FDA issue draft guidance on developing drugs for Duchenne Muscular Dystrophy

The US Food and Drugs Administration (FDA) has issued a draft Guidance for Industry document on “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment,” to assist drug companies in the clinical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.

A number of cell and gene therapy trials are currently being conducted for DMD in the US, and this guidance document is the first to be developed from an initial proposal independently prepared by an advocacy group, Parent Project Muscular Dystrophy (PPMD). The input of PPMD is in recognition of 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. The value of a patient-centered approach to informing and accelerating drug development in the UK was also addressed in a recent BIA and MHRA conference on Pathway of Innovation from Research to Patients.

Author: Anthony Lodge

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