COLLEGE PARK, Md., April 22, 2016 /PRNewswire-USNewswire/ -- The researchers from the UCLA Center for Duchenne Muscular Dystrophy, along with parents and patients, will travel to College Park, Maryland to make their case to the Food and Drug Administration (FDA) for an accelerated approval of eteplirsen, a non-toxic treatment designed to improve quality of life for Duchenne patients. Eteplirsen is currently the only drug in the FDA's approval pipeline for Duchenne, meaning that there are no other short-term prospects for fighting a disease that is one of the most common and devastating genetic diseases of childhood, affecting approximately 1 in 5,000 boys.
“We hope to convey to these FDA officials that this is a safe treatment that has a high likelihood to improve the quality of life for the boys who suffer from this tragically progressive disease,” said CDMD spokesperson Chris Moore. “We encourage anyone with an interest in helping these boys to talk with these dedicated scientists and parents.”
The original January advisory committee was postponed and is expected to draw a record-high 1200 attendees, including boys living with the disease and their families travelling from 38 states and all over the globe, some driving several days.
Accelerated approval under the FDA is a particular type of approval that the FDA may grant for a product for a serious or life-threatening disease or condition upon determination that the product has an effect on a surrogate or clinical endpoint that is reasonably likely to predict clinical benefit taking into account the severity, rarity or prevalence of the condition and availability or lack of alternative treatments.
"This is a promising drug in the pipeline for a ravaging disease, and on Monday we're going to help make the case to the FDA that there is substantial evidence of effectiveness that leads to improvement in both mobility and quality of life on the basis of restoring the protein that is genetically missing in all boys with Duchenne,” said M. Carrie Miceli, Ph.D, Professor of Microbiology Immunology and Molecular Genetics at UCLA and co-director of the Center for Duchenne Muscular Dystrophy. “From the available data, the benefits far outweigh the risks in this case."
The FDA's Center for Drug Evaluation and Research April 21, 2016 briefing document stated that they are not convinced of the drug's efficacy in treating the disease.
"We are not asking the FDA to lower the standard criteria for approval, we're asking them to take a long hard look at the results and make their determination based on the risks versus benefits for these boys. We have carefully analyzed the results of the trial with comparison to external control data, which indeed provides strong evidence to support accelerated approval. Further, the absence of serious side effects and lack of alternative treatments makes the case for approval compelling." said Stanley Nelson, Professor, M.D., Department of Human Genetics at the David Geffen School of Medicine at UCLA and a co-director of the Center for Duchenne Muscular Dystrophy.
Duchenne Muscular Dystrophy, which disproportionately effects boys, leaves patients with progressive muscle degeneration, which leads to loss of ambulation by age 11, loss of upper arm use in the teen years, and heart and respiratory failure leading to death often in the early 20's.
Established in 2006, the CDMD at UCLA was built as a result of collaborations among UCLA scientists, clinicians and researchers. Its efforts span from education and outreach to cutting- edge research, drug discovery, clinical trials and care. Funding for the CDMD's remarkable achievements to date has come from a combination of research grants and private philanthropy.
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SOURCE UCLA Center for Duchenne Muscular Dystrophy