Table of Contents
FDA Advisory Committee January 13, 2003
Agalsidase beta: Proposed indication and dose
Order of topics
Fabry Disease
Overview of clinical trials
FB9702: Design
FB9702: Results
AGAL-002: Design
AGAL-002: Endpoints
AGAL-002: Conduct
AGAL-002: Demographics and baseline characteristics
AGAL-002: Results
AGAL-002: Results
AGAL-002: Results
AGAL-002: Results
AGAL-002: Results
AGAL-002: Results
AGAL-002: Conclusions
AGAL-005: Single arm, open-label treatment extension to AGAL-002
Open-label extension treatment: Results
Requests & responses to first review cycle
Requests & responses to first review cycle
Requests & responses to first review cycle
Extension trial histology: 6 months
Extension trial histology: 6 months
Extension trial histology: 18 months
Long-term skin histology-skin superficial capillary endothelium
Open-label extension: Additional results
Open-label extension: Safety results
Open-label extension: Conclusions
AGAL-007
Additional safety data
Summary of safety and efficacy
Accelerated Approval
Accelerated Approval
Accelerated Approval
Accelerated Approval
Verification trial AGAL-008
Proposal for conversion of verification trial
Historical control data collection
Historical control: “Qualified” data set
Historical control: “Qualified” data set
Historical control: Failure to “qualify”
Historical control: demographics and characteristics
Characteristics of “qualified” dataset
Historical creatinine data: Examples
Historical creatinine data: Examples (continued)
Historical creatinine data: Examples (continued)
Historical creatinine data: Examples (continued)
Historical dataset
Historical Dataset
Issues in use of historical control
Comparability of historical to trial population
Comparability of patient-external factors
Accuracy of historical control analytic method
Robustness of historical data
Prior historical data analytical method
Assessment of prior proposed method
Assessment of prior proposed method
Issues in use of historical control
Prior historical analysis: Conclusions
New proposed analysis of historical data
Issues: New proposed analytical method
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