Primary Mitochondrial Diseases

A Virtual Rare Disease Workshop
May 22, 2025 | 10am-4pm (eastern)

The Reagan-Udall Foundation for the FDA, in collaboration with the Food and Drug Administration (FDA), will host a virtual, public workshop on "Primary Mitochondrial Diseases."

As part of the Foundation's rare disease focus, this workshop will explore opportunities to optimize therapeutic development addressing primary mitochondrial diseases and best practices in designing and interpreting clinical studies that can produce meaningful data to accelerate drug development. Speakers will include patient advocates, academic researchers, regulated industry, healthcare providers, and other key stakeholders.

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Agenda

10am

Welcome

10:05am

FDA Opening Remarks

10:10am

Setting the Stage: A Variety of Perspectives

  • Clinical Trial Design and Implementation: Patient Population Considerations
     
  • Primary Mitochondrial Disease Drug Development

11:10am

Reactor Panel

11:35am

10-minute Break

11:45am

Selecting Patient-focused Outcomes and Statistical Considerations

12:15pm

Presentation on COA Example

12:30pm

Reactor Panel

1pm

Break

1:30pm

Current Approaches, Challenges, and Opportunities

2:30pm

What Have We Learned and How Do We Move Forward

3:30pm

Closing Remarks and Adjourn