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Coagulation Factors 2011 - who does meet the market needs? A Pipeline Assessment, Market Survey and Corporate Benchmark Analysis

The report “Coagulation Factors 2011 - who does meet the market needs? A Pipeline Assessment, Market Survey and Corporate Benchmark Analysis” provides a description, evaluation and assessment of the recombinant coagulation factor R&D pipelines as of October 2011. The authors analyze and assess the target pipeline for each of the coagulation factors for haemophilia and work out the target profiles of development projects. A market research study was conducted to challenge industry's target profiles and evaluate the sensitivity for pricing.

The report is built upon primary and secondary search for information and use of a proprietary database and news portal. A market survey was conducted among haematologists from haemophilia care centers. In house analysts with experience in biopharmaceutical R&D and business development authored the report and analysed and assessed the findings.

Global sales of the five major recombinant products of coagulation factors VIII, IX and VIIa in 2010 were US$ 6.2 bln (with $ conversion rate October 19, 2010). The first Marketing Authorization Application for a novel recombinant coagulation factor has been submitted in the European Union and further novel wild-type and long-acting recombinant coagulation factors VIII, IX and VII are in advanced clinical stages with potential regulatory approvals within the next 2 to 4 years. Given the progress of the pipeline, a concentration process of players has started. First clinical data are available and will guide further definition of R&D in this field.

Scope of the report

  • R&D pipeline overview;
  • profiles of most promising coagulation factors in the pipeline
  • pipeline analysis for each coagulation factor
  • evaluation and assessment of protein engineering and drug delivery technologies
  • overview and analysis of marketed coagulation factors;
  • market research study with haematologists from haemophilia care centers
  • corporate product portfolios and R&D pipelines;
  • benchmark of competitors regarding achievement of product target profiles

Benefits from the report

  • Understand your position in a highly competitive environment
  • Learn what haematologists think about your product target profiles
  • Understand the market needs
  • Know pricing sensitivities for your haemophilia products
  • Learn from your competitors
  • Challenge your strategy with a view from outside the organization

SAMPLES

Table 9: Total Market Size (US$ mln) of Rec Coagulation Factors in Hemophilia

Year201020092008200720062005
F. VIIIa3,9793,7493,5493,2582,9872,665
F. IXa643568587543538503
F. VIIa1,532*1,3041,1791,0811,039934
Total in
US$ mln
6,1545,6215,3154,8734,5644,102
Growth+9.5%+5.8%+9.1%+6.8%+11.3% 

*, includes Coagil VII

Figure 17: Critical half-life value for long-acting Factor VIII

First clinical data of long-acting factor VIII products indicate that the half-life is only about 1.7 fold longer than that of wild-type factorVIII which could trranslate in one injection less per week, but not a once-weekly adminstration. Would this difference be suificient for you to switch from wild-type to this “long-acting” factor VIII?

Table of Contents

0. Abbreviations

1. Executive Summary

2. Overview

3. Coagulation Factor Markets

  • 3.1. Recombinant Factor VIIIa Market
  • 3.2. Recombinant Factor IXa Market
  • 3.3. Recombinant Factor VIIa Market
  • 3.4. Total Market Size of Recombinant Coagulation Factors for Hemophilia
  • 3.5. Recombinant Thrombin Market
  • 3.6. Total Market Sizes and Market Shares of Rec. Coagulation Factors
  • 3.7. Plasma-Derived Coagulation Factors

4. Pipeline Changes since September 2010

  • 4.1. Alnylam Pharmaceuticals
  • 4.2. Amsterdam Molecular Therapeutics
  • 4.3. Baxter
  • 4.4. Bayer Healthcare Pharmaceuticals
  • 4.5. Biogen Idec
  • 4.6. Catalyst Biosciences
  • 4.7. CSL
  • 4.8. Green Cross
  • 4.9. Grifols (Talecris Biotherapeutics)
  • 4.10. Ipsen & Inspiration Biopharmaceuticals
  • 4.11. LFB (GTC Biotherapeutics)
  • 4.12. Novo Nordisk
  • 4.13. Octapharma
  • 4.15. Pharmstandard
  • 4.16. Pfizer (Wyeth)
  • 4.17. ProFibrix
  • 4.18. Prolor Biotech
  • 4.19. PTC Therapeutics
  • 4.20. ReGenRx Biosciences

5. Target Pipeline Analysis

  • 5.1. Factor VIII
  • 5.2. Factor IX
  • 5.3. Factor VII
  • 5.4. Alternative Procoagulants & Adjuncts
  • 5.5. Factor XIII
  • 5.6. von Willebrand Factor (vWF)
  • 5.7. Fibrinogen & Thrombin

6. Market Research: Survey on Novel Coagulation Factors

  • 6.1. Methodology
  • 6.2. Results: Profile of Participant, Center and Usage of Factors
  • 6.3. Results:Valuation of Characteristics of New Hemophilia Products
  • 6.4. Results: Decisive Characterstics to Switch to New Hemophilia Products
  • 6.5. Results: Price Sensitivity of Characteristics of New Products
  • 6.6. Results: Factors Influencing Decision to Switch to New Products
  • 6.7. Results: Role of Plasma-Derived Coagulation Factors
  • 6.8. Assessment of Survey Results

7. Corporate Benchmark Analysis

  • 7.1. Time to Market
  • 7.2. Immunogenicity
  • 7.3. Administration Frequency
  • 7.4. Pricing: Me Toos and Biosimilars
  • 7.5. Non-Intravenous Administration
  • 7.6. Safety Features of Drug Substance and Drug Product
  • 7.7. Novel Procoagulants and Adjuvants

8. References

9. Tables in the Text

  • Table 1: Sales of Advate/Recombinate
  • Table 2: Sales of Kogenate (FS)
  • Table 3: Sales of Helixate
  • Table 4: Sales of Refacto (AF) / Xyntha
  • Table 5: 2010 Sales of Recombinant Factor VIIIa Products
  • Table 6: Growth of the Recombinant Factor VIIIa Market
  • Table 7: Sales of BeneFIX
  • Table 8: Sales of NovoSeven
  • Table 9: Total Market Size of Rec Coagulation Factors in Hemophilia
  • Table 10: CAGR of the Rec Coagulation Factor Market in Hemophilia
  • Table 11: Sales of Recothrom
  • Table 12: Market Shares of Individual Rec Coagulation Factors
  • Table 13: Sales of Thrombin-JMI Product Family
  • Table 14: Sales of Koate DVI
  • Table 15: Sales of Grifols' pdFVIII Products
  • Table 16: 2010 Sales of Selected Systemic pd Coagulation Factors
  • Table 17: 2010 Sales of Selected Topical pd Coagulation Factors
  • Table 18: Baxter's Coagulation Factors Pipeline
  • Table 19: BAX 326: Clinical Studies
  • Table 20: BAX 111: Clinical Studies
  • Table 21: Baxter's Strategic Goals in Hemophilia
  • Table 22: Bayer's Coagulation Factors Pipeline
  • Table 23: BAY 81-8973: Clinical Studies
  • Table 24: Bayer's Strategic Goals in Hemophilia
  • Table 25: Biogen Idec's Coagulation Factor Pipeline
  • Table 26: rFIXFc Clinical Studies
  • Table 27: rFVIIFc Clinical Studies
  • Table 28: Biogen Idec's Strategic Goals in Hemophilia
  • Table 29: Catalyst Biosciences' Coagulation Factor Pipeline
  • Table 30: Catalyst Biosciences Strategic Goals in Hemophilia
  • Table 31: CSL's Recombinant Coagulation Factor Pipeline
  • Table 32: CSL's Strategic Goals in Hemophilia
  • Table 33: Green Cross' Pipeline of Coagulation Factors in Hemophilia
  • Table 34: Green Cross's Strategic Goals in Hemophilia
  • Table 35: Grifols' Pipeline of Rec. Coagulation Factors in Hemophilia
  • Table 36: Grifols' Strategic Goals in Hemophilia
  • Table 37: Ipsen and Inspiration's Coagulation Factor Pipeline
  • Table 38: Ipsen and Inspiration's Strategic Goals in Hemophilia
  • Table 39: LFB's Pipeline of Recombinant Coagulation Factors
  • Table 40: Novo Nordisk's Pipeline of Coagulation Factors
  • Table 41: Turoctocog alfa: Clinical Studies
  • Table 42: NN7128: Clinical Studies
  • Table 43: Novo Nordisk's Strategic Goals in Hemophilia
  • Table 44: Octapharma's Recombinant Coagulation Factor Pipeline
  • Table 45: Human-cl rFVIII: Clinical Studies
  • Table 46: Octapharma's Strategic Goals in Hemophilia
  • Table 47: Pharmstandard's Coagulation Factor Pipeline
  • Table 48: Pfizer's Coagulation Factor Pipeline
  • Table 49: Pfizer's Strategic Goals in Hemophilia
  • Table 50: ProFibrix' Pipeline of Coagulation Factors
  • Table 51: ProFibrix' Strategic Goals with Coagulation Factors.
  • Table 52: PROLOR Biotech's Coagulation Factor Pipeline
  • Table 53: Product Characteristics of improved rhu F. VIII products
  • Table 54: Overview of rFVIII Pipeline
  • Table 55: Features of Novel Wild-Type rFVIII Molecules in R&D
  • Table 56: Pharmacokinetics of Long-Acting rFVIII Molecules
  • Table 57: Features of Novel Long-Acting rFVIII Molecules in R&D
  • Table 58: Overview of rFIX Pipeline
  • Table 59: Pharmacokinetics of Long-Acting rFIX Molecules
  • Table 60: Features of Novel Long-Acting rFIX Molecules in R&D
  • Table 61: Overview of rFVII Pipeline
  • Table 62: Features of Novel WT or Short-Acting rFVII Molecules
  • Table 63: Pharmacokinetics of Long-Acting rFVII Molecules
  • Table 64: Features of Novel Long-Acting rFVII Molecules in R&D
  • Table 65: Rating of characteristics of future hemophilia producs
  • Table 66: Summary of Ranking of Product Characteristics
  • Table 67: Benchmarking for Novel F.VIII Molecules
  • Table 68: Benchmarking for Novel F. IX Molecules
  • Table 69: Benchmarking for Novel F. VII Molecules
  • Table 70: Benchmarking for Novel Procoagulants & Adjuvants
  • Table 71: Potential First Approvals in EU or USA

10. Figures in the Text

  • Figure 1: Territory of residency of participants
  • Figure 2: Number of hemophilia patients in centers of participants
  • Figure 3: Qualification, function & responsibility of participants
  • Figure 4: Usage of rec and pd coagulation factors in centers
  • Figure 5: Valuation of characteristics of future hemophilia producs
  • Figure 6: Prioritization of characteristics of future hemophilia products
  • Figure 7: Single characteristics making to switch to new product
  • Figure 8: Combination of characteristics inducing a switch decision
  • Figure 9: Sensitivity for pricing of new products - single characteristic
  • Figure 10: Influence of territory on sensitivity for pricing
  • Figure 11: Sensitivity for pricing of new products - combined features
  • Figure 12: Sensitivity for pricing of a “Me Too” / “Biosimilar” product
  • Figure 13: Attributes for selection of new hemophilia products
  • Figure 14: Requested data for claim of “less inhibitor formation”
  • Figure 15: Impact of Aledort meta-analysis on prescribing praxis
  • Figure 16: Impact of Aledort meta-analysis on long-acting BDD FVIII
  • Figure 17: Critical half-life value for long-acting Factor VIII
  • Figure 18: Acceptance of porcine factor VIII
  • Figure 19: Prescribing frequency of plasma-derived coagulation factors
  • Figure 20: Reasons to prescribe plasma-derived coagulation factors

11. Drug Codes Described in the Text

  • ALN-APC
  • BAX 111
  • BAX 326
  • BAX 499
  • BAX 513
  • BAX 817
  • BAX 855
  • BAY 81-8973
  • BAY 86-6150
  • BAY 94-9027
  • BIIB029
  • Coagil VII
  • CSL627
  • CSL654
  • CSL689
  • GreenGene F
  • Hemodure Biopump
  • IB1001
  • JBT8157
  • MOD-3012
  • MOD-5023
  • NN1731
  • NN1810
  • NN1841
  • NN7008
  • NN7088
  • NN7128
  • NN7129
  • NN7451
  • NN7999
  • OBI-1
  • PF-05280602
  • PTC124
  • scAAV2/8-LP1-hFIXco
  • Tachosil

12. ADDENDUM

A) Tabulated R&D Pipelines of Coagulation Factors

  • Tab I: Rec Factor VIII with Normal Half-Life
  • Tab II: Rec Factor VIII with Prolonged Half-Life
  • Tab III: Gene Therapy for Hemophilia A
  • Tab IV: Rec Factor IX with Normal Half-Life
  • Tab V: Rec Factor IX with Prolonged Half-Life
  • Tab VI: Gene Therapy for Hemophilia B
  • Tab VII: Rec Factor VII with Short or Normal Half-Life
  • Tab VIII: Rec Factor VII with Prolonged Half-Life
  • Tab IX: Alternative Pro-Coagulants for Hemophilia A and B
  • Tab X: Rec Factor XIII
  • Tab XI: Rec Von Willebrand Factor
  • Tab XII: Rec Fibrinogen
  • Tab XIII: Rec Thrombin

B) Online Survey on Coagulation Factors 2011

  • Tab XIV: List of Hemophilia Centers/Institutions Contacted in the Survey
  • Tab XV: Content Online Survey Before Activation

Companies mentioned in the report

  • Alnylam Pharmaceuticals
  • Amsterdam Molecular Therapeutics
  • Archemix
  • Baxter
  • Bayer HealthCare Pharmaceuticals
  • Biogen Idec
  • Biotest
  • Bristol-Myers Squibb (Zymogenetics)
  • Catalyst Biosciences
  • CSL
  • Fresenius Kabi
  • Green Cross
  • Grifols (Talecris Biotherapeutics)
  • Ipsen & Inspiration Biopharmaceuticals
  • Johnson & Johnson (Ethicon; OMRIX Biopharmaceuticals)
  • Kedrion
  • LFB (GTC Biotherapeutics)
  • Medgenics
  • Nektar Therapeutics
  • Novo Nordisk
  • Nycomed
  • Octapharma
  • Pfizer (Wyeth; King Pharmaceuticals)
  • Pharming
  • ProFibrix
  • PROLOR Biotech
  • Proteomtech
  • ReGenRx Biosciences
  • Scil Proteins
  • Swedish Orphan Biovitrum
  • Xenetic Biosciences (Lipoxen)
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