Reagan-Udall Foundation for the FDA Announces Hybrid Public Meeting on Qualifying Biomarkers to Support Rare Disease Regulatory Pathways

Case Example: Heparan sulfate in neuronopathic lysosomal storage diseases

What: The Reagan-Udall Foundation for the FDA will host a public workshop to explore primary disease activity biomarkers in rare genetic diseases.  Attendees will hear perspectives from patient advocates, researchers, regulators, and representatives from regulated industry.  The discussions will include a series of case studies (heparan sulfate in neuronopathic lysosomal storage diseases) and a dialogue on the challenges in qualifying biomarkers to support rare disease approvals. 

 

When: February 21, 2024, 10am-4pm (eastern)

 

Where: Remote and in-person (1333 New Hampshire Ave, NW, Washington, DC 20036)

For members of the press who would like to attend in-person, please email admin@reaganudall.org with the subject line "Press Request."

 

Agenda

10:00AM

 


Welcome & Opening Remarks

Susan C. Winckler, RPh, Esq.
Reagan-Udall Foundation for the FDA

10:10AM

 

 

Biomarkers in Rare Genetic Diseases

  • Peter Marks, MD, PhD 
    Center for Biologics Evaluation and Research, FDA

     

    10:40AM

     

     

    Case Study: Understanding Neuronopathic Mucopolysaccharidoses (MPS)

    • Joseph Muenzer, MD, PhD
      Univ. of North Carolina at Chapel Hill
    • Mark Dant
      Ryan Foundation

      11:20AM

       

      Case Study: Measuring Glycosaminoglycans, including Heparan Sulfate  

      • Maria Fuller, PhD
        University of Adelaide

      11:50AM

      Q&A Session with Morning Case Study Presenters

      12:10PM

      Lunch Break (provided)

      12:40PM

       

       

       

      Case Study: Animal Model Translation to Human Application

      • Matthew Ellinwood, DVM, PhD
        National MPS Society
      • Nidal Boulos, PhD
        REGENXBIO Inc.
      • Patricia Dickson, MD
        Washington University School of Medicine, St. Louis

        1:20PM

         

         

         

        Case Study: Relationship Between Cerebrospinal Heparan Sulfate Levels and
        Clinical Outcomes

        • Eric Zanelli, PhD
          Allievex
        • Simon Jones, MBChB
          University of Manchester
        • Heather Lau, MD, MS
          Ultragenyx

          2:05PM

          Q&A Session with Afternoon Case Study Presenters

          2:35PM

          Break

          2:45PM

           

           

           

           

           

          Panel Discussion: Challenges in Qualifying Biomarkers to Support
          Rare Disease Approvals

          Moderator: Susan C. Winckler, RPh, Esq.
          Reagan-Udall Foundation for the FDA

          • John Crowley, JD, MBA
            Amicus Therapeutics, Inc.
            Biotechnology Innovation Organization (incoming)
          • Cherie Fathy, MD, MPH
            Center for Biologics Evaluation and Research, FDA
          • Carole Ho, MD
            Denali Therapeutics
          • Gavin Imperato, MD, PhD
            Center for Biologics Evaluation and Research, FDA
          • Edward Neilan, MD, PhD
            National Organization of Rare Diseases
          • Cara O'Neill, MD
            Cure Sanfilippo Foundation
          • James Wilson, MD, PhD
            University of Pennsylvania

          3:55PM

          Closing Remarks

          4:00PM

          Adjourn

           

           

           

           

          Contact: Register and learn more on the Foundation website. Media inquiries can be addressed to Mary McNamara  (mmcnamara@reaganudall.org; 202-276-7822).   

           

          (Denali Therapeutics, Orchard Therapeutics, REGENXBIO Inc., and Ultragenyx provided funding for this event.)

           

           

          About the Reagan-Udall Foundation for the FDA

          The Reagan-Udall Foundation for the FDA (FDA Foundation) is an independent 501(c)(3) created by Congress to advance regulatory science to help the U.S. Food and Drug Administration accomplish its mission. The FDA Foundation works to improve health and safety through stakeholder engagement and public-private partnerships that facilitate innovation, foster the use of real-world evidence, and identify modern tools and policies to keep pace with today’s rapidly evolving science.

          Media Contacts:

          Lea Ann Browning-McNee
          301-509-1846 (cell)
          Lmcnee@reaganudall.org  

          Mary McNamara
          202-276-7822 (cell)
          mmcnamara@reaganudall.org