Hemophilia A Market Report 2034: Epidemiology Data, Pipeline Therapies, Latest FDA, EMA, PDMA Approvals by DelveInsight

Hemophilia A companies are BioMarin Pharmaceutical, Roche, ApcinteX, Novo Nordisk, Sanofi, Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer

LAS VEGAS, NEVADA, UNITED STATES, May 27, 2024 /EINPresswire.com/ -- DelveInsight’s “Hemophilia A Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Hemophilia A, historical and forecasted epidemiology as well as the Hemophilia A market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

To Know in detail about the Hemophilia A market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Hemophilia A Market Forecast

Some of the key facts of the Hemophilia A Market Report:

The Hemophilia A market size is anticipated to grow with a significant CAGR during the study period (2020-2034).
The United States accounts for the largest market size (around 65%) of hematopoietic stem cell transplantation, in comparison to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
Myeloablative regimen, Nonmyeloablative/reduced-intensity regimen, filgrastim (G-CSF) + Mozobil, G-CSF + cyclophosphamide (CY) are some of the regimen and therapies currently being used in the market. Myeloablative regimen is expected to capture the largest market followed by potential upcoming drug Iomab-B.
In September 2023, the US Food and Drug Administration (FDA) approved APHEXDA (motixafortide) in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma.
In April 2024, Actinium Pharmaceuticals announced the results from the Phase III SIERRA trial of Iomab-B, which showed that an Iomab-B led bone marrow transplant (BMT) results in higher rates of remissions and durable Complete Remission (dCR), which is the primary endpoint of the SIERRA trial, as well as significant improvement in overall survival in TP53 positive patients.
Medac, licensor of Medexus's treosulfan commercialization rights, is addressing FDA inquiries on the treosulfan NDA resubmission following Phase III trial feedback, aiming for FDA acceptance by the first half of 2024.
Among the 7MM, the United States accounted for highest cases of hematopoietic stem cell transplantation (HSCT) with more than 40% cases, in 2023.
In terms of type-specific cases of HSCT, the percentage of autologous HSCT cases is more in comparison to allogeneic HSCT across the 7MM except for Japan.
Key Hemophilia A Companies: BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, and others
Key Hemophilia A Therapies: RG6357 (SPK-8011), RG6358 (SPK-8016), SerpinPC, Concizumab (NN7415), Fitusiran (ALN-AT3, SAR-439774), BIVV001 (Efanesoctocog alfa) (rFVIIIFc-VWF-XTEN), Marstacimab (PF-06741086), NNC0365-3769 A (Mim8), Giroctocogene fitelparvovec (SB-525 or PF-07055480), BAY2599023 (DTX201 AAV FVIII), and others
Based on DelveInsight's estimates, the number of hematopoietic stem cell transplantation (HSCT) cases in the United States accounted for over 40% of the total prevalent cases reported in the 7MM in 2023.
HSCT mainly includes two types of transplants, autologous and allogeneic. DelveInsight’s consultant estimates that the maximum number of type-specific cases of HSCT belonged to the autologous HSCT. There were around 60% cases of autologous HSCT in the United States in 2023.
The Hemophilia A market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Hemophilia A pipeline products will significantly revolutionize the Hemophilia A market dynamics.

Hemophilia A Overview

Hemophilia A is a genetic disorder characterized by a deficiency or dysfunction of clotting factor VIII, a protein essential for normal blood clotting. This condition is inherited in an X-linked recessive pattern, meaning it primarily affects males while females typically carry the genetic mutation without displaying symptoms.

Individuals with hemophilia A are prone to prolonged bleeding episodes, particularly after injury or surgery, due to the impaired ability of their blood to clot properly. Symptoms may include frequent nosebleeds, easy bruising, prolonged bleeding from cuts or injuries, and in severe cases, spontaneous bleeding into joints and muscles.

Treatment for hemophilia A often involves replacement therapy with clotting factor VIII concentrates to restore the missing or deficient clotting factor in the blood. This can be administered preventively to reduce the risk of bleeding (prophylactic therapy) or on-demand to manage bleeding episodes. In recent years, gene therapy has emerged as a promising approach for treating hemophilia A by introducing functional factor VIII genes into the body to produce the missing clotting factor.
While there is currently no cure for hemophilia A, ongoing advancements in treatment options and management strategies have significantly improved the quality of life for individuals living with this condition. Additionally, preventive measures such as avoiding activities with a high risk of injury and maintaining regular medical follow-ups can help minimize complications associated with hemophilia A.

Hemophilia A Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Hemophilia A Epidemiology Segmentation:

The Hemophilia A market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Total Prevalence of Hemophilia A
Prevalent Cases of Hemophilia A by severity
Gender-specific Prevalence of Hemophilia A
Diagnosed Cases of Episodic and Chronic Hemophilia A

Download the report to understand which factors are driving Hemophilia A epidemiology trends @ Hemophilia A Epidemiology Forecast

Hemophilia A Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Hemophilia A market or expected to get launched during the study period. The analysis covers Hemophilia A market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Hemophilia A Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Hemophilia A Key Companies

BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical

Hemophilia A Therapies

RG6357 (SPK-8011), RG6358 (SPK-8016), SerpinPC, Concizumab (NN7415), Fitusiran (ALN-AT3, SAR-439774), BIVV001 (Efanesoctocog alfa) (rFVIIIFc-VWF-XTEN), Marstacimab (PF-06741086), NNC0365-3769 A (Mim8), Giroctocogene fitelparvovec (SB-525 or PF-07055480), BAY2599023 (DTX201 AAV FVIII)

Discover more about therapies set to grab major Hemophilia A market share @ Hemophilia A Treatment Landscape
Hemophilia A Market Outlook

In the realm of the Hemophilia A Market, a variety of recombinant factor VIII (FVIII) products boasting high-specific activities are available, along with plasma-derived clotting factor products. Notably, the market is primarily dominated by recombinant products spanning multiple generations, including third and second-generation variants.

Despite the array of products currently accessible, none offer a definitive cure or comprehensive management of the condition. HEMLIBRA, a groundbreaking bispecific antibody developed by Genentech/Chugai/Roche, entered the scene in 2017 with initial approval for Hemophilia A patients harboring FVIII inhibitors in the US. Subsequently, in 2018, HEMLIBRA secured approval for routine prophylaxis to prevent or diminish the frequency of bleeding episodes in adults and pediatric patients, including newborns, with Hemophilia A devoid of FVIII inhibitors. This therapeutic intervention has also gained approval for Hemophilia A treatment across Europe and Japan, encompassing patients with or without inhibitors.

In early 2023, the FDA granted approval to ALTUVIIIO for routine prophylaxis, on-demand treatment to manage bleeding episodes, and perioperative management in both adults and pediatric populations with hemophilia A. Later, in June 2023, the FDA greenlit ROCTAVIAN, a revolutionary gene therapy designed for the treatment of severe hemophilia A in adults lacking pre-existing antibodies to adeno-associated virus serotype 5, as determined by an FDA-approved test. Notably, both ALTUVIIIO and ROCTAVIAN demonstrate superior efficacy and safety compared to HEMLIBRA.

Presently, the treatment landscape primarily revolves around non-inhibitor drug candidates. However, the Hemophilia A treatment arena is in a state of constant evolution, with numerous companies vigorously pursuing the development of novel therapies capable of potentially revolutionizing the treatment paradigm and offering a cure for Hemophilia A. Noteworthy players in this endeavor include Novo Nordisk, Sanofi, Pfizer/Sangamo, Roche/Spark Therapeutics, among others, each spearheading innovative therapeutic approaches that have the potential to reshape the Hemophilia A treatment market landscape.

Scope of the Hemophilia A Market Report:

Study Period: 2020–2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Hemophilia A Companies: BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, and others
Key Hemophilia A Therapies: RG6357 (SPK-8011), RG6358 (SPK-8016), SerpinPC, Concizumab (NN7415), Fitusiran (ALN-AT3, SAR-439774), BIVV001 (Efanesoctocog alfa) (rFVIIIFc-VWF-XTEN), Marstacimab (PF-06741086), NNC0365-3769 A (Mim8), Giroctocogene fitelparvovec (SB-525 or PF-07055480), BAY2599023 (DTX201 AAV FVIII), and others
Hemophilia A Therapeutic Assessment: Hemophilia A current marketed and Hemophilia A emerging therapies
Hemophilia A Market Dynamics: Hemophilia A market drivers and Hemophilia A market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
Hemophilia A Unmet Needs, KOL’s views, Analyst’s views, Hemophilia A Market Access and Reimbursement

To know more about Hemophilia A companies working in the treatment market, visit @ Hemophilia A Clinical Trials and Therapeutic Assessment

Table of Contents

1. Hemophilia A Market Report Introduction
2. Executive Summary for Hemophilia A
3. SWOT analysis of Hemophilia A
4. Hemophilia A Patient Share (%) Overview at a Glance
5. Hemophilia A Market Overview at a Glance
6. Hemophilia A Disease Background and Overview
7. Hemophilia A Epidemiology and Patient Population
8. Country-Specific Patient Population of Hemophilia A
9. Hemophilia A Current Treatment and Medical Practices
10. Hemophilia A Unmet Needs
11. Hemophilia A Emerging Therapies
12. Hemophilia A Market Outlook
13. Country-Wise Hemophilia A Market Analysis (2020–2034)
14. Hemophilia A Market Access and Reimbursement of Therapies
15. Hemophilia A Market Drivers
16. Hemophilia A Market Barriers
17. Hemophilia A Appendix
18. Hemophilia A Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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Hemophilia A Pipeline

"Hemophilia A Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Hemophilia A market. A detailed picture of the Hemophilia A pipeline landscape is provided, which includes the disease overview and
Hemophilia A treatment guidelines.

Hemophilia A Epidemiology

DelveInsight's 'Hemophilia A Epidemiology Forecast to 2034' report delivers an in-depth understanding of the disease, historical and forecasted Hemophilia A epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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