Would Patients With Hemophilia B Benefit From Switching to Extended Half-Life Factor Products?

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Presentation transcript:

Would Patients With Hemophilia B Benefit From Switching to Extended Half-Life Factor Products?

Introduction/Overview

Prophylaxis is the Recommended Therapeutic Regimen

Key Unmet Needs in Hemophilia B

Potential Barriers to Adherence in Hemophilia B Prophylaxis

Extended Half-Life Agents and Treatment Individualization

The Importance of Maintaining Trough Levels to Prevent Breakthrough Bleeding

Pharmacokinetics of 3 Extended Half-Life Agents

Improving Adherence in Hemophilia B

Higher Factor Activity Levels Can Reduce Average Number of Joint Bleeds

Any Joint Bleeding May Be Too Much Joint Bleeding

rFIX Molecules With Extended Half-Lives

rFIX-FP

PROLONG: FIX Activity After Injection of a Single Dose of rFIX-FP or Previous FIX Product (PK Population)

PROLONG: AsBR: Prophylaxis Patients

PROLONG: Safety Results

rFIX-Fc

B-LONG: rFIX-Fc Fusion Protein: PK Results

B-LONG: Median ABR

B-LONG: Safety Results

Kids B-LONG: Study Results

Glycol PEGylated rFIX

PARADIGM 2 N9-GP: Annualized Bleeding Rates Results

PARADIGM 2 N9-GP: Mean Predose FIX Activity

PARADIGM 5 N9-GP: Overview of Results

Prolonged Exposure to PEGylation in PWH-B: Cause For Concern?

Relevance of Differences Among the 3 Extended Half-Life rFIX Agents

Switching Patient From Standard to Extended Half-Life rFIX Agents

Summary and Conclusions

Abbreviations

Abbreviations (cont)