FDA's draft guidance on Duchenne muscular dystrophy and related dystrophinopathies is the culmination of an extensive effort led by a patient advocacy group that enlisted the support of patients, caregivers, academia and industry.
DMD drug development has been hindered by a lack of defined outcome measures and understanding of surrogate markers or biomarkers that could support approval. To address this, FDA invited Parent Project Muscular Dystrophy (PPMD) and the DMD community to submit a proposed draft guidance that could clarify a clinical development pathway for approval.
FDA spokesperson Kristofer Baumgartner said in a statement to BioCentury that PPMD's submission is the first time in the Center for Drug Evaluation and Research's knowledge that an advocacy group has submitted a proposed draft guidance, and that this type of engagement is an example of how early input from patients and caregivers can contribute to drug development.
PPMD President and CEO Pat Furlong told BioCentury that the group and its collaborators spent years looking for ways to ensure that DMD studies and endpoints would be sensitive to both