Non-profit group submits DMD guidance to FDA

Non-profit advocacy group Parent Project Muscular Dystrophy (PPMD) and a coalition of stakeholders, including biopharma representatives, submitted to FDA draft guidance on the clinical development of products to treat Duchenne muscular dystrophy (DMD). According to PPMD, FDA invited the Duchenne community to submit the document after convening a Duchenne policy forum last December. The guidance notes outcome measures for DMD trials depend on age and functional capacity of patients and that companies should consider monitoring pulmonary and cardiac status in younger patients. In patients 7-13 years of age, the guidance notes six-minute walk distance (6MWD) is the most commonly used primary outcome measure and identified North Star Ambulatory Assessment as a measure for trials of longer duration. ...