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 (the Foundation) is an independent 501(c)(3) created by Congress to advance the mission of the FDA to modernize product development, accelerate innovation, and enhance product safety. The Foundation works to advance regulatory science, support development and dissemination of reliable information, and facilitate engagement and information exchange.

Media Contacts:

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