Hemophilia B Treatment Market

Hemophilia B Treatment Market Outlook (2024 to 2034)

The global hemophilia B treatment was valued at US$ 3,987.0 million in 2023 and is expected to expand at a noteworthy CAGR of 7.6% to end up at US$ 8,965.8 million by 2034.

Hemophilia B is a rare bleeding disorder characterized by prolonged or spontaneous blood loss or hemorrhage because of the deficiency of coagulation factor. It is also called as the Christmas disease. Hemostasis is a physiological process of the human body and may be narrated as activity that halts the oozing out process of blood from a blood vessel. The coagulation cascade represents a complex of processes comprising the haemostasis that includes local vasoconstriction and platelet plug formation. More simply put, clotting factors are substances in blood, which assist in the creation of blood clots. There are clotting factors that act like proteins to slow down the flow of blood streams in a damaged blood vessel. All the 13 clotting factors are present as plasma in the blood without carrying their active coagulation characteristics, and are activated by trauma and injuries effects. If either of the clotting factors are missing or not functioning well coagulation cascade does not ‘ignite’ the process and this results in bleeding continuously.

Hemophilia is a group of bleeding disorders with three types namely, hemophilia A, B and C. A is due to the absence of clotting factor VIII and it is the most common bleeding disorder. Whereas hemophilia B results from the absence of factors IX and hemophilia C results due to shortage of factor XI. Factor XI is a very rare factor, largely evident in populations where it is estimated to be 1 in 1 million.

Report Attributes	Details
Hemophilia B Treatment Market Size (2023A)	US$ 3,987.0 Million
Estimated Market Value (2024E)	US$ 4,291.8 Million
Forecasted Market Value (2034F)	US$ 8,965.8 Million
Global Market Growth Rate (2024 to 2034)	7.6% CAGR
East Asia Market Share (2024)	14.2%
China Market Growth Rate (2024 to 2034)	8.8% CAGR
Key Companies Profiled	uniQure (CSL Behring); Pfizer; Hoffmann-La Roche Ltd; Octapharma; CSL Behring; Sanofi; Bayer AG; Baxter; Swedish Orphan Biovitrum AB; Grifols International S.A; Kedrion Biopharma, Inc.; Biomarin Pharmaceutical, Inc.; Chugai Pharmaceutical; Biogen, Inc.; Other Prominent Players

Historic Analysis (2019 to 2023) and Future (2024 to 2034) Pathway Analysis for the Hemophilia B Treatment Market

In the 1970s and 1990s, respectively, plasma-derived concentrates marked significant advances in hemophilia care and recombinant clotting factor concentrates. This has enabled not only the treatment of episodic bleeding but also permitted prophylactic regimens. As years passed, bioengineered factor VIII and IX molecules with extended half-life and non-factor treatments consisting of the bispecific antibody emicizumab replaced the traditional clotting factor replacement therapy, which marked new landmarks in the prophylaxis and treatment of Christmas disease. The lyophilized-factor concentrates (1970) had better outcomes in the management of joint bleeds and adverse effects of treatment, and enhanced the quality of life of a person. Many patients started receiving home infusions therapies. The life expectancy increased to almost 60 years of age. These concentrates were prepared from a large pool of blood donors, which led to several complications including exposure of patients to hepatitis A and hepatitis B. More severe complications of viral contamination in the 1980s included the emergence of HIV and AIDS, which killed many hemophiliacs. About 80% of the patient pool having coagulation disorder were exposed to HIV. Viricidal treatment of plasma-derived concentrate was instituted in 1992 to overcome these viruses. At about the same time, the cloning of the FVIII gene in 1984 followed the development of recombinant factor VIII (rFVIII). In 1995, preventive prophylaxis had already become a modality in the treatment of hemophilia in children. Moreover, the elimination of albumin from the rFVIII concentrate manufacturing process removed viral exposure in the 2000s.

Recent treatment standards in the management of hemophilia B with excessive bleeding risk include I.V. infusion of replacement FIX (factor IX) concentrate frequently, gene therapy (etranacogene dezaparvovec) with recombinant adeno-associated viral (AAV) vector serotype 5 (rAAV5) capsid have altered standard of care. These advances in the hemophilia B treatment market boosted historic market growth.

• Short Term (2024 to 2028): The market will see steady growth in the short term due to the introduction of various therapies in the management of hemophilia disorder i.e. factor replacement therapies and advanced gene therapies. The focus on the commercialization of drugs and the approval process that shows noticeable results even on single-dose administration will drive the market in 2024 to 2028. FDA and EMA are encouraging manufacturers to develop novel therapeutics in terms of personalized treatment that will mark significant growth in the coming years.
• Medium Term (2028 to 2030): Advancement in therapies will show better patient outcomes on disease management. Management of pediatric patients (most crucial to treat) with factor replacement therapies will show decreased bleeding occurrences. As exhaustive research and development activities rise in the field of disease management, partnership and collaboration among new entrants and global players will further enhance market growth in the medium term. Partnership and collaboration among big pharma companies to develop various treatment options and market emergence will drive the hemophilia B treatment market.
• Long Term (2030 to 2034): This is positive along the line of gene therapy in hemophilia B since it is likely to be the mainstream mode of treatment in the year 2030. With better safety data and long-term efficacy, patients may enjoy one-time curative remedies that will keep them from having to undergo frequent treatments. This would have an impact on patient’s quality of life and healthcare cost. In this regard, the improvement of organized healthcare in the developing world and advanced international cooperation is to provide an increase in accessibility to treatment in the analyzed period. The governments in these regions shall be more active in promoting the increased availability of these affordable gene therapies and other innovative treatment options. Thus, as the treatment becomes available more frequently, it will create a large number of customers for it, especially in the Asian-Pacific and Latin-American countries.

On the back of the aforementioned facts, the Hemophilia B Treatment market is anticipated to grow at a CAGR of 7.6% during the forecast period from 2024 to 2034, according to the Fact.MR, a market research and competitive intelligence provider.

Market Dynamics

What are the Factors Propelling Global Sales of Hemophilia B Treatment?

“Improvements in Therapeutics to Provide Better Patient Outcomes”

Studies suggest that the advancement in gene therapy is one of the factors that have been steadily increasing the global sale of hemophilia B treatment. It means the inventions of gene therapy are helpful to increase, or even rise to normal factor IX levels and significantly decrease or remove several factor replacement treatments in patients. Being new and better ways of managing the disease, this therapeutic innovation is a gain in the prospects of patients’ quality of life and disease management. Novel treatments administered to patients once and whose results may last for many years have led to increased demand for gene therapies in both developed and other markets. Since the future arrivals of advanced gene therapies into the market are going to be in clinical trials or under review for commercialization, the hemophilia B treatment market is anticipated to undergo significant changes worldwide and accelerate global market growth.

“Early Diagnosis and Diseases Awareness to Propel Market Growth”

Another key driver that fuels the global sales of Hemophilia B treatment is growing awareness and early diagnosis of the disease. Global awareness campaigns and initiatives for educating healthcare providers, patients, and their families about hemophilia that ensured diagnosis and subsequent treatment of the condition were carried out much earlier than a decade or so in the past. Greater awareness has helped overcome stigma and misconceptions of the disease, especially in less-developed countries with minimal healthcare access. The early diagnosis now enables patients to seek immediate treatment before complications set in-such as injuries to their joints or deadly bleeding episodes. Awareness and access to treatments were spread among the population through governments and NGOs that sometimes have representation in countries where hemophilia care is customarily insufficient. Nowadays patients are diagnosed at early stages, contributing to an increased demand for hemophilia B therapy globally.

What is the Challenges Encountered by Manufacturers of Hemophilia B Treatment?

“High Cost of Therapies and Regulatory Hurdles to Hamper Market Growth”

One of the biggest challenges faced by companies manufacturing treatments for hemophilia B is the extremely high cost of production, especially regarding advanced therapies like gene therapy and extended half-life factor IX products. Biologics and gene therapies require huge investments in research and development, manufacturing processes, and quality control. In addition, gene therapy production requires viral vector manufacturing, which requires specialized facilities and a regulatory oversight system. The above factors that drives up the cost of goods and make the scalability and affordability of these treatments challenging. The costs of production are further increased by the fact that most hemophilia treatments need cold-chain logistics-manufacturing, storage, and shipping at low temperatures to maintain the integrity of the molecules and preserve their efficacy. These logistical requirements add to the overall cost burden for manufacturers and healthcare systems and could further limit access to these life-saving therapies.

The regulatory environment is continuously changing, and the approval by the different health authorities could be a time-consuming and complicated affair. In various countries, most of these regulatory agencies usually ask for different requirements concerning clinical trials and safety data, together with post-marketing surveillance. All these may involve much time and money on the part of the manufacturers while fulfilling these diverse requirements, especially in the case of multinational companies who seek approval in several regions. Besides that, the very safety and long-term efficacy of gene therapies remain a concern for regulators, hence an additional layer of scrutiny. The companies have to be able to prove that their therapies offer obvious benefits over current treatments and that risks, including possible immune responses or off-target effects from gene therapy, are manageable. All these challenges in the way of regulation therefore act to dent the ability of new therapies to be commercialized into the marketplace and reduce the speed at which patients can gain access to potentially life-changing treatments.

Country-wise Insights

Why is the Adoption of Hemophilia B Treatment High in the United States?

“Extensive Research & Development to Project Growth Northwards”

The market in the United States is estimated at US$ 1,376.5 million in 2024 and is projected to expand at a CAGR of 7.8% through 2034. North America, especially the U.S., dominates the share in the hemophilia B treatment market.

The North America region already has established healthcare infrastructure, advanced therapies, and a high adoption rate for recombinant factor IX products and gene therapy. Besides that, the proactive attitude of the region toward the management of rare diseases, highly spent health care, and access to innovative treatments by patients continue to keep North America in the lead in the global market.

Why is Europe a Lucrative Market for Hemophilia B Treatment Manufacturers?

“Government Initiatives Promoting Domestic Drug Production”

Europe comes second in this regard, as it gives a strong impetus to innovative treatments and powerful care networks; among the well-developed countries in this regard are Germany and the UK. Nevertheless, the lead belongs to North America, since it is considered the region with the highest level of research and development, plus access to high-cost therapies.

Category-wise Insights

Which Treatment type is Widely Preferred for the Management of Hemophilia B Treatment?

“High Demand for Recombinant Products of Factor IX to Reduce Infections”

Demand for Recombinant Factor IX Concentrates leads the treatment type segment with a 45.6% market share in 2024 and is projected to grow at a CAGR of 7.5% from 2024 to 2034. Recombinant products of Factor IX are the gold standard in the management of hemophilia B. They are genetically engineered to be free from human plasma, hence reducing the risk of transmission of blood-borne pathogens like HIV and hepatitis. They offer higher safety profiles with fewer issues about immunogenicity compared to plasma-derived products. Gene therapy, though at its tender stage, has gained momentum with promising results pertaining to single-dose treatments for long-lasting effects. Since it is still in the nascent stages and has a highly expensive treatment value, it accounts for a smaller percentage. However, a trend reversal is envisioned in this arena. The most common mode of treatment for hemophilia B is by intravenous administration because this allows direct entry of clotting factors into the bloodstream for immediate action.

Why Do Adult Patient Demographics Account Extensive Use of Hemophilia B Treatment?

Adult patient demographics are estimated to account for a 52.5% share of the global market in 2024. Adults are more aware of the disease and health facilities. They usually require regular prophylaxis, especially among active patients or those who are at risk for joint damage due to repeated bleedings. Prophylactic therapies administered among adults further drive their dominance in the market. While pediatric treatment is indispensable, most especially for early intervention and prophylaxis to prevent joint damage, adults usually drive the larger share of the market because of the chronic and lifelong nature of the condition.

Which Type of Hemophilia B Requires Immediate Attention?

Severe hemophilia B dominates the global market with 49.2% market share in 2024, with less than 1% of normal factor IX activity, constituting the majority of patients under regular and intensive treatment. Most of the severe cases require a prophylactic infusion of Factor IX to prevent spontaneous bleeding and its complications. A higher frequency of treatment in severe cases keeps the demand for its replacement therapies higher, hence dominant. In cases of moderate or mild hemophilia, only on-demand treatment of bleeding episodes may be required which would therefore imply lower volumes of overall treatments compared with cases involving severe ones.

Which Distribution Channel is dominating the Global Hemophilia B Treatment Market?

The hospital segment is dominating the market with a 60.1% share in 2024. In the settings of a hospital, there is immediate access to specialized care, emergency services, and administration of factors, which are very critical in managing the condition. This is mainly available in hospitals, specifically for new diagnoses, management of acute bleeding, surgeries, and complications that require medical supervision.

Competitive Landscape

Key players in the hemophilia B treatment market are uniQure (CSL Behring), Pfizer, Hoffmann-La Roche Ltd, Octapharma, Sanofi, Bayer AG, Baxter, Swedish Orphan Biovitrum AB, Grifols International S.A, Kedrion Biopharma, Inc., Biomarin Pharmaceutical, Inc., Chugai Pharmaceutical, and Biogen, Inc.

Companies devote a significant portion of their revenue to R&D to resolve problems and offer creative solutions to draw in new customers and stay competitive. Companies are developing customized offerings for issues that meet industry-specific needs to compete with other players.

• In July 2024, Pfizer received FDA approval for its hemophilia B gene therapy i.e. BEQVEZ (fidanocogene elaparvovec).
• In November 2022, HEMGENIX (etranacogene dezaparvovec) uniQure’s second internally developed gene therapy drug received FDA approval for its world’s first hemophilia B treatment.

Fact.MR has provided detailed information about the price points of key manufacturers of hemophilia B treatments positioned across regions, sales growth, production capacity, and speculative technological expansion, in the recently published report.

Segmentation of Hemophilia B Treatment Industry Research

• By Treatment Type :

  • Recombinant Factor IX Concentrates
  • Plasma-derived Factor IX Concentrates
  • Gene Therapy
  • Extended Half-Life Factor IX

• By Patient Demographics :

  • Pediatrics
  • Adults

• By Severity of Hemophilia B :

  • Mild Hemophilia B
  • Moderate Hemophilia B
  • Severe Hemophilia B

• By Distribution Channel :

  • Hospitals
  • Specialty Clinics
  • Online Pharmacies

• By Region :

  • North America
  • Latin America
  • Western Europe
  • Eastern Europe
  • East Asia
  • South Asia & Pacific
  • Middle East & Africa