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Putting DM Front & Center with the FDA

MDF ran an article in the May 2015 Dispatch v.1 that described an array of projects MDF is undertaking as part of MDF 3.0: Accelerating Drug Development, a multi-year, $5M effort to get successful therapies to the DM community as soon as possible.

In that program overview, we described a strategic workshop MDF is hosting this fall for professionals from the Food and Drug Administration, academic research institutions and pharmaceutical companies interested in myotonic dystrophy therapy development. Our objective for this workshop is to ensure that all stakeholders have the information they need to move the drug review and approval process forward.

The workshop, which is scheduled for September 2015 in Washington, DC, will:

  • Educate attendees about the burden that myotonic dystrophy creates for patients and families;
  • Inform the identification of clinical endpoints to guide clinical trial design;
  • Describe what disease improvements would be clinically meaningful to patients and families, and what risks or trade-offs patients might consider in order to achieve certain improvements;
  • Highlight the challenges involved in measuring therapeutic impacts and disease changes in a heterogeneous and slowly progressing disease such as DM;
  • Establish a shared understanding of and commitment to the development of a thoughtful FDA regulatory pathway for DM therapy review and approval.

Dr. Stephen Spielberg, MD, PhD, former Deputy Commissioner at the FDA and now Editor-in-Chief of Therapeutic Innovation and Regulatory Science, will chair the full-day meeting. Dr. Spielberg will be joined by senior leadership from the FDA and leading DM investigators, who will present panel sessions on Myotonic Disease Symptoms and Pathology, Real-World Patient Data, DM Trial Endpoints Selection, Trial Design and Candidate Biomarkers. All sessions will include 30 minutes of discussion between presenters, FDA representatives and the more than 75 participants from academia and pharmaceutical companies who will attend the closed meeting.

This regulatory meeting will be the first of its kind for myotonic dystrophy, and we know the community will want a full report on how it goes. MDF will publish an account of the proceedings in a fall Dispatch, and via a journal article for the professional community shortly thereafter.

Look for ongoing updates on the more than 30 initiatives associated with MDF 3.0: Accelerating Drug Development over the coming months. And don’t hesitate to contact the Foundation if you have questions or comments.






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