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What Does It Take to Be a Long-Acting Replacement Therapy in Hemophilia A?

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Presentation on theme: "What Does It Take to Be a Long-Acting Replacement Therapy in Hemophilia A?"— Presentation transcript:

1 What Does It Take to Be a Long-Acting Replacement Therapy in Hemophilia A?

2

3 Introduction/Overview

4 Key Technologies in EHL Hemophilia Recombinant Factors

5 EHL Agents Extend Half-Life by Improving AUC and Clearance

6 The Importance of Maintaining Trough Levels to Prevent Breakthrough Bleeding

7 Current Unmet Needs in the Treatment of Hemophilia A

8 Phenotypic Variability Among PWH-A

9 Hemophilia A: Replacement Therapy Regimens

10 Long-Acting Factor VIII Replacement Therapies Offer Many Choices Relevant to the PWH-A's Phenotype

11 Benefits of Prophylaxis: Increased the Likelihood of Leading a More Normal Life

12 How Will EHL Concentrates Impact Prophylaxis?

13 rFVIIIFc: A-LONG Study Design

14 Factor VIII Activity vs Time Profile for Long-Acting rFVIIIFc and Standard rFVIII: A-LONG

15 Safety and Efficacy of BAY 94-9027: Study Design of PROTECT VIII

16 PROTECT VIII Summary Results: Bleeding and Dosing

17 AFFINITY Study Design

18 AFFINITY Results: Treatment Regimens Prior to Study Entry and at Study End

19 Safety Results: A-LONG; PROTECT VIII

20 AFFINITY Results: Safety and Tolerability

21 Case Study 1: College-Age Male

22 Case Study 2: Pediatric Patient

23 Summary and Conclusions

24 Abbreviations

25 Abbreviations (cont)


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