October 6, 2022 / Advocacy,Research

PPMD Submits Updated Community Draft Guidance for Duchenne, Becker, and Related Dystrophinopathies to FDA

An exciting update to the revised community-led Duchenne Guidance for FDA is that it has been officially submitted to the agency!

As you know, this Community Draft Guidance will serve as an update to the original guidance that was submitted in July 2014. Because of so many advancements in knowledge, understanding, care, clinical trials, and approvals, it is important for us to now “modernize” the Community-Led Guidance of 2014 to reflect this new landscape in Duchenne and Becker therapy development. This critical community guidance update process has involved over 100 stakeholders and has taken over a year to compile and submit. 

This new version includes updates to sections of the 2014 document as well as additional original sections covering cardiac, gene therapy, and informed consent sections for the FDA’s consideration.

PPMD thanks the FDA for their receptive response to our original 2014 submission. We have requested that the FDA officially open a new docket for this revised community guidance initiative to allow for broad public attention and commentary. We will continue to keep you updated as this process moves along, but we are ecstatic about this important step being completed. 

View the submitted draft guidance  >

About The Revised Guidance

The process of updating the Community Guidance has involved over 100 stakeholders including patients, caregivers, clinicians, researchers, drug companies, genetic counselors, and regulatory experts. This unprecedented effort brought together the leading experts in Duchenne serving on working groups in the following areas:

  • Benefit Risk Preferences and Patient Experience
  • Biomarkers
  • Diagnosis
  • Cardiac
  • Gene Therapy
  • Natural History, Trial Design, Outcome Measures

Members of each working group can be viewed here.

Two advisory boards were also involved in the update overseeing the process and commenting on each section of the Guidance; A Pharmaceutical Advisory Board comprised of 21 companies currently developing therapies for Duchenne and Becker, and a Community Advisory Board that included 20 Duchenne partner organizations. 

Community Advisory BoardPharmacy Advisory Board
ActionDuchenneAntisense Therapeutics Limited
Best Day Ever FoundationAstellas GT
Charley's FundAvidity Bio
Coalition DuchenneCumberland Pharma
Cure DuchenneDaiichi Sankyo
Duchenne AustraliaDyne
Duchenne UKEdgewise
EveryLife Foundation for Rare DiseasesEli Lilly
JB's Keys to DMDEntradaTX
Jesse's JourneyEmpirium
Jett FoundationFibroGen
Kindness Over Muscular DystrophyPfizer
Little Hercules FoundationPTC Bio
MD CanadaRegenex Bio
Muscular Dystrophy AssociationReveragen
My DMD HeroRoche
ParentProject OnlusSanthera
Powers PromiseSarepta
Save Our SonsSolid Bio
Team JosephTRINDS
The Hope for Gus FoundationVertex
Walking Strong

Join Our Mailing List