Hemophilia A Market

Key Findings

• The total Hemophilia A prevalent population in the 7MM comprised of 45,149 cases in 2021 and are projected to increase during the forecast period.
• The total Hemophilia A market size in the 7MM USD 8,510 million in 2021 and is projected to grow during the forecast period (2022-2032).
• Some of the leading pharma giants in the Hemophilia A Market include BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, and others.

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DelveInsight's "Hemophilia A- Market Insights, Epidemiology and Market Forecast– 2032" report delivers an in-depth understanding of the Hemophilia A, historical and forecasted epidemiology as well as the Hemophilia A market trends in the United States, EU-5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

Hemophilia A market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Hemophilia A market size from 2019 to 2032. The report also covers current Hemophilia A treatment practice/algorithm, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

• The United States
• EU-5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2019-2032

Hemophilia A Disease Understanding and Treatment Algorithm

Hemophilia A Overview

Hemophilia A is a genetic bleeding disorder in which an individual lacks or has low levels of proteins named clotting factor VIII. The mainstay treatment option has long been FVIII replacement therapy. Initially, FVIII replacement was accomplished by donated whole blood, subsequently by plasma and currently by recombinant human FVIII (rFVIII) replacement therapies, which revolutionized the treatment of Hemophilia A. Although hemophilia is usually diagnosed at birth, the disorder can also be acquired later in life if the body begins to produce antibodies that attack and destroy clotting factors. However, this acquired type of hemophilia is very rare.

Hemophilia A Diagnosis

The symptoms of Hemophilia A can vary greatly from one person to another, it ranges from mild to moderate to severe. The age of onset and frequency of bleeding episodes depend upon the amount of factor VIII protein and overall clotting ability of the blood. In most individuals, regardless of severity, bleeding episodes tend to be more frequent in childhood and adolescence than in adulthood.

Additionally, the diagnosis of Hemophilia A depends on the identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation, and a variety of specialized laboratory tests. The identification of a hemizygous F8 pathogenic variant on molecular genetic testing in a male proband confirms the diagnosis.

Hemophilia A Treatment

About 30% of severe Hemophilia A patients develop neutralizing anti-FVIII alloantibodies (inhibitors), which render the FVIII replacement ineffective. The standard of care therapy for patients with inhibitors is to induce immune tolerance with high-dose, high-frequency FVIII and treatment with bypassing agents (e.g. recombinant activated factor VII such as NOVOSEVEN, FEIBA). There are many approved therapies for the management of Hemophilia A which includes HEMLIBRA (for both inhibitor and non-inhibitor), SEVENFACT, KOGENATE FS, FEIBA, KOVALTRY, ELOCTATE, NUWIQ, AFSTYLA and others.

Hemophilia A Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of hemophilia A, age-specific prevalent cases of hemophilia A, severity-specific prevalent cases of hemophilia A, prevalent cases of hemophilia A with or without inhibitors and the treated patient pool of hemophilia A in the 7MM market covering the United States, EU-5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2019 to 2032.

Key Findings

This section provides glimpse of the Hemophilia A epidemiology in the 7MM

Country Wise- Hemophilia A Epidemiology

The epidemiology segment also provides the Hemophilia A epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

• The total prevalent population of Hemophilia A in the 7MM comprised of 45,149 cases in 2021 and are projected to increase during the forecast period.
• The total prevalent population of Hemophilia A in the United States is 14,146 in 2021.
• The United States contributed to the largest prevalent population of Hemophilia A, accounting for ~ 31% of the 7MM in 2021.
• In the EU5 countries, the diagnosed prevalence of Hemophilia A was found to be maximum in France followed by UK. While, the least number of cases were found in Spain, in 2021.
• In Japan, the total prevalent population of Hemophilia A is 5,542 in 2021 and is anticipated to rise during the forecast period.
• In 2021, the prevalent cases of mild in the United States were identified to be 3,360, moderate as 2,688 and severe as 7,391.
• In the US 2,217 cases accounts for inhibitor and 11,221 cases for non-inhibitor in 2021

Hemophilia A Drug Chapters

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

Concizumab: Novo Nordisk

Concizumab, under development by Novo Nordisk, is a high-affinity monoclonal antibody against tissue factor pathway inhibitor (TFPI) intended for bleeding prevention after subcutaneous administration. In the first quarter of 2022, the company announced that the US submission for inhibitor indications is expected later in 2022. The company also expects to submit concizumab for regulatory approval for the prophylactic treatment of Hemophilia A or B with inhibitors in the third quarter of 2022 in the US and Japan and by 2023 in EU (for all indications) and the UK.

Fitusiran: Sanofi

Fitusiran, currently under development by Sanofi (Genzyme)/ Alnylam Pharmaceuticals, is a subcutaneously administered small interfering RNA (siRNA) technology to target antithrombin. In the Q2 2022, the company announced data with lower doses expected in the second half of 2023. The company anticipates the First filing by 2024. The Pediatric (age 1–11) study is ongoing, and the company expects the first filing planned by 2026.

BIVV001: Sanofi in collaboration with Sobi

BIVV001, which is currently under development by Sanofi in collaboration with sobi, is a novel and investigational recombinant Factor VIII therapy designed to extend protection from bleeds with once-weekly prophylactic dosing for people with Hemophilia A. It is administered by the intravenous route. In Q1 2022, Sanofi announced that the positive pivotal trial readout is expected in the second half of 2022 and the company is also planning the US submissions in the mid of 2022. In the Q2 of 2022 results, the company recently announced positive Phase III data in which primary and key secondary endpoints were met. The company also submitted the FDA BLA, and Japan filing is estimated in H2 2022.

Marstacimab: Pfizer

Marstacimab, which is currently under development by Pfizer is a Tissue Factor Pathway Inhibitor monoclonal antibody to treat Hemophilia A and B with or without Inhibitors. . Currently, it is in Phase III clinical trial for the treatment of severe Hemophilia A and B with or without Inhibitors. The company also projected pivotal readout by Q2 2023 and projected submission for non-inhibitor indication Q3 2023.

Valrox: BioMarin Pharmaceutical

Valoctocogene roxaparvovec is under development by BioMarin Pharmaceutical, an experimental gene therapy based on adeno-associated virus (AAV) vectors. BioMarin expects that roctavian (valoctocogene roxaparvovec) for the treatment of severe Hemophilia A will be approved in Europe in the third quarter of 2022; re-submission of BLA in the US planned for September 2022. The Phase IIIb study to evaluate roctavian (valrox) with prophylactic corticosteroids has completed enrollment and is expected to read out in early 2023.

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

Hemophilia A Market Outlook

In the market, there are several recombinant factor VIII (FVIII) products available with high specific activities (the amount of desired clotting factor per mg of total protein). Plasma-derived clotting factors products are also available. However, the current market is mainly dominated by the recombinants of several generations (recombinant third-generation, and recombinant second generation). Although there are several products available at present, then again, none of these products might be able to cure or manage this situation, completely

HEMLIBRA—a product of “Genentech/Chugai/Roche”, is a novel bispecific antibody that was first approved in 2017 for patients with Hemophilia A with FVIII inhibitors in the US. However, in 2018, HEMLIBRA was approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with Hemophilia A without factor VIII inhibitors. This therapy is also approved for treatment for people with Hemophilia A without or without inhibitors in Europe as well as in Japan. In October 2021, Genentech/Roche filed for label expansion approval of HEMLIBRA in Europe for mild and moderate Hemophilia A without inhibitor. Now, the company is planning for Europe approval to include mild/moderate patients form Phase III data (HAVEN 6) in 2022. In June 2022, Chugai received approval in Japan for acquired Hemophilia A based on a domestic Phase III AGEHA study. HEMLIBRA is the only marketed therapy which is administered subcutaneously making this the most effective drug of patient’s choice. In the emerging pipeline Fitusuran and Concizumab are the potential candidates that can pose threat to HEMLIBRA’s popularity once approved, and can significantly decrease the market share of this drug.

Currently, the treatment hemisphere is mainly driven by non-inhibitors drug candidates. However, Hemophilia treatment landscape continues to evolve, and several companies are furiously working toward the development of new treatments that could potentially cure and change the treatment landscape of Hemophilia A. Key players, like BioMarin Pharmaceutical, Novo Nordisk, Sanofi, Pfizer/Sangamo, Roche/Spark Therapeutics, and others, are coming up with novel therapeutic approaches that can entirely change the treatment landscape of Hemophilia A. Potential emerging candidates pose an adverse threat to current Hemophilia A pharmaceuticals giants, by means their potential to transform the standard of care for Hemophilia A patients. During the forecast period, therapies like Valrox (Valoctocogene Roxaparvovec), BIVV001 (rFVIIIFc-VWF-XTEN), SPK-8011, Fitusiran, Concizumab (which is conducting trial for both inhibitors and non-inhibitors), Marstacimab (PF-06741086), giroctocogene fitelparvovec, and others are expected to enter in the seven major markets.

Key Findings

• This section includes a glimpse of the Hemophilia A in the 7MM market.
• The total market size of Hemophilia A (Hemophilia A) in the 7MM USD 8,510 million in 2021 and is projected to grow during the forecast period (2022-2032).
• According to the estimates, the highest market size of Hemophilia A is from the United States in 2021.
• Among the EU5 countries, France has the maximum revenue share followed by the United Kingdom in 2021 while Spain has the lowest market share.
• The market size of Hemophilia A in Japan is USD 956 million in 2021 which is expected to rise during the forecast period (2022-2032).
• The upcoming therapies of Hemophilia A are expected to combat the current unmet needs faced by the patients with Hemophilia A.

The United States Market Outlook

The total market size of Hemophilia A in the United States is expected to increase with a CAGR of 8.5% during the study period (2019–2032).

EU-5 Market Outlook

The total market size of Hemophilia A in EU5 is expected to increase with a CAGR of 7.6% during the study period (2019–2032).

Japan Market Outlook

The total market size of Hemophilia A in Japan is expected to increase with a CAGR of 5.1% during the study period (2019–2032).

Analyst Commentary

• The current emerging market of Hemophilia A possesses a robust pipeline. There are many emerging therapy in the higher stage i.e.,Concizumab (Novo Nordisk), Fitusiran (Sanofi), Valrox (BioMarin ), BIVV001 (Sanofi in collaboration with Sobi), SPK-8011(Spark Therapeutics) and others.
• There are many marketed drugs for Hemophilia A but HEMLIBRA is ruling the market due to its ease of administration which continue to have the highest patient share.
• Given the compartmentalization of the market competition, companies are looking for more drugs which can target the inhibitor segments with keeping in mind the frequency and duration of treatment.
• In the coming years we anticipate the launches of many novel candidates in this hemisphere which can be beneficial for the inhibitor patient segment.

Hemophilia A Drugs Uptake

This section focusses on the rate of uptake of the potential drugs expected to get launched in the market during the study period 2019-2032. The analysis covers Hemophilia A market uptake by drugs; patient uptake by therapies; and sales of each drug. For example- Concizumab, is a high-affinity monoclonal antibody. Currently, the drug is in the Phase III stage of clinical development for Hemophilia A. Novo Nordisk is expecting the US submission for inhibitor indications is expected later in 2022. The drug is expected to launch in US in inhibitor section 2023. As per our analysis, Concizumab drug uptake in the US is expected to be medium- fast with 80% of probability of success and probability adjusted peak share of 28%

Hemophilia A Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II, Phase III and Phase I stage. It also analyzes key players involved in developing targeted therapeutics.

Hemophilia A Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Hemophilia A emerging therapies.

KOL- Views

To keep up with current market trends, we take KOLs and SME's opinion working in the domain through primary research to fill the data gaps and validate our secondary research. Some of the leaders are – M.D., senior vice president, Center for Neuroscience and Behavioral Medicine, and director, Brain Tumor Institute, Children’s National Hospital and Senior Associate Scientist Emeritus at The Hospital for Sick Children Toronto, Canada. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Hemophilia A market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform competitive and market Intelligence analysis of the Hemophilia A market by using various competitive intelligence tools that include–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 A, explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
• Comprehensive insight has been provided into the Hemophilia A epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for Hemophilia A are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of Hemophilia A market; historical and forecasted is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM Hemophilia A market.

Report Highlights

• In the coming years, Hemophilia A market is set to change due emerging therapies in the pipeline, 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 A R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• The report also encompasses other major segments, i.e., age-specific, severity-specific cases of hemophilia A, prevalent pool based on inhibitor and non-inhibitor segment, and treated pool of Hemophilia A.
• Expected Launch of potential therapies, Concizumab (Novo Nordisk), Fitusiran (Sanofi), Valrox (BioMarin), BIVV001 (Sanofi), SPK-8011(Spark Therapeutics), and others might change the landscape in treatment of Hemophilia A.
• Currently there are many approved therapies targeting the non-inhibitor segments. The current emerging pipeline consists of a number of novel candidates targeting both inhibitor and non-inhibitor segments. Many drugs are also being studied with subcutaneous route of administration.

Hemophilia A Report Insights

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

Hemophilia A Report Key Strengths

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

Hemophilia A Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• SWOT
• Attribute Analysis

Key Questions

Market Insights:

• What was the Hemophilia A market share (%) distribution in 2019 and how it would look like in 2032?
• What would be the Hemophilia A total market size as well as market size by therapies across the 7MM during the study period (2019–2032)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest Hemophilia A market size during the study period (2019–2032)?
• At what CAGR, the Hemophilia A market is expected to grow at the 7MM level during the study period (2019–2032)?
• What would be the Hemophilia A market outlook across the 7MM during the study period (2019–2032)?
• What would be the Hemophilia A market growth till 2032 and what will be the resultant market size in the year 2032?
• How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What is the disease risk, burden and unmet needs of Hemophilia A?
• What is the historical Hemophilia A patient pool in the United States, the EU-5 (Germany, France, Italy, Spain, and the UK) and Japan?
• What would be the forecasted patient pool of Hemophilia A at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Hemophilia A?
• Out of the above-mentioned countries, which country would have the highest prevalent population of Hemophilia A during the study period (2019–2032)?
• At what CAGR the population is expected to grow across the 7MM during the study period (2019–2032)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the treatment of Hemophilia A? What are the current treatment guidelines for the treatment of Hemophilia A in the US and Europe?
• What are the Hemophilia A marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
• How many companies are developing therapies for the treatment of Hemophilia A?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of Hemophilia A?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hemophilia A 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 A and their status?
• What are the key designations that have been granted for the emerging therapies for Hemophilia A?
• What are the 7MM historical and forecasted market of Hemophilia A?

Reasons to buy

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