Hemophilia Market to Proliferate Immensly, Predicts DelveInsight | Key Companies to Look Out for

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Written by Doug Hampton
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The current therapeutic landscape of hemophilia in the United States is driven by several approved therapies. The hemophilia market is estimated to grow for the period 2019-2032. The major reason behind the hemophilia market upsurge is the launch of the most anticipated gene therapies, which are considered as a threat to the current hemophilia market.

LAS VEGAS, June 21, 2023 /PRNewswire/ — DelveInsight’s Hemophilia Market Insights report includes a comprehensive understanding of current treatment practices, hemophilia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Key Takeaways from the Hemophilia Market Report

  • As per DelveInsight analysis, the hemophilia market is expected to grow positively at a significant CAGR during the study period (2019–2032).
  • According to CDC, The current number of males living in the United States with hemophilia is believed to be between 30,000 and 33,000. In the United States, the estimated prevalence of hemophilia A is 12 cases per 100,000 males and 3.7 cases per 100,000 males for hemophilia B.
  • Globally, leading hemophilia companies such as ApcinteX, ASC Therapeutics, Ultragenix Pharmaceutical, BioMarin Pharmaceutical, CSL Behring, Freeline Therapeutics, Genentech, Inc., Novo Nordisk, Pfizer, Sanofi, Shire, Spark Therapeutics, Amarna therapeutics, Asklepios BioPharmaceutical, Bayer, Belief Biomed, Bioverativ, Catalyst Biosciences, Centessa Pharmaceuticals, Chameleon Biosciences, Chia Tai Tianqing Pharmaceutical Group, Expression Therapeutics, GC Pharma, GeneVentiv, Intellia tx, OPKO Health, Sangamo Therapeutics, Staidson Beijing BioPharmaceuticals, UBI Pharma, uniQure, and others are developing novel hemophilia drugs that can be available in the hemophilia market in the coming years.
  • Some key therapies for hemophilia treatment include ASC618, Valoctocogene Roxaparvovec, Emicizumab, AAV5-hFIXco-Padua (AMT-061), BAX 888, SPK-8011, Fitusiran, BAY2599023, Verbrinacogene setparvovec, PF-07055480, NNC0365-3769 (Mim8) PPX, Nonacog beta pegol, SPK-8016, Concizumab (NN7415), Giroctocogene fitelparvovec (SB-525 or PF07055480), SerpinPC, and others.
  • In February 2023, the US FDA approved ALTUVIIIO (efanesoctocog alfa) factor VIII replacement therapy for adults and children with hemophilia A. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A.
  •  In September 2022, Pfizer and Sangamo Therapeutics announced that the Phase III AFFINE study evaluating giroctocogene fitelparvovec, has re-opened recruitment.
  • In August 2022, EC granted conditional marketing authorization to ROCTAVIANgene therapy for treating severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIIIinhibitors and without detectable antibodies to adeno-associated virus serotype 5. 

Discover which therapies are expected to grab the major hemophilia market share @ Hemophilia Market Report

Hemophilia Overview

Haemophilia is a genetic bleeding disorder that causes blood to clot improperly. This can cause spontaneous as well as post-injury or surgical bleeding. Blood contains several proteins known as clotting factors, which can help prevent bleeding. Patients with hemophilia have low levels of either factor VIII (8) or factor IX (9). The number of factors in a person’s blood defines how severe their hemophilia is. The lower the concentration of the component, the more probable bleeding will occur, which can lead to major health complications. The main causes of hemophilia are hereditary. The symptoms of hemophilia vary based on the clotting factor levels. You may only bleed after surgery or trauma if the clotting factor level is somewhat low. If the insufficiency is severe, a person may bleed for no apparent reason. Clotting factor testing is required to diagnose hemophilia.

Hemophilia Epidemiology Segmentation

The hemophilia epidemiology section provides insights into the historical and current hemophilia patient pool and forecasted trends for individual seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders.

The hemophilia market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:

  • Total Hemophilia Diagnosed Prevalent Pool
  • Hemophilia Type-specific Prevalent Pool
  • Hemophilia Severity-specific Prevalent Pool
  • Hemophilia Inhibitor-specific Prevalent Pool 
  • Hemophilia Treated Prevalent Pool 

Hemophilia Treatment Market 

Currently, the major hemophilia treatment options are Factor Replacement Concentrates, which are derived from either recombinant DNA technology or human plasma, and Bypassing agents. Furthermore, this is an off-label therapeutic option, with Desmopressin Acetate (DDAVP) and Adjunctive treatments being available for the treatment of Hemophilia A. Many patients are given preventive infusions of third-generation products that contain no human or animal proteins. There are several such medications on the hemophilia market, including ADVATE (Takeda) and XYNTHA (Pfizer). Other products have been approved under this category in the past. Since then, short and longer-acting recombinant factor therapies have entered the market, with the most prominent being ELOCTATE and JIVI.

Several recombinant factor VIII (FVIII) preparations with highly specific activities are available in the hemophilia market. Clotting factors produced from plasma are also available. The contemporary market, however, is controlled mostly by recombinants of multiple generations. Although there are various products available at the moment, none of them may be able to entirely cure or control this situation. The future of hemophilia treatment is shifting towards longer half-life medicines as well as more creative techniques such as siRNA, bispecific antibodies, and gene therapy. In terms of future competition, more prolonged half-life factor products are on the way, as are other novel technologies like as gene treatments and bi-specific antibodies.

To know more about hemophilia treatment, visit @ Hemophilia Treatment Drugs 

Key Hemophilia Therapies and Companies

  • ASC618: ASC Therapeutics
  • Valoctocogene Roxaparvovec: BioMarin Pharmaceutical
  • Emicizumab: Genentech, Inc.
  • AAV5-hFIXco-Padua (AMT-061): CSL Behring
  • BAX 888: Shire
  • SPK-8011: Spark Therapeutics
  • Fitusiran: Sanofi
  • BAY2599023: Bayer/Ultragenix Pharmaceutical
  • Verbrinacogene setparvovec: Freeline Therapeutics
  • PF-07055480: Pfizer
  • NNC0365-3769 (Mim8) PPX: Novo Nordisk A/S
  • Nonacog beta pegol: Novo Nordisk A/S
  • SPK-8016: Spark Therapeutics
  • Concizumab (NN7415): Novo Nordisk
  • Giroctocogene fitelparvovec (SB-525 or PF07055480): Pfizer/SangamoTherapeutics
  • SerpinPC: ApcinteX

Learn more about the FDA-approved drugs for hemophilia @ Drugs for Hemophilia Treatment 

Hemophilia Market Dynamics

The dynamics of the hemophilia market are anticipated to change in the coming years owing to the improvement in the rise in the number of healthcare spending across the world. Major Pharma giants are thoroughly working toward the development of new treatment therapies for this indication, in order to provide better relief for the symptoms and hence improve the Quality of life (QoL) of patients with hemophilia A. 

Currently, non-inhibitor drug candidates are driving the hemophilia treatment landscape. Emerging potential candidates pose a negative threat to current hemophilia A pharmaceutical behemoths due to their ability to change the standard of care for hemophilia A patients. The hemophilia treatment landscape is evolving, and multiple companies are working feverishly to develop novel therapies that could potentially cure hemophilia and transform the treatment space. Companies are focusing on the development of gene therapies that produce bispecific antibodies for the treatment of hemophilia A, which will have a favorable impact on the hemophilia market size in the next few years.

However, several factors are impeding the growth of the hemophilia market. Despite the widespread availability of safe and effective replacement therapy, patients with hemophilia A have a significant treatment burden, including breakthrough bleeding, increasing joint damage, and a high rate of inhibitor development. There is still a significant unmet need in the treatment of individuals with hemophilia, as it affects roughly one-third of patients with severe hemophilia A and raises treatment costs and morbidity concerns. Once the inhibitors are formed, they are linked to a decrease in FVIII efficacy in blood coagulation, which negatively impacts patients’ health and quality of life while considerably increasing hemophilia treatment expenditures.

Furthermore, the hemophilia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the hemophilia market growth.

Report Metrics

Details

Study Period

2019–2032

Coverage

7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]

Key Hemophilia Companies

ApcinteX, ASC Therapeutics, Ultragenix Pharmaceutical, BioMarin Pharmaceutical, CSL Behring, Freeline Therapeutics, Genentech, Inc., Novo Nordisk, Pfizer, Sanofi, Shire, Spark Therapeutics, Amarna therapeutics, Asklepios BioPharmaceutical, Bayer, Belief Biomed, Bioverativ, Catalyst Biosciences, Centessa Pharmaceuticals, Chameleon Biosciences, Chia Tai Tianqing Pharmaceutical Group, Expression Therapeutics, GC Pharma, GeneVentiv, Intellia tx, OPKO Health, Sangamo Therapeutics, Staidson Beijing BioPharmaceuticals, UBI Pharma, uniQure, and others

Key Hemophilia Therapies

ASC618, Valoctocogene Roxaparvovec, Emicizumab, AAV5-hFIXco-Padua (AMT-061), BAX 888, SPK-8011, Fitusiran, BAY2599023, Verbrinacogene setparvovec, PF-07055480, NNC0365-3769 (Mim8) PPX, Nonacog beta pegol, SPK-8016, Concizumab (NN7415), Giroctocogene fitelparvovec (SB-525 or PF07055480), SerpinPC, and others

Scope of the Hemophilia Market Report

  • Therapeutic Assessment: Hemophilia current marketed and emerging therapies
  • Hemophilia Market Dynamics: Attribute Analysis of Emerging Hemophilia Drugs
  • Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
  • Unmet Needs, KOL’s Views, Analyst’s Views, Hemophilia Market Access and Reimbursement

Discover more about hemophilia drugs in development @ Hemophilia Clinical Trials

Table of Contents

1.

Hemophilia Market Key Insights

2.

Hemophilia Market Report Introduction

3.

Hemophilia Market Overview at a Glance

4.

Hemophilia Market Executive Summary

5.

Disease Background and Overview

6.

Hemophilia Treatment and Management

7.

Hemophilia Epidemiology and Patient Population

8.

Patient Journey

9.

Hemophilia Marketed Drugs

10.

Hemophilia Emerging Drugs

11.

Seven Major Hemophilia Market Analysis

12.

Hemophilia Market Outlook

13.

Potential of Current and Emerging Therapies

14.

KOL Views

15.

Unmet Needs

16.

SWOT Analysis

17.

Appendix

18.

DelveInsight Capabilities

19.

Disclaimer

20.

About DelveInsight

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SOURCE DelveInsight Business Research, LLP

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