Background: On June 9th, the U.S. Food and Drug Administration (FDA) released a draft containing guidance to assist drug companies working to develop drugs for the treatment of Duchenne muscular dystrophy (DMD) and related diseases including Becker muscular dystrophy, DMD-associated cardiomyopathy and symptomatic DMD carrier status in females.
The guidance relates FDA perspective on a number of important aspects of DMD drug development, such as early phase clinical development, treatment across different disease stages, drug efficacy and safety considerations, clinical trial procedures, accelerated approval and benefit-risk considerations. After a 60-day comment period, the FDA will review and release a final version.
Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D .:“This is a very exciting development for our DMD families. With the availability of FDA guidance, companies developing treatments for DMD will now have easy access to the FDA’s perspectives on Duchenne. This could potentially allow companies to initiate faster and more efficient Duchenne drug development programs as we progress towards treatments and cures.
“We look forward to reviewing the guidance in depth and providing feedback to the FDA during the comment period. We applaud the collaborative efforts of our friends at Parent Project Muscular Dystrophy and all of our families in the DMD community for their part in helping to establish guidelines that will facilitate the drug development process for DMD.
“The Muscular Dystrophy Association (MDA) is committed to bringing safe and effective treatments and cures to children and adults living with life-threatening neuromuscular diseases as quickly as possible. Today, there are new treatments in the pipeline that were inconceivable just a few years ago and more potential treatments are expected during the next five years than in the past five decades. As these treatments enter the regulatory review phase, MDA continues to work hand-in-hand with families, health care professionals, scientists and the Food and Drug Administration to ensure it has the necessary information and support to efficiently make decisions on potential life-changing drugs and to move these treatments from the labs to our families’ living rooms.”
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.
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