PharmaPoint: Hemophilia A and B Recombinant Factor Replacement Therapy – Global Drug Forecast and Market Analysis to 2024

Pages: 333 Published: December 01, 2015 Report Code: GDHC116PIDR

  • Overview
  • Key Players
  • Contents
  • Tables
  • Figures
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    iSpeech

Congenital hemophilia A and B are bleeding disorders that are caused by genetic X-linked deficiencies in the blood clotting Factors VIII and IX, respectively. Hemophilia manifests as mild, moderate, or severe inability to form clots, depending upon the endogenous levels of circulating factor in an individual patient. The mainstay of treatment for hemophilia is to replace the missing FVIII or FIX, produced using plasma-derived or recombinant methods. Some patients develop inhibitors to FVIII or FIX treatment and need a bypassing agent, such as FVIIa, to resolve bleeds. Patients of hemophilia A or B often require lifelong treatment to prevent and control bleeding episodes, and this therefore represents a lucrative sales opportunity.

GlobalData estimates the 2014 sales for recombinant hemophilia A and B markets at approximately $5.38 billion across the 7MM+A covered in this report. The market is expected to reach $6.25 billion in 2024 at a CAGR of 1.52%. This growth will be mainly driven by an increasing use of routine prophylaxis and a greater use of recombinant products in place of plasma-derived therapies.

The hemophilia recombinant treatment pipeline is strong, and crowded with multiple long-acting recombinant clotting factors; however, GlobalData expects a lack of initiative for hemophilia A patients to switch to long-acting rFVIII, and a limited scope for premium pricing for new agents, restraining the market growth. Overall, Baxalta is expected to remain as the market leader in the hemophilia space throughout the forecast period, dominating the market with its current gold standard therapy for hemophilia A, Advate, supported by the launch its of pegylated rFVIII, Adynovate.

Scope

Overview of hemophilia, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.

Annualized hemophilia recombinant therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in six patient segments (including on-demand, prophylactic, and inhibitor), forecast from 2014 to 2024.

Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the hemophilia recombinant therapeutics market

Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for different lines of therapy. The most promising candidate in Phase III development is profiled.

Analysis of the current and future market competition in the global hemophilia recombinant therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Key Highlights

What will be the impact of the new long-acting rFVIII and rFIX products in the hemophilia market sales? Led by Biogen’s Eloctate and Alprolix, moderate and high rates of adoption of the long-acting rFVIII and rFIX therapies, respectively, are expected.

With the growing importance of long-acting Factors, what are the strategies of the companies that have short-acting assets to remain competitive? What are the other approaches that new entrants employ?

Prevention of inhibitor formation remains as a major unmet need in the hemophilia space. What are the other unmet needs and opportunities in the hemophilia market?

Key Players

Alnylam

Baxalta (Baxter)

Bayer

Biogen

CSL Behring

Emergent BioSolutions

Green Cross

LFB (rEVO Biologics)

Novo Nordisk

Octapharma

Pfizer

Roche/Chugai

Spark Therapeutics

uniQure

Table of Contents

1Table of Contents

1.1List of Tables

1.2List of Figures

2Introduction

2.1Catalyst

2.2Related Reports

2.3Upcoming Related Reports

3Disease Overview

3.1Etiology and Pathophysiology

3.1.1Etiology

3.1.2Pathophysiology

3.1.3Prognosis and Quality of Life

3.2Symptoms

3.2.1Hemophilia A and B

3.2.2Inhibitors

4Epidemiology

4.1Disease Background

4.2Risk Factors and Comorbidities

4.3Global Trends

4.4Forecast Methodology

4.4.1Sources Used

4.4.2Sources Not Used

4.4.3Forecast Assumptions and Methods

4.5Epidemiological Forecast for Hemophilia A (2014–2024)

4.5.1Diagnosed Prevalent Cases of Hemophilia A

4.5.2Diagnosed Prevalent Cases of Hemophilia A by Age

4.5.3Diagnosed Prevalent Cases of Hemophilia A by Sex

4.5.4Age-Standardized Diagnosed Prevalence of Hemophilia A

4.6Epidemiological Forecast for Hemophilia B (2014–2024)

4.6.1Diagnosed Prevalent Cases of Hemophilia B

4.6.2Diagnosed Prevalent Cases of Hemophilia B by Age

4.6.3Diagnosed Prevalent Cases of Hemophilia B by Sex

4.6.4Age-Standardized Diagnosed Prevalence of Hemophilia B

4.7Epidemiological Forecast for Hemophilia A and Hemophilia B (2014–2024)

4.7.1Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

4.7.2Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Sex

4.8Discussion

4.8.1Conclusions on Epidemiological Trends

4.8.2Limitations of the Analysis

4.8.3Strengths of the Analysis

5Disease Management

5.1Treatment Overview

5.1.1Hemophilia A and B Diagnosis

5.1.2Hemophilia A and B Treatment

5.1.3Diagnosis of Inhibitors

5.1.4Treatment of Inhibitors

5.2US

5.2.1Clinical Practice

5.3France

5.3.1Clinical Practice

5.4Germany

5.4.1Clinical Practice

5.5Italy

5.5.1Clinical Practice

5.6Spain

5.6.1Clinical Practice

5.7UK

5.7.1Clinical Practice

5.8Japan

5.8.1Clinical Practice

5.9Argentina

5.9.1Clinical Practice

6Competitive Assessment

6.1Overview

6.2Strategic Competitive Assessment

6.3Product Profiles – Hemophilia A

6.3.1Advate (Octocog Alfa)

6.3.2Kogenate FS/Helixate FS (Octocog Alfa)

6.3.3Xyntha/ReFacto AF (Moroctocog Alfa)

6.3.4Recombinate (Octocog Alfa)

6.3.5Eloctate (Efmoroctocog Alfa)

6.3.6NovoEight (Turoctocog Alfa)

6.3.7Nuwiq (Simoctocog Alfa)

6.4Product Profiles – Hemophilia B

6.4.1BeneFIX (Nonacog Alfa)

6.4.2Alprolix (Eftrenonacog Alfa)

6.4.3Rixubis (Nonacog Gamma)

6.4.4IXinity (Trenonacog Alfa)

6.5Product Profiles – Hemophilia A and B with Inhibitors

6.5.1NovoSeven RT (Eptacog Alfa)

7Opportunity and Unmet Need

7.1Overview

7.2Opportunity Analysis

7.2.1Longer-Lasting Recombinant Factors

7.2.2Lower-Cost Recombinant Replacement Therapies

7.2.3Lower Risk of Inhibitor Formation in Previously Untreated Patients

7.2.4More Convenient Drug Administration

7.3Unmet Needs Gap Analysis

7.3.1Long-Acting FVIII Replacement Therapies

7.3.2Gene Therapy Treatments for Hemophilia A and B

8Pipeline Assessment

8.1Overview

8.2Late-Stage Development Candidates – Hemophilia A

8.2.1Kovaltry (Octocog Alfa; BAY 81-8973)

8.2.2Adynovate (BAX-855)

8.2.3BAY 94-9027 (Damoctocog Alfa Pegol)

8.2.4N8-GP (Turoctocog Alfa Pegol)

8.2.5CSL-627 (rFVIII-SC)

8.2.6GreenGene F (Beroctocog Alfa; GC1101C)

8.3Late-Stage Development Candidates – Hemophilia B

8.3.1CSL-654 (rIX-FP; Albutrepenonacog Alfa)

8.3.2N9-GP (NN-7999; Nonacog Beta Pegol)

8.4Late-Stage Development Candidates – Hemophilia A and B Bypassing Agents (Patients with Inhibitors)

8.4.1BAX-817 (rFVIIa BI)

8.4.2LR-769 (rhFVIIa)

8.4.3CSL-689 (rVIIa-FP)

8.5Promising Drugs in Early-Stage Development

8.5.1Alternative Coagulation Promoters

8.5.2Gene Therapies

9Current and Future Players

9.1Overview

9.2Trends in Corporate Strategy

9.3Company Profiles

9.3.1Baxalta

9.3.2Bayer

9.3.3Biogen

9.3.4CSL Behring

9.3.5Novo Nordisk

9.3.6Pfizer

10Market Outlook

10.1Global Markets

10.1.1Forecast

10.1.2Drivers and Barriers – Global Market

10.2US

10.2.1Forecast

10.2.2Key Events

10.2.3Drivers and Barriers

10.35EU

10.3.1Forecast

10.3.2Key Events

10.3.3Drivers and Barriers – France

10.3.4Drivers and Barriers – Germany

10.3.5Drivers and Barriers – Italy

10.3.6Drivers and Barriers – Spain

10.3.7Drivers and Barriers – UK

10.4Japan

10.4.1Forecast

10.4.2Key Events

10.4.3Drivers and Barriers

10.5Argentina

10.5.1Forecast

10.5.2Key Events

10.5.3Drivers and Barriers

11Appendix

11.1Bibliography

11.2Abbreviations

11.3Methodology

11.4Forecasting Methodology

11.4.1Diagnosed Hemophilia Patients

11.4.2Percent Drug-Treated Patients

11.4.3Drugs Included in Each Therapeutic Class

11.4.4Launch Dates

11.4.5General Pricing Assumptions

11.4.6Individual Drug Assumptions

11.4.7Pricing of Pipeline Agents

11.5Primary Research — KOLs Interviewed for this Report

11.6Primary Research — Prescriber Survey

11.7Real-World Data

11.8About the Authors

11.8.1Analyst

11.8.2Therapy Area Director

11.8.3Epidemiologists

11.8.4Global Head of Healthcare

11.9About GlobalData

11.10Contact Us

11.11Disclaimer

List of Tables

Table 1: Symptoms of Hemophilia A and B

Table 2: Relationship of Bleeding Severity with Clotting Factor Level

Table 3: Hemophilia – Risk Factors and Comorbidities

Table 4: 9MM, Diagnosed Prevalence of Hemophilia A (per 100,000 Population), Both Sexes, All Ages, 2004–2013

Table 5: 9MM, Diagnosed Prevalence of Hemophilia B (per 100,000 Population), Both Sexes, All Ages, 2004–2013

Table 6: 9MM, Sources and Diagnosed Prevalence Data for Hemophilia A and B

Table 7: 9MM, Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, All Ages, N, 2014–2024

Table 8: 9MM, Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, by Age, N, Row (%), 2014

Table 9: 9MM, Diagnosed Prevalent Cases of Hemophilia A, by Sex, All Ages, N, Row (%), 2014

Table 10: 9MM, Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, All Ages, N, 2014–2024

Table 11: 9MM, Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, by Age, N, Row (%), 2014

Table 12: 9MM, Diagnosed Prevalent Cases of Hemophilia B, by Sex, All Ages, N, Row (%), 2014

Table 13: 9MM, Diagnosed Prevalent Cases of Hemophilia, Both Sexes, All Ages, N, 2014–2024

Table 14: 9MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, by Sex, All Ages, N, Row (%), 2014

Table 15: Most Prescribed Hemophilia A and B Drugs in the Global Markets, 2015

Table 16: Country Profile — US, 2014

Table 17: Country Profile — France, 2014

Table 18: Country Profile — Germany, 2014

Table 19: Country Profile — Italy, 2014

Table 20: Country Profile — Spain, 2014

Table 21: Country Profile — UK, 2014

Table 22: Country Profile — Japan, 2014

Table 23: Country Profile — Argentina, 2014

Table 24: Leading Recombinant Replacement Therapies for Hemophilia A and B and Patients with Inhibitors, 2015

Table 25: Product Profile – Advate

Table 26: Advate SWOT Analysis, 2015

Table 27: Global Sales Forecast ($m) for Advate, 2014–2024

Table 28: Product Profile – Kogenate FS/Helixate FS

Table 29: Kogenate FS/Helixate FS SWOT Analysis, 2015

Table 30: Global Sales Forecast ($m) for Kogenate FS/Kogenate Bayer, 2014–2024

Table 31: Global Sales Forecast ($m) for Helixate FS/Helixate NexGen, 2014–2024

Table 32: Product Profile – Xyntha/ReFacto AF

Table 33: Xyntha/ReFacto AF SWOT Analysis, 2015

Table 34: Global Sales Forecast ($m) for Xyntha/ReFacto AF, 2014–2024

Table 35: Product Profile – Recombinate

Table 36: Recombinate SWOT Analysis, 2015

Table 37: Global Sales Forecast ($m) for Recombinate, 2014–2024

Table 38: Product Profile – Eloctate

Table 39: Eloctate SWOT Analysis, 2015

Table 40: Global Sales Forecast ($m) for Eloctate, 2014–2024

Table 41: Product Profile – NovoEight

Table 42: NovoEight SWOT Analysis, 2015

Table 43: Global Sales Forecast ($m) for NovoEight, 2014–2024

Table 44: Product Profile – Nuwiq

Table 45: Nuwiq SWOT Analysis, 2015

Table 46: Global Sales Forecast ($m) for Nuwiq, 2014–2024

Table 47: Product Profile – BeneFIX

Table 48: BeneFIX SWOT Analysis, 2015

Table 49: Global Sales Forecast ($m) for BeneFIX, 2014–2024

Table 50: Product Profile – Alprolix

Table 51: Alprolix SWOT Analysis, 2015

Table 52: Global Sales Forecast ($m) for Alprolix, 2014–2024

Table 53: Product Profile – Rixubis

Table 54: Rixubis SWOT Analysis, 2015

Table 55: Global Sales Forecast ($m) for Rixubis, 2014–2024

Table 56: Product Profile – IXinity

Table 57: IXinity SWOT Analysis, 2015

Table 58: Global Sales Forecast ($m) for IXinity, 2014–2024

Table 59: Product Profile – NovoSeven RT

Table 60: NovoSeven RT SWOT Analysis, 2015

Table 61:Global Sales Forecast ($m) for NovoSeven RT, 2014–2024

Table 62: Overall Unmet Needs – Current Level of Attainment

Table 63: Hemophilia A – Phase III Pipeline, 2015

Table 64: Product Profile – Kovaltry

Table 65: Kovaltry SWOT Analysis, 2015

Table 66: Global Sales Forecast ($m) for Kovaltry, 2014–2024

Table 67: Product Profile – Adynovate

Table 68: Adynovate SWOT Analysis, 2015

Table 69: Global Sales Forecast ($m) for Adynovate, 2014–2024

Table 70: Product Profile – BAY 94-9027

Table 71: Efficacy of BAY 94-9027 in the PROTECT VIII study

Table 72: BAY 94-9027 SWOT Analysis, 2015

Table 73: Global Sales Forecast ($m) for BAY 94-9027, 2014–2024

Table 74: Product Profile – N8-GP

Table 75: N8-GP SWOT Analysis, 2015

Table 76: Global Sales Forecast ($m) for N8-GP, 2014–2024

Table 77: Product Profile – CSL-627

Table 78: CSL-627 SWOT Analysis, 2015

Table 79: Global Sales Forecast ($m) for CSL-627, 2014–2024

Table 80: Product Profile – GreenGene F

Table 81: GreenGene F SWOT Analysis, 2015

Table 82: Global Sales Forecast ($m) for GreenGene F, 2014–2024

Table 83: Hemophilia B – Phase III Pipeline, 2015

Table 84: Product Profile – CSL-654

Table 85: CSL-654 SWOT Analysis, 2015

Table 86: Global Sales Forecast ($m) for CSL-654, 2014–2024

Table 87: Product Profile – N9-GP

Table 88: N9-GP SWOT Analysis, 2015

Table 89: Global Sales Forecast ($m) for N9-GP, 2014–2024

Table 90: Hemophilia A and B with Inhibitors – Phase III Pipeline, 2015

Table 91: Product Profile – BAX-817

Table 92: BAX-817 SWOT Analysis, 2015

Table 93: Global Sales Forecast ($m) for BAX-817, 2014–2024

Table 94: Product Profile – LR-769

Table 95: LR-769 SWOT Analysis, 2015

Table 96: Global Sales Forecast ($m) for LR-769, 2014–2024

Table 97: Product Profile – CSL-689

Table 98: CSL-689 SWOT Analysis, 2015

Table 99: Global Sales Forecast ($m) for CSL-689, 2014–2024

Table 100: Alternative Coagulation Promoters – Early-Stage Pipeline, 2015

Table 101: Hemophilia A and B Gene Therapies – Early-Stage Pipeline, 2015

Table 102: Key Companies in the Hemophilia A and B Recombinant Therapies Market, 2014–2024

Table 103: Baxalta’s Hemophilia Recombinant Portfolio Assessment, 2015

Table 104: Baxalta SWOT Analysis, 2015

Table 105: Bayer’s Hemophilia Recombinant Portfolio Assessment, 2015

Table 106: Bayer SWOT Analysis, 2015

Table 107: Biogen’s Hemophilia Recombinant Portfolio Assessment, 2015

Table 108: Biogen SWOT Analysis, 2015

Table 109: CSL Behring’s Hemophilia Recombinant Portfolio Assessment, 2015

Table 110: CSL Behring SWOT Analysis, 2015

Table 111: Novo Nordisk’s Hemophilia Recombinant Portfolio Assessment, 2015

Table 112: Novo Nordisk SWOT Analysis, 2015

Table 113: Pfizer’s Hemophilia Recombinant Portfolio Assessment, 2015

Table 114: Pfizer SWOT Analysis, 2015

Table 115: Sales Forecast ($m) for Hemophilia A and B Recombinant Therapies in the 7MM+A, 2014–2024

Table 116: Global Hemophilia A and B Market – Drivers and Barriers, 2014–2024

Table 117: Sales Forecast ($m) for Hemophilia A and B Recombinant Therapies in the US, 2014–2024

Table 118: Key Events Impacting Sales for Hemophilia A and B in the US, 2014–2024

Table 119: US Hemophilia A and B Market – Drivers and Barriers, 2014–2024

Table 120: Sales Forecasts ($m) for Hemophilia A and B Recombinant Therapies in the 5EU, 2014–2024

Table 121: Key Events Impacting Sales for Hemophilia A and B Recombinant Therapies in the 5EU, 2014–2024

Table 122: French Hemophilia A and B Recombinant Therapies Market – Drivers and Barriers, 2014–2024

Table 123: German Hemophilia A and B Recombinant Therapies Market – Drivers and Barriers, 2014–2024

Table 124: Italian Hemophilia A and B Recombinant Therapies Market – Drivers and Barriers, 2014–2024

Table 125: Spanish Hemophilia A and B Recombinant Therapies Market – Drivers and Barriers, 2014–2024

Table 126: UK Hemophilia A and B Recombinant Therapies Market – Drivers and Barriers, 2014–2024

Table 127: Sales Forecast ($m) for Hemophilia A and B Recombinant Therapies in Japan, 2014–2024

Table 128: Key Events Impacting Sales for Hemophilia A and B in Japan, 2014–2024

Table 129: Japanese Hemophilia A and B Market – Drivers and Barriers, 2014–2024

Table 130: Sales Forecast ($m) for Hemophilia A and B Recombinant Therapies in Argentina, 2014–2024

Table 131: Key Events Impacting Sales for Hemophilia A and B in Argentina, 2014–2024

Table 132: Argentine Hemophilia A and B Market – Drivers and Barriers, 2014–2024

Table 133: Key Launch Dates

Table 134: Physicians Surveyed

List of Figures

Figure 1: 9MM, Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, All Ages, N, 2014–2024

Figure 2: 9MM, Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, by Age, N, 2014

Figure 3: 9MM, Diagnosed Prevalent Cases of Hemophilia A, by Sex, All Ages, N, 2014

Figure 4: 9MM, Age-Standardized Diagnosed Prevalence of Hemophilia A (per 100,000 Population), by Sex, 2014

Figure 5: 9MM, Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, All Ages, N, 2014–2024

Figure 6: 9MM, Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, by Age, N, 2014

Figure 7: 9MM, Diagnosed Prevalent Cases of Hemophilia B, by Sex, All Ages, N, 2014

Figure 8: 9MM, Age-Standardized Prevalence of Diagnosed Hemophilia B (per 100,000 Population), by Sex, 2014

Figure 9: 9MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, All Ages, N, 2014–2024

Figure 10: 9MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, by Sex, All Ages, N, 2014

Figure 11: Competitive Assessment of Late-Stage Pipeline Agents in Hemophilia A, 2014–2024

Figure 12: Competitive Assessment of Late-Stage Pipeline Agents in Hemophilia B, 2014–2024

Figure 13: Competitive Assessment of Late-Stage Pipeline Agents for Hemophilia A and B Patients with Inhibitors, 2014–2024

Figure 14: Company Portfolio Analysis in Hemophilia A and B, 2014–2024

Figure 15: Global Sales for Hemophilia A and B Recombinant Therapies by Region, 2014–2024

Figure 16: Sales for Hemophilia A and B recombinant therapies in the US by Drug Class, 2014–2024

Figure 17: Sales for Hemophilia A and B Recombinant Therapies in the 5EU by Drug Class, 2014–2024

Figure 18: Sales for Hemophilia A and B Recombinant Therapies in Japan by Drug Class, 2014–2024

Figure 19: Sales for Hemophilia A and B Recombinant Therapies in Argentina by Drug Class, 2014–2024

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