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