for the entry of expensive gene therapies are expected to affect market growth negatively.
- Level of unmet needs in hemophilia patients is high but is expected to decrease due to advances in treatments and the introduction of novel therapies.
- There are major market opportunities for the development of novel therapies, cheaper therapies for prophylaxis treatment, more effective treatments for hemophilia B patients with inhibitors and more therapies with convenient administration routes.
Overview of hemophilia A and B, including -
- Disease overview - etiology, pathophysiology, symptoms, prognosis and quality of life
- Disease management - diagnosis and treatment
Patient segmentation -
- Mild, Moderate, and Severe
- Hemophilia A and B with and without inhibitors
- Males and Females
- All ages, Pediatrics and Adults
Annualized hemophilia A and B therapeutics market revenue and treatment usage patterns -
- Patient shares and number of patients treated for marketed and pipeline products
- Duration of and, daily and annual cost of therapies per patient
- Product sales by usage and total sales of marketed and pipeline products
Key topics covered include -
- Competitive assessment of marketed and pipeline products including SWOTs and, clinical and commercial positioning.
- Unmet needs and opportunities including current and future level of attainment and gap analysis
- Pipeline assessment - analysis of commercial and clinical attributes of promising drugs in clinical development and comprehensive analysis of promising approaches and emerging trends in early stage development
- Analysis of the current and future players for the global hemophilia A and B market, including strength of marketed and pipeline products and trends in corporate strategy, profiles of leading companies in the hemophilia market.
- Market outlook of the 8MM including forecast, key events and, market drivers and barriers.
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 A and B 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 analyzing 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.