CureDuchenne, the California-based nonprofit organization dedicated to finding cures for Duchenne muscular dystrophy, released the following statement from its co-founder and CEO, Debra Miller, following the meeting of the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA). The FDA will consider the committee’s discussions, and has set a December 27 deadline for whether to approve the drug.
“We are hopeful that the FDA will approve drisapersen, and that it can become a useful tool against Duchenne, and we are grateful to the panel for listening to patients and parents. Their voices, not only those of doctors and researchers, are critical to the process of drug approval. There are currently no treatments available for this fatal genetic disease, and there is a great sense of urgency for those living with the disease to have treatments that can slow its progression.
“While we urge the FDA to approve drisapersen, it is only one of many Duchenne drugs in development. This disease, with its complex genetic roots, is unlikely to be cured by any single drug, which is why we have invested in different types of therapies that target all aspects of the disease.
“To the Duchenne community, your daily courage inspires our sustained efforts to work toward new, innovative therapies. We will not rest until all patients with Duchenne muscular dystrophy have access to treatment and, ultimately, a cure.”
Additional Background
Duchenne muscular dystrophy is a fatal disease affecting more than 300,000 patients, mostly boys and young men, worldwide. Because of a variant in one exon – a segment of genetic code in the cells of Duchenne patients – the body is unable to produce dystrophin, a protein critical to the formation of muscle tissue. Exon-skipping drugs, in effect, tell the body to ignore the damaged genetic material, thus encouraging the cells to produce new dystrophin and thereby maintain muscle strength.
Through a successful venture philanthropy model, CureDuchenne has leveraged more than $100 million from biotech and pharmaceutical companies and other foundations to fund research leading to a cure.
In the last year, CureDuchenne has supported the development of several promising therapies, including a $1 million investment in MyoTherix Inc., a biotechnology company focused on developing novel therapeutics for the treatment of Duchenne and other muscular dystrophies. It also has invested $1.5 million in Capricor Therapeutics, a biotechnology company, to advance promising research to treat heart muscle failure associated with Duchenne muscular dystrophy.
Following the Advisory Committee’s meeting, the action date for completion of FDA review of the drisapersen NDA is December 27, 2015.

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