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  4. Developing Therapies for Primary Mitochondrial Diseases: Bridging the Gaps - 09/06/2019 - 09/06/2019
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Workshop | Mixed

Event Title
Developing Therapies for Primary Mitochondrial Diseases: Bridging the Gaps
September 6, 2019

September 6, 2019
Event Location
White Oak Campus: The Great Room
Conference Center

10903 New Hampshire Ave
Building 31, Room 1503
Silver Spring, MD 20993
United States

Mitochondria Images

Given the complexities of primary mitochondrial diseases and, by extension, the challenges of designing informative clinical trials in this group of rare diseases, this scientific symposium will bring together academic physicians, FDA regulatory experts, and experts from related disciplines to exchange ideas on ways to address the challenges and to promote drug development in this challenging field.


Persons interested in attending this public workshop either in-person or by web viewing must register on-line by 5 pm on September 5, 2019.

Persons with disabilities having problems accessing the PDF file(s) below may call (301) 796-3634 for assistance.


8:00 - 8:05 am  Welcome and Introduction, Dragos Roman, FDA

8:05 - 8:30 am  Integrating Mitochondrial Biology into Designing Drug Development Programs, Robert Naviaux, UCSD

8:30 - 9:00 am  Lessons Learned from Previous Drug Development Programs, Jim Carr, Stealth Biotherapeutics & Matt Klein, BioElectron

9:00 - 9:45 am  Leveraging Natural History Data when Designing Clinical Trials, Michio Hirano, Columbia University, Philip Yeske, UMDF & Anita Zaidi, FDA

9:45 - 10:15 am  Patient Population Selection and Consideration for Pediatric Patient Enrollment in Clinical Trials, Amel Karaa, Massachusetts General Hospital & Melanie Bhatnagar, FDA

10:15 - 11:00 am  Panel Discussion, Moderated by Amel Karaa (Massachusetts General Hospital) with Robert Naviaux (UCSD), Matthew Klein (BioElectron), Irina Anselm (Boston Children’s Hospital), Kathrine Neveu (Patient Representative), Michio Hirano (Columbia University), Melanie Bhatnagar (FDA) & Patroula Smpokou (FDA)

11:00 - 11:45 am  Open discussion and Q & A

11:45 - 1:00 pm  Lunch

1:00 - 1:45 pm Defining and Assessing Clinical Benefits: Regulatory, Scientific, and Patient Perspectives, Sophia Hufnagel, FDA, Bruce Cohen, Akron Children’s Hospital & Phil Yeske, UMDF

1:45 - 2:15 pm  Panel Discussion Moderated by Philip Yeske (UMDF) with Andrea Gropman (Children’s National Health System), Mindy Leffler (Casimir Trials), Emily Milligan (Barth Syndrome Foundation), Jim Carr (Stealth Biotherapeutics), Ebony Dashiell-Aje (FDA), Teresa Buracchio (FDA/DNP), Dragos Roman (FDA) & Michio Hirano (Columbia University) & Rachel Witten (FDA)

2:15 - 2:45 pm  Clinical Trial Design and Statistical Considerations, Frank Sasinowski, Hyman, Phelps & McNamara, P.C. and Yan Wang, FDA

2:45 - 3:15 pm  Panel Discussion Panel Discussion Moderated by Alex Sherman (ALS Network and Massachusetts General Hospital) with Frank Sasinowski (Hyman, Phelps & McNamara, P.C.), Bruce Cohen (Akron Children’s Hospital), Tristan Massie, FDA, Yan Wang (FDA), Gregory Levin (FDA), Ilan Irony (FDA) & Jim Carr (Stealth Biotherapeutics)

3:15 - 3:45 pm  Open Discussion and Q&A

3:45 - 4:00 pm  Summary and Closing Remarks - who is doing this?


Webcast is available the morning of the workshop. Please register and the webcast link will be emailed the day before the workshop. You must register before noon September 5, 2019

*FDA welcomes the attendance of the public at its advisory committee meetings and will make every effort to accommodate persons with physical disabilities or special needs. If you require special accommodations due to a disability, please contact the Project Manager at least seven days in advance of the meeting.

Morning Session

Afternoon Session


Laurie-Anne Sayles, Project Manager
Center for Drug Evaluation and Research
Office of New Drugs
Public Meeting & Symposia Project Management Team


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