Today PPMD is pleased to share that the revised community-led Duchenne Guidance has been published in the Journal of Neuromuscular Diseases!

This Community Draft Guidance was submitted to the FDA at the end of 2022. Serving as an update to the original guidance that was submitted in July 2014, the revision of this document is important in light of the  many advancements in knowledge, understanding, care, clinical trials, and approvals we’ve seen within the Duchenne and Becker therapy development landscape since its original submission. 

This critical process of “modernizing” the Community Guidance involved over 100 stakeholders and spanned more than a year. The updated version includes revisions to sections of the 2014 document as well as additional original sections covering cardiac, gene therapy, and informed consent sections.

While PPMD continues to encourage the FDA to officially open a new docket for this revised initiative to allow for broad public attention and commentary, we know it is critical that these updates are shared with the broader community. Therefore, PPMD along with the Guidance Steering Committee elected to publish the entire document and make it publicly available. The publication contains the executive summary outlining the updates to the document, with the entire document being available as a supplement. 

It is our hope to utilize this updated Guidance as a living document for drug development for those with dystrophinopathy.

Over the past few years, drug development in Duchenne has seen rapid advancements. We have witnessed multiple approvals, novel trial design strategies, an evolving natural history of the disease, and cutting edge therapeutic development. It is critical that we reflect these fast-paced changes in more real time so the entire field can advance together.

Read the article in the Journal of Neuromuscular Diseases here.