FDA issued a draft guidance for industry, “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment,” to assist drug companies in the clinical development of drugs for the treatment of X-linked Duchenne muscular dystrophy (DMD) and related diseases, including Becker muscular dystrophy, DMD-associated dilated cardiomyopathy, and symptomatic carrier states in females.
For the first time, FDA said its guidance was preceded by the submission on June 25, 2014, of a proposed draft guidance independently prepared by an advocacy group, Parent Project Muscular Dystrophy (PPMD). PPMD’s proposed draft guidance was posted on the web for public comment. Both the proposed guidance and public comments submitted to FDA were carefully considered in developing FDA’s draft guidance.
This draft guidance addresses FDA’s current thinking regarding the clinical development program and clinical trial designs for drugs to support an indication for the treatment of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 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. Read the full release