On the Approval of the First Drug Addressing the Underlying Cause of Duchenne

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Coalition Duchenne, a Newport Beach, California based charity committed to raising awareness for Duchenne muscular dystrophy, and funding for Duchenne research, wishes to convey its gratitude for the efforts of everyone involved in the U.S. Food and Drug Administration approval of Sarepta Therapeutics’ novel Duchenne drug Eteplirsen (which has been renamed Exondys 51).

Catherine Jayasuriya and Dusty Brandom

We now look forward to a class of drugs addressing other exons on the dystrophin gene that will treat a large proportion of boys and young men with Duchenne

Coalition Duchenne, a Newport Beach, California based charity committed to raising awareness for Duchenne muscular dystrophy, and funding for Duchenne research, wishes to convey its gratitude for the efforts of everyone involved in the U.S. Food and Drug Administration approval of Sarepta Therapeutics’ novel Duchenne drug Eteplirsen (which has been renamed Exondys 51). Researchers, clinicians, parents, educators, Duchenne organizations, and boys and young men with Duchenne are all celebrating the first approval of a drug that addresses the underlying cause of Duchenne.

“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research.

“I am excited for Sarepta’s success. It is clear that commercialization of drugs by companies like Sarepta is the fastest way to help our sons,” said Catherine Jayasuriya, founder and executive director of Coalition Duchenne.

Catherine funded the Dusty Brandom Fellowship at the University of Western Australia, commencing in 2005, in support of Dr. Steve Wilton’s research, elements of which are now licensed by Sarepta. The Dusty Brandom Fellowship was named after Catherine’s son Dusty who is now 23 years old and has Duchenne.

“To wait for this FDA approval was excruciating, but many of us in the community have been working hard for this outcome for over a decade. When we funded the Dusty Brandom Fellowship, oligonucleotides and exon skipping were a dream that we felt needed to be realized,” said Catherine. “Congratulations Sarepta on realizing that dream. We now look forward to a class of drugs addressing other exons on the dystrophin gene that will treat a large proportion of boys and young men with Duchenne - men like my son Dusty.”

Exondys 51 skips a damaged part of the gene that codes for dystrophin, the protein missing in boys and young men with Duchenne. That damaged part of the gene, exon 51, was chosen because it is one of the more common genetic mutations.

“Dusty has a relatively rare mutation on exon 16. Exondys 51 will not help him. However, follow on drugs using the same technology will. Some of the other exons have relatively small populations of boys but we believe they may respond spectacularly to Sarepta's technology. We must move this technology forward for all the boys and young men with Duchenne,” said Catherine.

About Coalition Duchenne

Coalition Duchenne was founded in 2011 to raise global awareness for Duchenne muscular dystrophy, to fund research and to find a cure for Duchenne. Coalition Duchenne is a 501c3 non-profit corporation. Its vision is to change the outcome for boys and young men with Duchenne, to rapidly move forward to a new reality of longer, fulfilled lives, by funding the best opportunities for research and creating awareness.

Coalition Duchenne has several research initiatives that are making advances in potential cardiac and pulmonary treatments for sufferers of Duchenne muscular dystrophy. These include the pioneering collaboration with a team at Cedars-Sinai Medical Center in Los Angeles, California, led by Eduardo Marbán MD, PhD working on cardiac-derived stem cells and exosomes in Duchenne. This technology has been licensed by Capricor Therapeutics and they have launched an FDA trial. Coalition Duchenne was also instrumental advancing Phrixus Pharmaceuticals’ novel therapy Carmeseal. Phrixus recently announced a preclinical study showing strong results that showed Carmeseal protecting both skeletal and cardiac muscle in Duchenne.

For more information about Coalition Duchenne, visit http://www.coalitionduchenne.org.

About Duchenne muscular dystrophy

Duchenne muscular dystrophy is a progressive muscle wasting disease. It is the most common fatal genetic disease that affects children. Duchenne occurs in 1 in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that codes for the protein dystrophin. This is a vital protein that helps connect the muscle fiber to the cell membranes. Without dystrophin the muscle cells become unstable, are weakened and lose their functionality. Life expectancy ranges from the mid teenage years to age 30.

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