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

Introduction/Overview

Key Technologies in EHL Hemophilia Recombinant Factors

EHL Agents Extend Half-Life by Improving AUC and Clearance

The Importance of Maintaining Trough Levels to Prevent Breakthrough Bleeding

Current Unmet Needs in the Treatment of Hemophilia A

Phenotypic Variability Among PWH-A

Hemophilia A: Replacement Therapy Regimens

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

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

How Will EHL Concentrates Impact Prophylaxis?

rFVIIIFc: A-LONG Study Design

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

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

PROTECT VIII Summary Results: Bleeding and Dosing

AFFINITY Study Design

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

Safety Results: A-LONG; PROTECT VIII

AFFINITY Results: Safety and Tolerability

Case Study 1: College-Age Male

Case Study 2: Pediatric Patient

Summary and Conclusions

Abbreviations

Abbreviations (cont)