FDA issues draft guidance on developing drugs for Duchenne Muscular Dystrophy

[6/9/2015] FDA today issued a draft guidance for industry, “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment,” to assist drug companies in the clinical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For the first time, the development of FDA guidance was preceded by the submission on June 25, 2014, of a proposed draft guidance independently prepared by an advocacy group, Parent Project Muscular Dystrophy (PPMD). FDA values PPMD’s effort and input and appreciates the insights provided by the DMD community. PPMD’s proposed draft guidance was posted on the web for public comment. Both the proposed guidance and public comments submitted to FDA were carefully considered in developing FDA’s draft guidance.

FDA recognizes the unmet medical need that exists in patients with DMD, the devastating nature of the disease for patients and their families and the urgency to make new treatments available. This example of collaboration between engaged stakeholders and FDA highlights how input from patients and caregivers can contribute to drug development.

This draft guidance addresses FDA’s current thinking regarding the clinical development program and clinical trial designs for drugs to support an indication for the treatment of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. This guidance does not address the development of drugs to treat secondary complications of muscle degeneration in dystrophinopathies.

Stakeholders and interested parties may view the Federal Register notice for information on how to submit comments to the public docket .