FDA issues draft guidance produced with the help of PPMD on developing drugs for Duchenne Muscular Dystrophy
FDA has issued a draft guidance for industry, “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” to assist drug companies in the clinical development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. This draft guidance addresses FDA’s current thinking regarding the clinical development program and clinical trial designs for drugs to support an indication for the treatment of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The development of this document was preceded and guided by a proposed draft guidance independently prepared by an advocacy group, Parent Project Muscular Dystrophy (PPMD). Below is a summary of a paper describing how PPMD took up the challenge of drafting these guidelines.
FDA has released a press statement stating that they “value(s) PPMD’s effort and input and appreciates the insights provided by the DMD community.” FDA believes that this is an excellent example of collaboration between engaged stakeholders and a beneficial approach to get contributions from patients and caregivers on drug development.
Stakeholders and interested parties may view the Federal Register notice for information on how to submit comments to the public docket.
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