Hemophilia B Market

DelveInsight’s ‘Hemophilia B - Market Insights, Epidemiology, and Market Forecast—2030’ report delivers an in-depth understanding of the Hemophilia B, historical and forecasted epidemiology as well as the Hemophilia B market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.


The Hemophilia B market report provides current treatment practices, emerging drugs, Hemophilia B market share of the individual therapies, current and forecasted Hemophilia B market size from 2018 to 2030 segmented by seven major markets. The Report also covers current Hemophilia B treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2018–2030

Hemophilia B Disease Understanding and Treatment Algorithm

Hemophilia B Overview

Hemophilia is defined as an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. It is caused by a gene defect that decides how factors VIII, IX, or XI are produced by the body. One of the Hemophilia type is Hemophilia B, also called factor IX (FIX) deficiency or Christmas disease, is a genetic disorder caused by missing or defective factor IX, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a change in a gene. Clotting factors are specialized proteins needed for blood clotting, the process by which blood seals a wound to stop bleeding and promote healing. The two main symptoms are bleeding for longer than normal, and bruising easily. With this disease, even a small bump to the head can be serious. Hemophilia B can range from mild to moderate to severe. Individuals with mild hemophilia have factor IX levels between 5 and 40% of normal; those with moderate hemophilia have factor levels from 1 to 5% of normal; and individuals with severe hemophilia have factor levels <1% of normal.


Hemophilia B Diagnosis

Diagnosis of hemophilia B is made by investigating the patient’s personal history of bleeding, the patient’s family history of bleeding and inheritance, and laboratory testing. Several different specialized tests are necessary to confirm a diagnosis of hemophilia B. In order to determine if an individual has hemophilia B, specialized blood coagulation tests are used to measure how long it takes the blood to clot. The initial test is the activated partial thromboplastin time (aPTT). If the results of the aPTT test are abnormal, more specific blood tests must be used to determine if the cause of the abnormal aPTT is due to a deficiency of factor IX/hemophilia B, factor VIII/hemophilia A or another clotting factor. A specific factor assay also determines the severity level of the factor deficiency.


Hemophilia B Treatment

Currently, there is no cure for hemophilia B; however, the condition can be managed with the help of available treatment choices. In order to achieve sufficient blood clotting and avoid complications associated with the condition, the basic treatment of hemophilia B is the replacement of factor IX. At present, recombinant products or products extracted from human blood or plasma are widely used to substitute factor IX to achieve an adequate amount. Apart from this, Fresh frozen plasma is also used for treatment purposes. It is extracted from human blood and is used only when factor IX concentrate is not available to treat patients with factor IX deficiency. Currently, recombinant factor IX concentrates such as Alprolix, Benefix, Idelvion, Ixinity, Rebinyn, and Rixubis are licensed in the United States for the treatment of hemophilia B. Apart from these, bypassing agents such as Feiba, Novoseven, and Sevenfact and human plasma-derived coagulation factor IX – AlphaNine SD and Mononine – have bagged the FDA approval as well.

Hemophilia B Epidemiology

Key Findings

In 2020, the overall prevalent cases of Hemophilia B accessed to be around 10,739 in the 7MM, which are expected to grow during the study period, i.e., 2018–2030.


The disease epidemiology covered in the report provides historical as well as forecasted Hemophilia B epidemiology [segmented as Total diagnosed Prevalent Pool of Hemophilia B, Severity-specific Prevalent Pool of Hemophilia B, Prevalent population of Hemophilia B with or without Inhibitors and Treated Prevalent Population of Hemophilia B] scenario of Hemophilia B in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2020 to 2030.

Country Wise- Hemophilia B Epidemiology

  • In the United States, the total number of prevalent cases of Hemophilia B was 4,134 in the year 2020 which are expected to grow during the study period, i.e., 2018–2030.
  • In the year 2020, the total prevalent cases of Hemophilia B were 5,408 in EU-5, which are expected to grow during the study period, i.e., 2018–2030.
  • In Japan, the total number of prevalent cases of Hemophilia B was 1,198 in the year 2020 which are expected to grow during the study period, i.e., 2018–2030.
  • Moderate and severe accounts for ~75% of the Hemophilia B patient pool. In the 7MM, there were around 8,054 moderate and severe Hemophilia B cases in 2020.
  • In the 7MM, approximately 5% of Hemophilia B cases develop inhibitors, i.e., 537 patients with inhibitors in 2020. In addition to this, 95% of the Hemophilia B cases were observed without inhibitors. The number of cases for both categories will increase during the study period.

Hemophilia B Drug Chapters

Drug chapter segment of the Hemophilia B report encloses the detailed analysis of Hemophilia B developmental stage pipeline drugs. It also helps to understand the Hemophilia B clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.


Hemophilia B Marketed Drugs


Sevenfact (coagulation factor VIIa [recombinant]-jncw): HEMA Biologics/LFB Pharmaceuticals

Sevenfact from Hema Biologics is a bypassing agent that got regulatory approval in April 2020, from the US FDA as the first new bypassing agent in over 20 years for adults and adolescents. It is also known by its other investigational names, such as Novo7, rFVIIa, and LR769. The active ingredient of Sevenfact is a recombinant analog of activated human coagulation factor (F) VII (FVIIa). It is indicated for the treatment and control of bleeding episodes in adult and adolescent (12–<18 years) hemophilia A and B patients with inhibitors to Factors VIII and IX. It is worth mentioning that it is not indicated for the treatment of congenital factor VII deficiency


Rebinyn (Nonacog beta pegol): Novo Nordisk

Rebinyn is the brand name for nonacog beta pegol; N9-GP in the US. Refixia is the trade name for N9-GP in Europe and Japan. In May 2017, the US FDA approved the Biologics License Application (BLA) for Rebinyn for the treatment of adults and children with hemophilia B. Rebinyn is a purified recombinant human Factor IX (rFIX) with a 40 kDa polyethylene-glycol (PEG) conjugated to the protein. In June 2017, following a positive recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in March, the European Commission later granted marketing authorization for Refixia (nonacog beta pegol; N9-GP) for the treatment of adolescents and adults with hemophilia B.


Alprolix: Bioverativ Therapeutics/Sobi

Alprolix is a medicine used to treat and prevent bleeding in patients with hemophilia B. It can be used in patients of all ages. Alprolix is available as a powder and solvent that are mixed together to make a solution for injection into a vein. The dose and frequency of treatment depend on the patient’s body weight and whether Alprolix is used to treat or prevent bleeding, as well as the severity of the patient’s factor IX deficiency, the extent and location of the bleeding, and the patient’s age and health. In June 2007, the EMA granted an Orphan drug Designation to Alprolix for hemophilia B patients.


Idelvion: CSL Behring

Idelvion, coagulation factor IX (recombinant), albumin fusion protein (rFIX-FP), is used to control and prevent bleeding episodes in people with hemophilia B. Idelvion (albutrepenonacog alfa) is an intravenous injection, which can be self-administered or given by a caregiver. The therapy has been designed to extend the time it stays active in the blood over existing alternatives. According to CSL Behring, the lab-made albumin linked to rFIX has a low potential for immune reactions, a well-known clearance mechanism, and a good safety profile. It can significantly impact the treatment of hemophilia B as it maintains factor IX activity levels above 5% over a prolonged period; this provides excellent bleeding control. In March 2016, the US FDA approved Idelvion which is the first and only factor IX therapy that delivers high-level protection with up to 14-day dosing in appropriate patients.

Note: Full and detailed list of emerging therapies will be provided in the final report.


Hemophilia B Emerging Drugs


Concizumab: Novo Nordisk

Concizumab, which is under development by Novo Nordisk is a high-affinity monoclonal antibody against Tissue Factor Pathway Inhibitor intended for bleeding prevention after subcutaneous administration. Currently, it is in Phase III clinical trial for Hemophilia A and B with and without Inhibitors. The data for phase III is expected in the first half of 2021. In addition to this, the randomized trials of Explorer7 (NCT04083781) and Explorer8 (NCT04082429) tested the use of subcutaneous (under the skin) Concizumab as a preventive or prophylactic treatment for hemophilia. Explorer 7 explores the use of concizumab with inhibitors of FVIII or Repair in patients with hemophilia A or B. The trial's purpose is to assess the safety and efficacy of once-daily prophylactic subcutaneous concizumab in reducing bleeding over three years.


Fitusiran: Sanofi/Alnylam Pharmaceuticals

Fitusiran is a subcutaneously administered small interfering RNA (siRNA) technology to target antithrombin (AT). Currently, it is in Phase III clinical trial for the treatment of Severe Hemophilia A and B Patients with inhibitors and without inhibitors. It works by silencing the gene responsible for AT, which inhibits the protein’s anticoagulant function, which further compensates for the imbalance caused by deficiencies in other clotting proteins, such as factor VIII (Hemophilia B) or factor IX (hemophilia B). This candidate is based on an Alnylam delivery technology that enables increased potency and durability with subcutaneous (under-the-skin) injection, according to the companies.


Etranacogene dezaparvovec (AMT-061): CSL Behring/uniQure

Etranacogene dezaparvovec (AMT-061) is an experimental gene therapy for treating hemophilia B, which is being developed by uniQure. CSL Behring will market the drug internationally under a recent agreement. AMT-061 was given a breakthrough designation by the US FDA and granted PRIME eligibility by the European Medicines Agency. In December 2020, uniQure announced that its hemophilia B gene therapy program, including the pivotal Phase III HOPE-B study, has been placed on clinical hold by the US FDA. Patient dosing is complete in each of uniQure’s three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients.


Fidanacogene elaparvovec (SPK-9001/PF-06838435): Pfizer/Spark Therapeutics

Fidanacogene elaparvovec (previously known as SPK-9001 or PF-06838435) is a novel, investigational bio-engineered AAV vector utilizing a high-activity F9 transgene for hemophilia B or factor IX deficiency. Currently, SPK-9001 is under Phase III (NCT03861273) clinical trial study wherein the study will evaluate the efficacy and safety of SPK-9001 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less).


AMT-060 (AAV5-hFIX): UniQure

AMT-060 is uniQure’s first-generation gene therapy, consisting of an AAV5 vector carrying a gene cassette with the wild-type FIX gene. AMT-060 and AMT-061, which is another identical gene therapy apart from two nucleotide substitutions in the coding sequence for FIX, are being developed by UniQure Biopharma. The gene variant used in AMT-060 is the wild-type FIX protein. All other critical quality attributes of AMT-060 are expected to be comparable to those of AMT-061, as both utilize the same AAV5 capsid and proprietary insect cell-based manufacturing platform. Presently, the AMT-060 is in Phase I/II (NCT02396342) of clinical studies. This ongoing trial is an open-label, multi-center study that includes 10 patients with documented severe or moderately-severe hemophilia B.


Verbrinacogene setparvovec (FLT-180a): Freeline Therapeutics

FLT180a is an experimental next-generation gene therapy for hemophilia B being developed by Freeline. It uses the company’s AAV protein shell, called AAVS3, to deliver a functional version of F9 — the gene that provides instructions to make FIX — to liver cells. An ongoing Phase I/II (NCT03641703) trial is currently enrolling patients in the UK to evaluate the safety and efficacy of FLT180a. The company expects to expand enrolment globally. This trial aims to investigate the long-term safety and durability of FIX activity in participants who have been dosed with a new gene therapy product (FLT180a) in earlier clinical studies. Up to 50 participants who have already been administered with FLT180a in the EU and US will take part in this study. FLT180a has received orphan drug designation and regenerative medicine advanced therapy, or RMAT, a designation from the FDA and PRIority MEdicines, or PRIME, a designation from the EMA.

Note: Full and detailed list of emerging therapies will be provided in the final report.

Hemophilia B Market Outlook

Key Findings

The Hemophilia B market size in the 7MM is expected to change during the study period 2018–2030, at a CAGR of 8.9%. According to the estimates, the highest market size of Hemophilia B is accessed in the United States followed by France, the United Kingdom, Japan, in 2020.


The United States Market Outlook

In United States, the total market size of Hemophilia B therapies is expected to increase at a CAGR of 9.9% during the study period (2018–2030).


EU-5 Countries: Market Outlook

In EU-5, the total market size of Hemophilia B therapies is expected to increase at a CAGR of 7.9% during the study period (2018–2030).


Japan Market Outlook

The total market size of Hemophilia B therapies in Japan is estimated to rise at a CAGR of 7.3% , during the study period (2018–2030).

Hemophilia B Pipeline Development Activities

The drugs which are in pipeline include:


1. Etranacogene dezaparvovec (AMT-061): UniQure Biopharma B.V./ CSL Behring

2. Fidanacogene elaparvovec (SPK-9001/PF-06838435): Pfizer/Spark Therapeutics

3. Fitusiran: Genzyme, a Sanofi Company/ Alnylam Pharmaceuticals

4. Concizumab (NN7415): Novo Nordisk

5. Marstacimab (PF-06741086): Pfizer

6. AMT-060: UniQure Biopharma B.V.

7. SerpinPC: ApcinteX Ltd

8. FLT180a: Freeline Therapeutics

9. SB-FIX: Sangamo Therapeutics

And others…….

Note: Detailed emerging therapies assessment will be provided in the final report.

Hemophilia B Drugs Uptake

Analysts Insight

The current standard of care treatment for severe hemophilia B is the prophylactic intravenous replacement of coagulation factor IX (FIX) to prevent spontaneous bleeding. Persons with hemophilia without prophylactic treatment receive therapy in case of bleeding which is known as on demand treatment. It is projected that the future of hemophilia care is going to trend toward more innovative forthcoming approaches like antibodies, siRNA, and gene therapies. In the upcoming years, the increase in hemophilia B market size is mainly driven by the introduction of pipeline candidates such as Fidanacogene elaparvovec (SPK 9001/PF-06838435), Etranacogene dezaparvovec (AMT-061), Fitusiran, Marstacimab (PF-06741086), Concizumab, FLT180a, and AMT-060 (AAV5-hFIX) during the forecast period (2021–2030). Important areas of unmet medical need are addressed by new therapies, such as reducing the need for intravenous infusions and mitigating the risk of producing neutralizing antibodies or inhibitors. If these treatments are eventually successfully commercialized, will change the current standard of care for Hemophilia B patients. Pfizer/Spark Therapeutics' Fidanacogene elaparvovec, uniQure's Etranacogene dezaparvovec and Sanofi (Genzyme)/Alnylam' Fitusiran for hemophilia B are leading towards the regulatory approval.

Access and Reimbursement Scenario in Hemophilia B Therapies

As per HAS (Haute Autorité de Santé) assessment, the last update for patients with hemophilia B was given in November 2020 (congenital factor IX deficiency). In patients of all age groups, Idelvion (albutrepenonacog alfa) is recommended and the opinion was in favor of reimbursement for the treatment and prophylaxis of patient bleeding. National Health Service (NHS) England had also agreed to reimburse this product with effect from the 20th September 2016. In addition, Alprolix (eftrenonacog alfa) was authorized for reimbursement throughout the UK in September 2016. Along with Scotland, Wales, and Northern Ireland, where Alprolix is already refunded, NHS England declared reimbursement.

KOL-Views

To keep up with current market trends, we take KOLs and SME’s opinion working in the Hemophilia B domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Hemophilia B market trend. This will support the clients in potential upcoming novel treatment by identifying the over Hemophilia B scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Hemophilia B Market by using various Competitive Intelligence tools that includes – SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

  • The report covers the descriptive overview of Hemophilia B, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies.
  • Comprehensive insight has been provided into the Hemophilia B epidemiology and treatment in the 7MM.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Hemophilia B is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of Hemophilia B market; historical and forecasted is included in the report, covering drug outreach in the 7MM.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Hemophilia B market.

Report Highlights

  • In the coming years, Hemophilia B market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia B R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Major players are involved in developing therapies for Hemophilia B. Launch of emerging therapies, will significantly impact the Hemophilia B market.
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Hemophilia B.
  • Our in-depth analysis of the pipeline assets across different stages of development different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the over Hemophilia B scenario of the research and development activities.

Hemophilia B Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Hemophilia B Pipeline Analysis
  • Hemophilia B Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Hemophilia B Report Key Strengths

  • 10 Years Forecast
  • 7MM Coverage
  • Hemophilia B Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake

Hemophilia B Report Assessment

  • SWOT Analysis
  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Conjoint Analysis
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the Hemophilia B Market share (%) distribution in 2018 and how it would look like in 2030?
  • What would be the Hemophilia B total market size as well as market size by therapies across the 7MM during the study period (2018–2030)?
  • What are the key findings pertaining to the market across the 7MM and which country will have the largest Hemophilia B market size during the study period (2018–2030)?
  • At what CAGR, the Hemophilia B market is expected to grow in the 7MM during the study period (2018–2030)?
  • What would be the Hemophilia B market outlook across the 7MM during the study period (2018–2030)?
  • What would be the Hemophilia B market growth till 2030 and what will be the resultant market size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and a subsequent analysis of the associated trends?
  • Hemophilia B patient types/pool where unmet need is more and whether emerging therapies will be able to address the residual unmet need?
  • How emerging therapies are performing on the parameters like efficacy, safety, route of administration (RoA), treatment duration and frequencies on the basis of their clinical trial results?
  • Among the emerging therapies, what are the potential therapies which are expected to disrupt the Hemophilia B market?


Epidemiology Insights:

  • What is the disease risk, burden and unmet needs of the Hemophilia B?
  • What is the historical Hemophilia B patient pool in the seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
  • What would be the forecasted patient pool of Hemophilia B in the 7 major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
  • What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Hemophilia B?
  • Out of Hemophilia B the 7MM countries, which country would have the highest prevalent population of Hemophilia B during the study period (2018–2030)?
  • At what CAGR the population is expected to grow in the 7MM during the study period (2018–2030)?
  • What are the various recent and upcoming events which are expected to improve the diagnosis of Hemophilia B?


Current Treatment Scenario and Emerging Therapies:

  • What are the current options for the treatment of Hemophilia B?
  • What are the current treatment guidelines for the treatment of Hemophilia B in the US, Europe and Japan?
  • How many companies are developing therapies for the treatment of Hemophilia B?
  • How many therapies are developed by each company for the treatment of Hemophilia B?
  • How many emerging therapies are in mid stage, and late stage of development for the treatment of Hemophilia B?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hemophilia B therapies?
  • What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Hemophilia B and their status?
  • What are the key designations that have been granted for the emerging therapies for Hemophilia B?
  • What is the global historical and forecasted market of Hemophilia B?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Hemophilia B market.
  • To understand the future market competition in the Hemophilia B market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Hemophilia B in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
  • Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for Hemophilia B market.
  • To understand the future market competition in the Hemophilia B market.

1. Key Insights

2. Executive Summary of Hemophilia B

3. SWOT Analysis of Hemophilia B

4.Hemophilia B Market Share (%) Distribution Overview at a Glance: By Country

5. Epidemiology an d Market Methodology

6. Hemophilia B: Disease Background and Overview

6.1. Classification of Hemophilia

6.2. Sign and Symptoms of Hemophilia B

6.3. The severity of Hemophilia B

6.4. Risk factors and causes of Hemophilia B

6.5. Pathophysiology

6.6. Genetics of Hemophilia B

6.7. Hemophilia and Inhibitors

6.8. Diagnosis

6.9. Diagnostic Algorithm

6.10. Differential diagnosis of Hemophilia B

6.11. Diagnostic Guidelines for Emergency Department Management of Individuals with Hemophilia and Other Bleeding Disorders

6.12. World Federation of Hemophilia (WFH) Guidelines for Diagnosis of Hemophilia

7. Epidemiology and Patient Population

7.1. Epidemiology Key Findings

7.2.  Assumptions and Rationale: 7MM

7.3.  Epidemiology Scenario: 7MM

7.3.1. Total Diagnosed Prevalent Population of Hemophilia B in the 7MM

7.3.2. Severity- Specific Prevalent Population of Hemophilia B in the 7MM

7.3.3. Prevalent population of Hemophilia B with or without Inhibitors the 7MM

7.3.4. Treated Prevalent Population of Hemophilia B in the 7MM

7.4. United States Epidemiology

7.4.1. Total Diagnosed Prevalent Population of Hemophilia B in the United States

7.4.2. Severity- Specific Prevalent Population of Hemophilia B in the United States

7.4.3. Prevalent population of Hemophilia B with Inhibitors and Without Inhibitors in the United States

7.4.4. Treated Prevalent Population of Hemophilia B in the United States

7.5. EU-5 Country-wise Epidemiology

7.6. Germany Epidemiology

7.6.1. Total Diagnosed Prevalent Population of Hemophilia B in Germany

7.6.2. Severity- Specific Prevalent Population of Hemophilia B in Germany

7.6.3. Prevalent population of Hemophilia B with or without Inhibitors in Germany

7.6.4. Treated Prevalent Population of Hemophilia B in Germany

7.7. France Epidemiology

7.7.1. Total Diagnosed Prevalent Population of Hemophilia B in France

7.7.2. Severity- Specific Prevalent Population of Hemophilia B in France

7.7.3. Prevalent population of Hemophilia B with or without Inhibitors in France

7.7.4. Treated Prevalent Population of Hemophilia B in France

7.8. Italy Epidemiology

7.8.1. Total Diagnosed Prevalent Population of Hemophilia B in Italy

7.8.2. Severity-Specific Prevalent Population of Hemophilia B in Italy

7.8.3. Prevalent population of Hemophilia B with or without Inhibitors in Italy

7.8.4. Treated Prevalent Population of Hemophilia B in Italy

7.9. Spain Epidemiology

7.9.1. Total Diagnosed Prevalent Population of Hemophilia B in Spain

7.9.2. Severity- Specific Prevalent Population of Hemophilia B in Spain

7.9.3. Prevalent population of Hemophilia B with or without Inhibitors in Spain

7.9.4. Treated Prevalent Population of Hemophilia B in Spain

7.10. United Kingdom Epidemiology

7.10.1. Total Diagnosed Prevalent Population of Hemophilia B in the United Kingdom

7.10.2. Severity-Specific Prevalent Population of Hemophilia B in the United Kingdom

7.10.3. Prevalent population of Hemophilia B with or without Inhibitors in the United Kingdom

7.10.4. Treated Prevalent Population of Hemophilia B in the United Kingdom

7.11. Japan Epidemiology

7.11.1. Total Diagnosed Prevalent Population of Hemophilia B in Japan

7.11.2. Severity- Specific Prevalent Population of Hemophilia B in Japan

7.11.3. Prevalence of Hemophilia B with Inhibitors and Without Inhibitors in Japan

7.11.4. Treated Prevalent Population of Hemophilia B in Japan

8. Current Treatment Practices: Hemophilia B

8.1. Treatment for Inhibitors

8.2. Treatment Algorithm

8.3. Guidelines for Emergency Department Management of Individuals with Hemophilia and Other Bleeding Disorders

8.4. National Hemophilia Foundation Guidelines for the Management of Hemophilia

8.4.1. Prophylactic factor replacement therapy

9. Unmet Needs

10. Patient Journey of Hemophilia B

11. Key Endpoints in Hemophilia B

12. Marketed Therapies

12.1. Sevenfact (coagulation factor VIIa [recombinant]-jncw): (HEMA Biologics/LFB Pharmaceuticals

12.1.1. Product Description

12.1.2. Regulatory Milestones

12.1.3. Other Developmental Activities

12.1.4. Pivotal Clinical Trials

12.2. Rebinyn (Nonacog beta pegol): Novo Nordisk

12.2.1. Product Description

12.2.2. Regulatory Milestones

12.2.3. Other Developmental Activities

12.2.4. Pivotal Clinical Trials

12.2.5. Ongoing Current Pipeline Activity

12.3. Idelvion: CSL Behring

12.3.1. Product Description

12.3.2. Regulatory Milestones

12.3.3. Other Developmental Activities

12.3.4. Pivotal Clinical Trials

12.4. Alprolix: Bioverativ Therapeutics/Sobi

12.4.1. Product Description

12.4.2. Regulatory Milestones

12.4.3. Other Developmental Activities

12.4.4. Pivotal Clinical Trial

12.5. Ixinity (Trenonacog alfa): Medexus Pharmaceuticals/ Aptevo Therapeutics

12.5.1. Product Description

12.5.2. Regulatory Milestones

12.5.3. Other Developmental Activities

12.5.4. Pivotal Clinical Trials

12.6. Rixubis: Takeda (Shire/Baxter)

12.6.1. Product Description

12.6.2. Regulatory Milestones

12.6.2.1. Other Developmental Activities

12.6.3. Pivotal Clinical Trial

13. Emerging Therapies

13.1. Key Cross of Emerging Therapies

13.2.  Marstacimab (PF-06741086): Pfizer

13.2.1. Product Description

13.2.1.1. Other Developmental Activities

13.2.2. Clinical Developmental Activities

13.2.3. Safety and Efficacy

13.3.  Fidanacogene elaparvovec (SPK-9001/PF-06838435): Pfizer/Spark Therapeutics

13.3.1. Product Description

13.3.2. Other Developmental Activities

13.3.3. Clinical Developmental Activities

13.3.4. Safety and Efficacy

13.4. Fitusiran (ALN-AT3, SAR-439774): Sanofi (Genzyme)/Alnylam Pharmaceuticals

13.4.1. Product Description

13.4.2. Other Developmental Activities

13.4.3. Clinical Developmental Activities

13.4.4. Safety and Efficacy

13.5. Etranacogene dezaparvovec (AMT-061): CSL Behring/uniQure

13.5.1. Product Description

13.5.2. Other Developmental Activities

13.5.3. Clinical Developmental Activities

13.5.4. Safety and Efficacy

13.6. Concizumab (NN-7415): Novo Nordisk

13.6.1. Product Description

13.6.2. Other Developmental Activities

13.6.3. Clinical Developmental Activities

13.6.4. Safety and Efficacy

13.7. Verbrinacogene setparvovec (FLT-180a): Freeline Therapeutics

13.7.1. Product Description

13.7.2. Other Developmental Activities

13.7.3. Clinical Developmental Activities

13.7.4. Safety and Efficacy

13.8. SerpinPC (AP-0101): ApcinteX

13.8.1. Product Description

13.8.2. Other Developmental Activities

13.8.3. Clinical Developmental Activities

13.9. AMT-060 (AAV5-hFIX): UniQure

13.9.1. Product Description

13.9.2. Other Developmental Activities

13.9.3. Clinical Developmental Activities

13.9.4. Safety and Efficacy

13.10. SB-FIX: Sangamo Therapeutics

13.10.1. Product Description

13.10.2. Other Developmental Activities

13.10.3. Clinical Developmental Activities

14. Conjoint Analysis of Hemophilia B

15. Hemophilia B: Seven Major Market Analysis

15.1. Key Findings

15.2. Market Outlook

15.3. 7MM Market Size

15.3.1. Market Size of Hemophilia B in 7MM

15.3.2. Market Size of Hemophilia B by Therapies in the 7MM

15.4. United States: Market Size

15.4.1. Total Market size of Hemophilia B in the United States

15.4.2. Market Size of Hemophilia B by Therapies in the US

15.5. EU-5 countries: Market Size

15.6.  Germany Market Size

15.6.1. Total Market size of Hemophilia B in Germany

15.6.2. Market Size of Hemophilia B by therapies in Germany

15.7. France Market Size

15.7.1. Total Market size of Hemophilia B in France

15.7.2. Market Size of Hemophilia B by therapies in France

15.8. Italy Market Size

15.8.1. Total Market size of Hemophilia B in Italy

15.8.2. Market Size of Hemophilia B by therapies in Italy

15.9. Spain Market Size

15.9.1. Total Market size of Hemophilia B in Spain

15.9.2. Market Size of Hemophilia B by therapies in Spain

15.10. United Kingdom Market Size

15.10.1. Total Market size of Hemophilia B in the United Kingdom

15.10.2. Market Size of Hemophilia B by therapies in the United Kingdom

15.11. Japan Market Size

15.11.1. Total Market size of Hemophilia B in Japan

15.11.2. Market Size of Hemophilia B by therapies in Japan

16. Market Access and Reimbursement of Hemophilia B

16.1. Access and Reimbursement Overview for Current and Future Therapies

16.2. NICE assessment

16.3. IQWIG assessment

16.4. HAS assessment

16.5. Future Therapy Assessment

17. Market Drivers of Hemophilia B

18. Market Barriers of Hemophilia B

19. Appendix

19.1. Bibliography

19.2. Report Methodology

20. DelveInsight Capabilities

21. Disclaimer

22. About DelveInsight

List of Table

Table 1: Summary of Hemophilia B; Market, Epidemiology, and Key Events (2018–2030)

Table 2: Clotting Factor Tests

Table 3: Differential diagnosis of hemophilia B

Table 4: Total Diagnosed Prevalence of Hemophilia B in the 7MM (2018–2030)

Table 5: Severity- Specific Prevalent Population of Hemophilia B in the 7MM (2018–2030)

Table 6: Prevalent population of Hemophilia B with or without Inhibitors the 7MM (2018–2030)

Table 7: Treated Prevalent Population of Hemophilia B in the 7MM (2018–2030)

Table 8: Total Diagnosed Prevalent Population of Hemophilia B in the United States (2018–2030)

Table 9: Severity- Specific Prevalent Population of Hemophilia B in the United States (2018–2030)

Table 10: Prevalent population of Hemophilia B with Inhibitors and Without Inhibitors in the United States (2018–2030)

Table 11: Treated Prevalent Population of Hemophilia B in the United States (2018–2030)

Table 12: Total Diagnosed Prevalent Population of Hemophilia B in Germany (2018–2030)

Table 13: Severity- Specific Prevalent Population of Hemophilia B in Germany (2018–2030)

Table 14: Prevalent population of Hemophilia B with or without Inhibitors in Germany (2018–2030)

Table 15: Treated Prevalent Population of Hemophilia B in Germany (2018–2030)

Table 16: Total Diagnosed Prevalent Population of Hemophilia B in France (2018–2030)

Table 17: Severity- Specific Prevalent Population of Hemophilia B in France (2018–2030)

Table 18: Prevalent population of Hemophilia B with or without Inhibitors in France (2018–2030)

Table 19: Treated Prevalent Population of Hemophilia B in France (2018–2030)

Table 20: Total Diagnosed Prevalent Population of Hemophilia B in Italy (2018–2030)

Table 21: Severity-Specific Diagnosed Prevalence of Hemophilia B in Italy (2018–2030)

Table 22: Prevalent population of Hemophilia B with or without Inhibitors in Italy (2018–2030)

Table 23: Treated Prevalent Population of Hemophilia B in Italy (2018–2030)

Table 24: Total Diagnosed Prevalent Population of Hemophilia B in Spain (2018–2030)

Table 25: Severity-Specific Prevalent Population of Hemophilia B in Spain (2018–2030)

Table 26: Prevalent population of Hemophilia B with or without Inhibitors in Spain (2018–2030)

Table 27: Treated Prevalent Population of Hemophilia B in Spain (2018–2030)

Table 28: Total Diagnosed Prevalent Population of Hemophilia B in the United Kingdom (2018–2030)

Table 29: Severity-Specific Prevalent Population of Hemophilia B in the United Kingdom (2018–2030)

Table 30: Prevalent population of Hemophilia B with or without Inhibitors in the United Kingdom (2018–2030)

Table 31: Treated Prevalent Population of Hemophilia B in the United Kingdom (2018–2030)

Table 32: Total Diagnosed Prevalent Population of Hemophilia B in Japan (2018–2030)

Table 33: Severity- Specific Prevalent Population of Hemophilia B in Japan (2018–2030)

Table 34: Prevalence of Hemophilia B with Inhibitors and Without Inhibitors in Japan (2018–2030)

Table 35: Treated Prevalent Population of Hemophilia B in Japan (2018–2030)

Table 36: Approved drugs for Hemophilia B

Table 37: Definitions of factor replacement therapy protocols

Table 38: List of Few Marketed Therapies

Table 39: Dosing for Treatment and Control of Bleeding

Table 40: Dosing for On-demand Treatment and Control of Bleeding Episodes

Table 41: Dosing for Perioperative Management

Table 42: Rebinyn (Nonacog beta pegol); Clinical Trial Description, 2020

Table 43: Dosing of On-demand Control and Prevention of Bleeding Episodes

Table 44: Dosing for Perioperative Management of Bleeding

Table 45: Dosing for On-demand Treatment and Control of Bleeding Episodes

Table 46: Dosing for Perioperative Management

Table 47: Dosing for Control and Prevention of Bleeding Episodes

Table 48: Dosing for Perioperative Management

Table 49: Key Cross of Emerging Therapies

Table 50: Marstacimab (PF-06741086), Clinical Trial Description

Table 51: Fidanacogene elaparvovec (SPK-9001/ PF-06838435), Clinical Trial Description

Table 52: Fitusiran, Clinical Trial Description

Table 53: Etranacogene dezaparvovec (AMT-061), Clinical Trial Description

Table 54: Concizumab, Clinical Trial Description

Table 55: Verbrinacogene setparvovec (FLT-180a), Clinical Trial Description

Table 56: SerpinPC; Clinical Trial Description

Table 57: AMT-060 (AAV5-hFIX), Clinical Trial Description

Table 58: SB-FIX, Clinical Trial Description

Table 59: Market Size of Hemophilia B in the 7MM, in USD Million (2018–2030)

Table 60: Total Market Size of Hemophilia B in the United States, in USD Million (2018–2030)

Table 61: Total Market Size of Hemophilia B in Germany, in USD Million (2018–2030)

Table 62: Total Market Size of Hemophilia B in France, in USD Million (2018–2030)

Table 63: Total Market Size of Hemophilia B in Italy, in USD Million (2018–2030)

Table 64: Total Market Size of Hemophilia B in Spain, in USD Million (2018–2030)

Table 65: Total Market Size of Hemophilia B in the United Kingdom, in USD Million (2018–2030)

Table 66: Total Market Size of Hemophilia B in Japan, in USD Million (2018–2030)

Table 67: NICE assessment for Hemophilia B Therapies

Table 68: IQWIG assessment of Hemophilia B Therapies

List of Figures

Figure 1: Hemophilia B SWOT Analysis

Figure 2: Types of Hemophilia

Figure 3: Signs and Symptoms of Hemophilia B

Figure 4: Severity of the disease on the basis of clotting factors

Figure 5: Inheritance Pattern for Hemophilia B

Figure 6: Severity of the disease on the basis of clotting factors

Figure 7: Vitamin K-dependent carboxylation

Figure 8: Inheritance pattern if the mother is the carrier of Hemophilia B gene

Figure 9: Inheritance pattern if the father has Hemophilia B

Figure 10: Inheritance pattern if the mother is a carrier and the father has Hemophilia B

Figure 11: Inhibitors and Hemophilia B

Figure 12: Diagnostic Algorithm

Figure 13: Total Diagnosed Prevalent Population of Hemophilia B in the 7MM (2018–2030)

Figure 14: Severity- Specific Prevalent Population of Hemophilia B in the 7MM (2018–2030)

Figure 15: Prevalent population of Hemophilia B with or without Inhibitors in the 7MM (2018–2030)

Figure 16: Treated Prevalent Population of Hemophilia B in the 7MM (2018–2030)

Figure 17: Total Diagnosed Prevalent Population of Hemophilia B in the United States (2018–2030)

Figure 18: Severity- Specific Prevalent Population of Hemophilia B in the United States (2018–2030)

Figure 19: Prevalent population of Hemophilia B with Inhibitors and Without Inhibitors in the United States (2018–2030)

Figure 20: Treated Prevalent Population of Hemophilia B in the United States (2018–2030)

Figure 21: Total Diagnosed Prevalent Population of Hemophilia B in Germany (2018–2030)

Figure 22: Severity- Specific Prevalent Population of Hemophilia B in Germany (2018–2030)

Figure 23: Prevalent population of Hemophilia B with or without Inhibitors in Germany (2018–2030)

Figure 24: Treated Prevalent Population of Hemophilia B in Germany (2018–2030)

Figure 25: Total Diagnosed Prevalent Population of Hemophilia B in France (2018–2030)

Figure 26: Severity-Specific Prevalent Population of Hemophilia B in France (2018–2030)

Figure 27: Prevalent population of Hemophilia B with or without Inhibitors in France (2018–2030)

Figure 28: Treated Prevalent Population of Hemophilia B in France (2018–2030)

Figure 29: Total Diagnosed Prevalent Population of Hemophilia B in Italy (2018–2030)

Figure 30: Severity- Specific Prevalent Population of Hemophilia B in Italy (2018–2030)

Figure 31: Prevalent population of Hemophilia B with or without Inhibitors in Italy (2018–2030)

Figure 32: Treated Prevalent Population of Hemophilia B in Italy (2018–2030)

Figure 33: Total Diagnosed Prevalent Population of Hemophilia B in Spain (2018–2030)

Figure 34: Severity- Specific Prevalent Population of Hemophilia B in Spain (2018–2030)

Figure 35: Prevalent population of Hemophilia B with or without Inhibitors in Spain (2018–2030)

Figure 36: Treated Prevalent Population of Hemophilia B in Spain (2018–2030)

Figure 37: Total Diagnosed Prevalent Population of Hemophilia B in the United Kingdom (2018–2030)

Figure 38: Severity-Specific Prevalent Population of Hemophilia B in the United Kingdom (2018–2030)

Figure 39: Prevalent population of Hemophilia B with or without Inhibitors in the United Kingdom (2018–2030)

Figure 40: Treated Prevalent Population of Hemophilia B in the United Kingdom (2018–2030)

Figure 41: Total Diagnosed Prevalent Population of Hemophilia B in Japan (2018–2030)

Figure 42: Severity- Specific Prevalent Population of Hemophilia B in Japan (2018–2030)

Figure 43: Prevalence of Hemophilia B with Inhibitors and Without Inhibitors in Japan (2018–2030)

Figure 44: Treated Prevalent Population of Hemophilia B in Japan (2018–2030)

Figure 45: Hemophilia B without History of Inhibitor

Figure 46: Hemophilia B with History of Inhibitor

Figure 47: Unmet Needs

Figure 48: Patient Journey

Figure 49: Mechanism of SerpinPC inhibiting APC

Figure 50: Sangamo’s In Vivo Genome Editing Approach Using Zinc Finger Nucleases

Figure 51: Market Size of Hemophilia B in the 7MM, in USD Million (2018–2030)

Figure 52: Total Market Size of Hemophilia B by therapies in the 7MM, in USD Million (2018–2030)

Figure 53: Total Market Size of Hemophilia B in the United States, in USD Million (2018–2030)

Figure 54: Total Market Size of Hemophilia B by therapies in the United States, in USD Million (2018–2030)

Figure 55: Total Market Size of Hemophilia B in Germany, in USD Million (2018–2030)

Figure 56: Total Market Size of Hemophilia B by therapies in Germany, in USD Million (2018–2030)

Figure 57: Total Market Size of Hemophilia B in France, in USD Million (2018–2030)

Figure 58: Total Market Size of Hemophilia B by therapies in France, in USD Million (2018–2030)

Figure 59: Total Market Size of Hemophilia B in Italy, in USD Million (2018–2030)

Figure 60: Total Market Size of Hemophilia B by therapies in Italy, in USD Million (2018–2030)

Figure 61: Total Market Size of Hemophilia B in Spain, in USD Million (2018–2030)

Figure 62: Total Market Size of Hemophilia B by therapies in Spain, in USD Million (2018–2030)

Figure 63: Total Market Size of Hemophilia B in the United Kingdom, in USD Million (2018–2030)

Figure 64: Total Market Size of Hemophilia B by therapies in the United Kingdom, in USD Million (2018–2030)

Figure 65: Total Market Size of Hemophilia B in Japan, in USD Million (2018–2030)

Figure 66: Total Market Size of Hemophilia B by therapies in Japan, in USD Million (2018–2030)

Figure 67: Market Drivers

Figure 68: Market Barriers

UniQure Biopharma B.V./ CSL Behring

Pfizer/Spark Therapeutics

Genzyme, a Sanofi Company/ Alnylam Pharmaceuticals

Novo Nordisk

Pfizer

UniQure Biopharma B.V.

ApcinteX Ltd

Freeline Therapeutics

Sangamo Therapeutics

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  • Hemophilia B treatment algorithm
  • Hemophilia B drugs
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