The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment of Duchenne muscular dystrophy (DMD) and related dystrophinopathies including Becker muscular dystrophy (BMD), DMD-associated dilated cardiomyopathy (DCM), and symptomatic carrier states in females. Specifically, this guidance addresses FDA’s current thinking regarding the clinical development program and clinical trial designs for drugs to support an indication for the treatment of one or more of these dystrophinopathies. The most prominent pathology in dystrophinopathies is degeneration of skeletal and cardiac muscle leading to progressive loss of muscle function, respiratory and cardiac failure, and premature death. This draft guidance is intended to serve as a focus for continued discussions among the Division of Neurology Products, pharmaceutical sponsors, the academic community, and the public. This guidance does not address the development of drugs to treat secondary complications of muscle degeneration in dystrophinopathies (e.g., drugs specifically for heart failure or for pulmonary infections).
Development of this guidance was preceded by the submission to FDA of a proposed draft guidance independently prepared by a consortium of stakeholders including patients, parents and caregivers, clinicians, scientific experts, and industry representatives. The proposed draft guidance submitted by the consortium was made available through a Federal Register notice seeking public comment. Both the independently prepared proposed draft guidance and the public comments received in response to the Federal Register notice were considered in writing this guidance.
This guidance does not contain discussion of the general issues of statistical analysis or clinical trial design. Those topics are addressed in the ICH guidances for industry E9 Statistical Principles for Clinical Trials and E10 Choice of Control Group and Related Issues in Clinical Trials, respectively.