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Coagulation Factor Deficiency Market - US to Have the Lion's Share Among the 7MM's | DelveInsight

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News provided by

DelveInsight Business Research, LLP

10 Jul, 2024, 21:31 GMT

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The market for coagulation factor deficiency treatments is experiencing robust growth, driven by advancements in biotechnology and the increasing prevalence of bleeding disorders. Innovations in therapeutic options and improved patient access to healthcare contribute to the expanding coagulation factor deficiency market landscape.

LAS VEGAS, July 10, 2024 /PRNewswire/ -- DelveInsight's Coagulation Factor Deficiency Market Insights report includes a comprehensive understanding of current treatment practices, coagulation factor deficiency emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Takeaways from the Coagulation Factor Deficiency Market Report

  • According to DelveInsight's analysis, the market size for coagulation factor deficiency was found to be USD 13.5 billion in the 7MM in 2023.
  • The total 7MM prevalent cases of coagulation factor deficiency in 2023 were 105K out of which the highest prevalent cases were estimated in the United States.
  • Leading coagulation factor deficiency companies such as Novo Nordisk, Spark Therapeutics, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Biotest AG, Centessa Pharmaceuticals (Apcintex), Staidson Biopharma Inc., Ultragenyx Pharmaceutical, Bayer, and others are developing novel coagulation factor deficiency drugs that can be available in the coagulation factor deficiency market in the coming years.
  • The promising coagulation factor deficiency therapies in the pipeline include Concizumab (NN7415), RG6357 (SPK-8011), Fitusiran (ALN-AT3, SAR-439774), Marstacimab (PF-06741086), NNC0365-3769 A (MIM8), BT524, SERPINPC, STSP-0601, BAY2599023 (DTX201 AAV FVIII), and others.
  • In March 2024, ReciBioPharm signed a collaboration agreement with GeneVentiv Therapeutics, a preclinical gene therapy company, to advance the development of an adeno-associated virus (AAV)-based universal gene therapy for hemophilia, and reportedly the first to treat hemophilia patients with inhibitors.
  • In June 2023, the FDA accepted the company's BLA for fidanacogene elaparvovec for the treatment of adults with hemophilia B. In parallel, the European marketing authorization application (MAA) for fidanacogene elaparvovec has also been accepted and is under review by the European Medicines Agency (EMA).
  • In May 2023, Novo Nordisk notified the National Hemophilia Foundation that they had received a Complete Response Letter (CRL) from the US Food and Drug Administration (FDA) for their investigational, subcutaneous therapy concizumab. While Novo Nordisk had anticipated potential FDA approval of the therapy in the Spring of 2023, the additional information outlined in the CRL will stretch that timeline.

Discover which therapies are expected to grab the major coagulation factor deficiency market share @ Coagulation Factor Deficiency Market Report

Coagulation Factor Deficiency Overview

Coagulation factor deficiency diseases, also referred to as bleeding or clotting disorders, encompass a range of inherited or acquired conditions marked by the absence or malfunction of specific proteins essential for blood clotting. These proteins, known as coagulation factors, are vital for stopping bleeding by forming clots to close injured blood vessels.

There are several coagulation factors, labeled from I to XIII, that function in a complex and coordinated way to achieve hemostasis. Deficiencies in any of these factors can cause abnormal bleeding, varying in severity from mild to severe, depending on the specific factor affected and the extent of the deficiency.

Blood disorders are typically diagnosed by examining a range of symptoms and conducting various blood tests, which differ depending on the specific factor involved. Coagulation factor I deficiency is generally identified through a series of blood tests that measure the amount of fibrinogen in the blood. However, a low fibrinogen level can sometimes indicate liver or kidney disorders. For coagulation factor II, diagnosis is usually made using prothrombin time (PT) and partially activated thromboplastin time (aPTT) tests. Deficiency levels can range from 2% to 50% of normal, with patients at or near 50% usually experiencing normal or no bleeding issues.

Coagulation Factor Deficiency Epidemiology Segmentation

The coagulation factor deficiency epidemiology section provides insights into the historical and current coagulation factor deficiency patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.

The coagulation factor deficiency market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

  • Total Coagulation Factor Deficiency Diagnosed Prevalent Cases
  • Factor-Specific Coagulation Factor Deficiency Prevalent Cases 

Coagulation Factor Deficiency Treatment Market 

The treatment of coagulation factor deficiency typically involves replacing the missing factor in the blood, supplemented by additional therapies when bleeding persists. There are several treatments available for managing coagulation factor deficiencies. For moderate conditions like mucosal tract hemorrhage or heavy menstruation, physicians often prescribe antifibrinolytic drugs and hormones. Discovered after 1950, therapies such as Aminocaproic acid (EACA) and tranexamic acid (TA) help stabilize clots in areas like the mouth, bladder, and uterus. These adjunctive therapies are also beneficial for dental surgeries but are less effective for internal bleeding. These medications can be administered orally or by injection.

ESPEROCT (Turoctocog alfa pegol, also known as N8-GP) is an extended half-life glycopegylated factor VIII molecule used for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding.

This product has been evaluated in five prospective, multi-center clinical trials involving previously treated patients with severe hemophilia A and no history of inhibitors. The results of these trials have shown that the overall safety profile of ESPEROCT is comparable to other long-acting FVIII products, with no safety concerns identified after more than 5 years of clinical exposure.

JIVI (antihemophilic factor [recombinant] PEGylated-aucl), developed by Bayer, is designed for previously treated adults and adolescents (12 years and older) with hemophilia A (congenital Factor VIII deficiency). It is used for:

  • On-demand treatment and control of bleeding episodes
  • Perioperative management of bleeding
  • Routine prophylaxis to reduce the frequency of bleeding episodes.

JIVI functions by replacing the deficient or absent factor VIII (FVIII) in patients with hemophilia A. It is also known as Damoctocog alfa pegol.

To know more about coagulation factor deficiency treatment guidelines, visit @ Coagulation Factor Deficiency Management 

Coagulation Factor Deficiency Pipeline Therapies and Key Companies

  • Concizumab (NN7415): Novo Nordisk
  • RG6357 (SPK-8011): Roche (Spark Therapeutics)
  • Fitusiran (ALN-AT3, SAR-439774): Sanofi (Genzyme)/Alnylam Pharmaceuticals
  • Marstacimab (PF-06741086): Pfizer
  • NNC0365-3769 A (MIM8): Novo Nordisk A/S
  • BT524: Biotest AG
  • SERPINPC: Centessa Pharmaceuticals (Apcintex)
  • STSP-0601: Staidson Biopharma Inc.
  • BAY2599023 (DTX201 AAV FVIII): Bayer/Ultragenyx Pharmaceutical

Discover more about coagulation factor deficiency drugs in development @ Coagulation Factor Deficiency Clinical Trials 

Coagulation Factor Deficiency Market Dynamics

The coagulation factor deficiency market dynamics are expected to change in the coming years. The coagulation factor deficiency market is primarily driven by several key factors. The increasing prevalence of hemophilia and other bleeding disorders is a significant driver, as these conditions require ongoing management and treatment with coagulation factor concentrates. Advances in biotechnology have led to the development of novel therapies, such as recombinant and plasma-derived coagulation factors, which offer improved safety and efficacy profiles, further stimulating market growth. 

Additionally, rising awareness about bleeding disorders and the importance of early diagnosis and treatment has led to increased demand for coagulation factor products. Government initiatives and support for rare disease treatment, along with favorable reimbursement policies, are also contributing to market expansion. Moreover, ongoing research and development activities aimed at discovering new treatments and improving existing ones are expected to create lucrative opportunities in the market.

Furthermore, potential therapies are being investigated for the treatment of coagulation factor deficiency, and it is safe to predict that the treatment space will significantly impact the coagulation factor deficiency market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the coagulation factor deficiency market in the 7MM.

However several factors may impede the growth of the coagulation factor deficiency market. One of the primary challenges is the high cost of treatment, particularly with recombinant and plasma-derived therapies, which limits accessibility for many patients. Additionally, there is a lack of awareness and diagnosis in many regions, particularly in low-income and rural areas, which leads to underdiagnosis and undertreatment of these conditions. 

Regulatory hurdles also pose a significant barrier, as the approval process for new therapies is often lengthy and stringent, delaying the availability of potentially life-saving treatments. Furthermore, the coagulation factor deficiency market is constrained by limited research and development funding, which impedes the discovery of innovative therapies. Lastly, the logistical challenges associated with the storage and distribution of these specialized products, which often require cold chain logistics, add another layer of complexity to coagulation factor deficiency market penetration and access.

Coagulation Factor Deficiency Market Report Metrics

Details

Study Period

2020–2034

Coverage

7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Coagulation Factor Deficiency Market Size

USD 13.5 Billion

Key Coagulation Factor Deficiency Companies

Novo Nordisk, Spark Therapeutics, Sanofi, Alnylam Pharmaceuticals, Pfizer, Biotest AG, Centessa Pharmaceuticals (Apcintex), Staidson Biopharma Inc., Ultragenyx Pharmaceutical, Bayer, and others

Key Pipeline Coagulation Factor Deficiency Therapies

Concizumab (NN7415), RG6357 (SPK-8011), Fitusiran (ALN-AT3, SAR-439774), Marstacimab (PF-06741086), NNC0365-3769 A (MIM8), BT524, SERPINPC, STSP-0601, BAY2599023 (DTX201 AAV FVIII), and others

Scope of the Coagulation Factor Deficiency Market Report

  • Therapeutic Assessment: Coagulation Factor Deficiency current marketed and emerging therapies
  • Coagulation Factor Deficiency Market Dynamics: Key Market Forecast Assumptions of Emerging Coagulation Factor Deficiency Drugs and Market Outlook
  • Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
  • Unmet Needs, KOL's views, Analyst's views, Coagulation Factor Deficiency Market Access and Reimbursement

Download the report to understand which factors are driving coagulation factor deficiency market trends @ Coagulation Factor Deficiency Market Trends

Table of Contents

1

KEY INSIGHTS

2

REPORT INTRODUCTION

3

COAGULATION FACTOR DEFICIENCY MARKET OVERVIEW AT A GLANCE

3.1

MARKET SHARE (%) DISTRIBUTION OF COAGULATION FACTOR DEFICIENCY IN 2020

3.2

MARKET SHARE (%) DISTRIBUTION OF COAGULATION FACTOR DEFICIENCY IN 2034

4

EXECUTIVE SUMMARY OF COAGULATION FACTOR DEFICIENCY

5

KEY EVENTS

6

EPIDEMIOLOGY AND MARKET METHODOLOGY

7

COAGULATION FACTOR DEFICIENCY DISEASE – OVERVIEW

7.1

INTRODUCTION

7.2

PROCESS OF CLOTTING

7.3

TYPE OF CLOTTING FACTORS AND THEIR FUNCTIONS

7.4

MECHANISM

7.5

TYPE OF COAGULATION FACTOR DEFICIENCY

7.5.1

Coagulation Factor I Deficiency (Fibrinogen)

7.5.1.1

Afibrinogenemia

7.5.1.2

Hypofibrinogenemia

7.5.1.3

Dysfibrinogeneima

7.5.1.4

Hypodysfibrinogenemia

7.5.2

Coagulation Factor II Deficiency (Prothrombin)

7.5.3

Coagulation Factor V Deficiency

7.5.4

Coagulation Factor VII Deficiency

7.5.5

Coagulation Factor VIII Deficiency (Hemophilia A)

7.5.6

Coagulation Factor IX Deficiency (Hemophilia B)

7.5.7

Coagulation Factor X Deficiency

7.5.8

Coagulation Factor XI Deficiency (Hemophilia C)

7.5.9

Coagulation Factor XII Deficiency

7.5.10

Coagulation Factor XIII Deficiency

7.5.11

Von Willebrand Disease

7.6

SYMPTOMS ASSOCIATED WITH DEFICIENCY OF COAGULATION FACTORS

7.7

CAUSES OF COAGULATION FACTOR DEFICIENCY DISEASE

7.7.1

Inherited (Genetic) Causes:

7.7.2

Acquired Causes:

7.8

TESTING

7.9

DIAGNOSIS

7.9.1

Diagnosis of Hemophilia A

7.9.1.1

Establishing the Diagnosis

7.9.1.2

Molecular Genetic Testing

7.9.1.3

Screening Tests

7.9.1.4

Clotting Factor Tests

7.9.1.5

Inhibitor Testing

7.9.2

Diagnosis of Hemophilia B

7.9.3

Diagnosis of Von Willebrand disease

7.1

TREATMENT AND MANAGEMENT

7.10.1

Treatment

7.10.1.1

Non replacement therapies

7.10.1.2

Replacement therapies

7.10.2

Management

7.10.2.1

Healthy lifestyle

7.10.2.2

Dental care

7.10.2.3

Vaccinations

7.10.2.4

Disease management in the case of females

8

EPIDEMIOLOGY AND PATIENT POPULATION

8.1

ASSUMPTIONS AND RATIONALE: 7MM

8.2

KEY FINDINGS

8.3

Total Diagnosed Prevalence of Coagulation Factor Deficiency in the 7mm

8.4

Factor-Specific Prevalence of Coagulation Factor Deficiency in the 7mm

8.5

THE UNITED STATES

8.5.1

Total Diagnosed Prevalence of Coagulation Factor Deficiency in the United States

8.5.2

Factor-specific Prevalence of Coagulation Factor Deficiency in the United States

8.6

EU4 AND THE UK

8.6.1

Total Diagnosed Prevalence of Coagulation Factor Deficiency in the EU4 and the UK

8.6.2

Factor-specific Prevalence of Coagulation Factor Deficiency in the EU4 and the UK

8.7

JAPAN

8.7.1

Total Diagnosed Prevalence of Coagulation Factor Deficiency in the Japan

8.7.2

Factor-specific Prevalence of Coagulation Factor Deficiency in the Japan

9

PATIENT JOURNEY

10

MARKETED THERAPIES

10.1

KEY CROSS

10.2

ESPEROCT (N8-GP; TUROCTOCOG ALFA PEGOL): NOVO NORDISK

10.2.1

Product Description

10.2.2

Regulatory Milestones

10.2.3

Other Developmental Activities

10.2.4

RecentClinical Development

10.2.4.1

Clinical Trials Information

10.2.5

Safety and efficacy

10.2.5.1

Summary of Pivotal Trials

10.2.6

Product Profile

10.3

JIVI (FORMERLY BAY94-9027): BAYER

10.3.1

Product Description

10.3.2

Regulatory Milestones

10.3.3

Other Developmental Activities

10.3.4

Recent Clinical Development

10.3.4.1

Clinical Trials Information

10.3.5

Safety and efficacy

10.3.5.1

Summary of Pivotal Trials

10.3.6

Product Profile

10.4

WILATE: OCTAPHARMA

10.4.1

Product Description

10.4.2

Regulatory Milestones

10.4.3

Recent Clinical Development

10.4.3.1

Clinical Trials Information

10.4.4

Safety and Efficacy

10.4.4.1

Summary of Pivotal Trials

10.4.5

Product Profile

10.5

ADYNOVATE (ADYNOVI; BAX 855): TAKEDA

10.5.1

Product Description

10.5.2

Regulatory Milestones

10.5.3

Other Developmental Activities

10.5.4

Recnt Clinical Development

10.5.4.1

Clinical Trials Information

10.5.5

Safety and efficacy

10.5.5.1

Summary of Pivotal Trials

10.5.6

Product Profile

10.6

ELOCTATE [ELOCTA (EFMOROCTOCOG ALFA)]: SANOFI/SOBI

10.6.1

Product Description

10.6.2

Regulatory Milestones

10.6.3

Other Developmental Activities

10.6.4

Safety and efficacy

10.6.4.1

Summary of Pivotal Trials

10.6.5

Product Profile

10.7

AFSTYLA (LONOCTOCOG ALFA): CSL BEHRING

10.7.1

Product Description

10.7.2

Regulatory Milestones

10.7.3

Other Developmental Activities

10.7.4

Safety and efficacy

10.7.4.1

Summary of Pivotal Trials

10.7.5

Product Profile

10.8

NUWIQ (SIMOCTOCOG ALFA): OCTAPHARMA

10.8.1

Product Description

10.8.2

Regulatory Milestone

10.8.3

Other Developmental Activities

10.8.4

Safety and Efficacy

1.1.1.1

Summary of Pivotal Clinical trial

10.8.5

Product Profile

10.9

KOVALTRY (BAY 81-8973): BAYER

10.9.1

Product Description

10.9.2

Regulatory Milestone

10.9.3

Other Developmental Activity

10.9.4

Safety and Efficacy

10.9.4.1

Summary of Pivotal Clinical Trial

10.9.5

Product Profile

10.10

OBIZUR: TAKEDA

10.10.1

Product Description

10.10.2

Regulatory Milestones

10.10.3

Other Developmental Activities

10.10.4

Recent Clinical Development

10.10.4.1

Clinical trials information

10.10.5

Safety and Efficacy

10.10.5.1

Summary of Pivotal Trials

10.10.6

Product Profile

10.11

KOGENATE FS (OCTOCOG ALFA): BAYER

10.11.1

Product Description

10.11.2

Regulatory Milestones

10.11.3

Other Developmental Activities

10.11.4

Safety and Efficacy

10.11.4.1

Summary of Pivotal Trials

10.11.5

Product Profile

10.12

XYNTHA (REFACTO AF): PFIZER

10.12.1

Product Description

10.12.2

Regulatory Milestones

10.12.3

Other Developmental Activities

10.12.4

Safety and Efficacy

10.12.4.1

Summary of Pivotal Trials

10.12.5

Product Profile

10.13

FEIBA: TAKEDA

10.13.1

Product Description

10.13.2

Regulatory Milestones

10.13.3

Other Developmental Activities

10.13.4

Recent Clinical Developmen

10.13.4.1

Clinical trials information

10.13.5

Safety and Efficacy

10.13.5.1

Summary of Pivotal Trials

10.13.6

Product Profile

10.14

HEMLIBRA (EMICIZUMAB-KXWH): CHUGAI/ GENENTECH/ROCHE

10.14.1

Product Description

10.14.2

Regulatory Milestones

10.14.3

Other Developmental Activities

10.15

SEVENFACT [COAGULATION FACTOR VIIA (RECOMBINANT)-JNCW]: HEMA BIOLOGICS/LFB PHARMACEUTICALS

10.15.1

Product Description

10.15.2

Regulatory Milestones

10.15.3

Other Developmental Activities

10.15.4

Recent Clinical Development

10.15.4.1

Clinical trials information

10.15.5

Safety and Efficacy

10.15.5.1

Summary of Pivotal Trial

10.15.6

Product Profile

10.16

HEMGENIX (ETRANACOGENE DEZAPARVOVEC): CSL BEHRING/UNIQURE

10.16.1

Product Description

10.16.2

Regulatory Approval

10.16.3

Other Development Activities

10.16.4

Recent Clinical Development

10.16.5

Safety and Efficacy

10.16.6

Product Profile

10.17

REBINYN (NONACOG BETA PEGOL): NOVO NORDISK

10.17.1

Product Description

10.17.2

Regulatory Milestones

10.17.3

Other Developmental Activities

10.17.4

Recent Clinical Development

10.17.4.1

Clinical Trials Information

10.17.5

Safety and Efficacy

10.17.5.1

Summary of Pivotal Trials

10.17.6

Product Profile

10.18

IDELVION: CSL BEHRING

10.18.1

Product Description

10.18.2

Regulatory Milestones

10.18.3

Other Developmental Activities

10.18.4

Safety and efficacy

10.18.4.1

Summary of Pivotal Trial

10.18.5

Product Profile

10.19

ALPROLIX: SANOFI /BIOVERATIV THERAPEUTICS/SOBI

10.19.1

Product Description

10.19.2

Regulatory Milestones

10.19.3

Other Developmental Activities

10.19.4

Safety and Efficacy

10.19.4.1

Summary of Pivotal Trial

10.19.5

Product Profile

10.2

IXINITY (TRENONACOG ALFA): MEDEXUS PHARMACEUTICALS/APTEVO THERAPEUTICS

10.20.1

Product Description

10.20.2

Regulatory Milestones

10.20.3

Other Developmental Activities

10.20.4

Safety and Efficacy

10.20.4.1

Summary of Pivotal Trials

10.20.5

Product Profile

10.21

RIXUBIS: TAKEDA (SHIRE/BAXTER)

10.21.1

Product Description

10.21.2

Regulatory Milestones

10.21.3

Other Developmental Activities

10.21.4

Safety and Efficacy

10.21.4.1

Summary of Pivotal Trial

10.21.5

Product Profile

10.22

VONVENDI: SHIRE

10.22.1

Product Description

10.22.2

Mechanism of Action

10.22.3

Regulatory Milestones

10.22.4

Advantages and Disadvantages

10.22.5

Recent Clinical Development

10.22.5.1

Clinical Trials Information

10.22.6

Safety and Efficacy

10.22.7

Product Profile

10.23

HUMATE-P/HAEMATE P: CSL BEHRING

10.23.1

Product Description

10.23.2

Mechanism of Action

10.23.3

Regulatory Milestones

10.23.4

Advantages and Disadvantages

10.23.5

Safety and Efficacy

10.23.6

Product Profile

10.24

ALPHANATE: GRIFOLS BIOLOGICAL INC.

10.24.1

Product Description

10.24.2

Mechanism of Action

10.24.3

Regulatory Milestones

10.24.4

Advantages and Disadvantages

10.24.5

Safety and Efficacy

10.24.6

Product Profile

10.25

COAGADEX: BIO PRODUCTS LABORATORY

10.25.1

Product Description

10.25.2

Regulatory Milestones

10.25.3

Other Developmental Activities

10.25.4

Pivotal Clinical Trial

10.25.4.1

Summary of Pivotal Clinical Trials

10.25.5

Safety and Efficacy

10.25.6

Product Profile

10.26

ANDEXXA: ALEXION ASTRAZENECA RARE DISEASE

10.26.1

Product Description

10.26.2

Regulatory Milestones

10.26.3

Other Developmental Activities

10.26.4

Pivotal Clinical Trial

1.1.1.2

Summary of Pivotal Clinical Trials

10.26.5

Safety and Efficacy

10.26.6

Product Profile

10.27

KCENTRA: CSL BEHRING

10.27.1

Product Description

10.27.2

Regulatory Milestones

10.27.3

Other Developmental Activities

10.27.4

Ongoing Current Clinical Pipeline Activity

10.27.5

Pivotal Clinical Trial

10.27.5.1

Summary of Pivotal Clinical Trials

10.27.6

Safety and Efficacy

10.27.7

Product Profile

10.28

TRETTEN® (CATRIDECACOG): NOVONORDISK

10.28.1

Product Description

10.28.2

Regulatory Milestones

10.28.3

Pivotal Clinical Trial

10.28.4

Safety and Efficacy

10.28.5

Product Profile

10.29

OCTAPLEX: OCTAPHARMA

10.29.1

Product Description

10.29.2

Regulatory Milestones

10.29.3

Other Developmental Activities

10.29.4

Recent Clinical Development

10.29.5

Pivotal Clinical Trial

10.29.6

Safety and Efficacy

10.29.7

Product Profile

10.3

RIASTAP: CSL BEHRING

10.30.1

Product Description

10.30.2

Regulatory Milestones

10.30.3

Other Developmental Activities

10.30.4

Pivotal Clinical Trial

10.30.5

Safety and Efficacy

10.30.6

Product Profile

10.31

ROCTAVIAN (VALOCTOCOGENE ROXAPARVOVEC): BIOMARIN PHARMACEUTICAL

10.31.1

Product Description

10.31.2

Regulatory Milestone

10.31.3

Other developmental activities

10.31.4

Recent clinical developmental activities

10.31.5

Safety and efficacy

10.31.6

Product Profile

10.32

ALTUVIIIO (EFANESOCTOCOG ALFA) (RFVIIIFC-VWF-XTEN): SANOFI

10.32.1

Product Description

10.32.2

Regulatory Milestone

10.32.3

Other developmental activity

10.32.4

Recent Clinical development

10.32.4.1

Clinical trial information

10.32.5

Safety and efficacy

10.32.6

Product Profile

11

EMERGING DRUGS

11.1

KEY CROSS

11.2

CONCIZUMAB (NN7415): NOVO NORDISK

11.2.1

Product description

11.2.2

Other developmental activities

11.2.3

Clinical development Activities

11.2.3.1

Clinical trial information

11.2.4

Safety and efficacy

11.3

FIDANACOGENE ELAPARVOVEC: PFIZER/SPARK THERAPEUTICS

11.3.1

Product Description

11.3.2

Other Developmental Activities

11.3.3

Clinical Developmental Activities

11.3.3.1

Clinical Trials Information

11.3.4

Safety and Efficacy

11.4

RG6357 (SPK-8011): ROCHE (SPARK THERAPEUTICS)

11.4.1

Product description

11.4.2

Other developmental activity

11.4.3

Clinical development Activities

11.4.3.1

Clinical trial information

11.4.4

Safety and efficacy

11.5

FITUSIRAN (ALN-AT3, SAR-439774): SANOFI (GENZYME)/ALNYLAM PHARMACEUTICALS

11.5.1

Product description

11.5.2

Other developmental activity

11.5.3

Clinical development Activities

11.5.3.1

Clinical trial information

11.5.4

Safety and efficacy

11.6

MARSTACIMAB (PF-06741086): PFIZER

11.6.1

Product description

11.6.2

Other developmental activities

11.6.3

Clinical development Activities

11.6.3.1

Clinical trial information

11.6.4

Safety and efficacy

11.7

GIROCTOCOGENE FITELPARVOVEC (SB-525 OR PF-07055480): PFIZER/SANGAMO THERAPEUTICS

11.7.1

Product description

11.7.2

Other developmental activity

11.7.3

Clinical development Actvities

11.7.3.1

Clinical trial information

11.7.4

Safety and efficacy

11.8

NNC0365-3769 A (MIM8): NOVO NORDISK A/S

11.8.1

Product description

11.8.2

Other developmental activity

11.8.3

Clinical development Activities

11.8.3.1

Clinical trial information

11.8.4

Safety and efficacy

11.9

BT524: BIOTEST AG

11.9.1

Product Description

11.9.2

Other Developmental Activities

11.9.3

Clinical Development Activities

11.9.3.1

Clinical Trials Information

11.9.4

Safety and Efficacy

11.10

SERPINPC: CENTESSA PHARMACEUTICALS (APCINTEX)

11.10.1

Product description

11.10.2

Other developmental activities

11.10.3

Clinical development Activities

11.10.3.1

Clinical trial information

11.10.4

Safety and efficacy

11.11

STSP-0601: STAIDSON BIOPHARMA INC.

11.11.1

Product Description

11.11.2

Clinical Development Activities

11.11.2.1

Clinical Trials Information

11.11.3

Safety and Efficacy

11.12

MARZEPTACOG ALFA: GC BIOPHARMA

11.12.1

Product Description

11.12.2

Other Developmental Activities

11.12.3

Clinical Development Activities

11.12.3.1

Clinical Trials Information

11.12.4

Safety and Efficacy

11.13

AB023: ARONORA, INC.

11.13.1

Product Description

11.13.2

Other Developmental Activities

11.13.3

Clinical Development Activities

11.13.3.1

Clinical Trials Information

11.13.4

Safety and Efficacy

11.14

BAY2599023 (DTX201 AAV FVIII): BAYER/ULTRAGENYX PHARMACEUTICAL

11.14.1

Product description

11.14.2

Other developmental activity

11.14.3

Clinical development Activities

11.14.3.1

Clinical trial information

11.14.4

Safety and efficacy

11.15

OPK88005 (FACTOR VIIA-CTP): OPKO HEALTH, INC.

11.15.1

Product Description

11.15.2

Other Developmental Activities

11.15.3

Clinical Development Activities

11.15.3.1

Clinical Trials Information

11.15.4

Safety and Efficacy

12

COAGULATION FACTOR DEFICIENCY: SEVEN MAJOR MARKET ANALYSIS

12.1

KEY FINDINGS

12.2

MARKET OUTLOOK

12.3

KEY MARKET FORECAST ASSUMPTIONS

12.4

TOTAL MARKET SIZE OF COAGULATION FACTOR DEFICIENCY IN THE 7MM

12.5

UNITED STATES MARKET SIZE

12.5.1

Total Market Size of Coagulation Factor Deficiency in the United States

12.6

EU4 AND THE UK MARKET SIZE

12.6.1

Total Market Size of Coagulation Factor Deficiency in the EU4 and the UK

12.7

JAPAN

12.7.1

Total Market Size of Coagulation Factor Deficiency in Japan

13

MARKET ACCESS AND REIMBURSEMENT

13.1

HEMOPHILIA A

13.1.1

The US

13.1.1.1

Current Therapies

13.1.2

HAS Assessment for Some Other Therapies

13.1.3

HTA Assessment of Hemlibra by NHS and IQWiG

13.1.4

IQWIG Assessment for Some Other Therapies

13.1.5

Future Therapies: Gene Therapy

13.2

HEMOPHILIA B

13.2.1

Access and Reimbursement Overview for Current and Future Therapies

13.2.2

NICE Assessment

13.2.3

IQWIG Assessment

13.2.4

HAS Assessment

13.3

CLOTTING FACTORS

13.3.1

The US

13.3.1.1

CMS

13.3.2

UK (NHS)

13.3.3

EU (HTA Assessment)

13.3.3.1

IQWIG

13.3.3.2

AIFA

13.3.4

Japan

14

UNMET NEEDS

15

SWOT ANALYSIS

16

KOL VIEWS

17

APPENDIX

17.1

BIBLIOGRAPHY

17.2

REPORT METHODOLOGY

18

DELVEINSIGHT CAPABILITIES

19

DISCLAIMER

20

ABOUT DELVEINSIGHT

Related Reports

Coagulation Factor Deficiency Epidemiology Forecast

Coagulation Factor Deficiency Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the coagulation factor deficiency epidemiology trends.

Coagulation Factor Deficiency Pipeline

Coagulation Factor Deficiency Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key coagulation factor deficiency companies, including SymBio Pharmaceuticals, AlloVir, among others.

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Hemophilia Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia companies, including 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, among others.

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Hemophilia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Hemophilia companies, including Intellia tx, Amarna therapeutics, Expressi Ontherapeutics, GC Pharma, Chameleon Biosciences, Pfizer, UBI Pharma, GeneVentiv, Chia Tai Tianqing Pharmaceutical Group, Bayer, ASC Therapeutics, Catalyst Biosciences, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, CSL Behring,  Sanofi, Novo Nordisk, Centessa Pharmaceuticals, OPKO Health, among others.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

Contact Us
Shruti Thakur  
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