Massachusetts Governor Sending Top Aide To Support Duchenne Drug Approval

WASHINGTON, April 24, 2016 /PRNewswire-USNewswire/ -- The Jett Foundation, a Kingston Massachusetts-based Duchenne patient advocacy organization, is pleased to announce that Governor Charlie Baker will be sending his Deputy Chief of Staff, Carlo Basile to the Food and Drug Administration (FDA) Peripheral and Central Nervous System Advisory Committee Meeting on April 25.  The Committee, which will meet in College Park, Maryland, has been convened to evaluate eteplirsen, a potential Duchenne muscular dystrophy therapy manufactured by Sarepta Therapeutics of Cambridge. Basile's nine-year old son suffers from Duchenne.   

"Duchenne is a deadly, pediatric disease with no FDA approved treatment. Massachusetts families affected by Duchenne deserve access to safe and effective therapies for this horrible disease, and we look forward to seeing real progress for families soon," Governor Baker said. "I have asked Carlo to attend to demonstrate my administration's strong support for the Duchenne Community as they share their powerful stories with the agency."

"We are very pleased to have Carlo join us on Monday," said Christine McSherry, Founder and Executive Director of The Jett Foundation, a Massachusetts based non-profit organization. "The FDA must consider the severity of Duchenne when evaluating new drug applications for safe, and potentially efficacious therapies for Duchenne, a 100 percent fatal rare disease. We hope that the FDA will quickly complete its review and grant accelerated approval to this potential life-saving therapy."

Sarepta Therapeutics is seeking Accelerated Approval for eteplirsen based on a validated clinical endpoint from a small trial of 12 patients, representative of the rarity of the disease and its mutations. Patients and families affected by Duchenne believe that eteplirsen's data is strong and that the 12 boys in Sarepta's 4.5-year trial are benefitting from treatment. Ten of the boys are still walking and the disease, overall, is far less progressed in these boys than the natural history of Duchenne would predict. None of the 130 plus patients exposed to eteplirsen through the original 12-patient trial and subsequent confirmatory and safety trials, have experienced any adverse events from the drug.

On February 24, 2016, 38 Duchenne researchers and clinicians sent a letter to FDA in support of eteplirsen's data. The clinicians who signed that letter have collectively treated 5,000 Duchenne patients over the course of 15 years. Their scientific and medical evaluation of eteplirsen's data led them to conclude that the most scientifically robust and ethical way forward was for the FDA to grant eteplirsen accelerated approval and follow up with confirmatory trials to ensure efficacy.

On April 16, 2016, 24 Members of the United States Senate, including Senators Elizabeth Warren (D-MA) and Edward Markey (D-MA), sent a letter to FDA urging the agency to use accelerated approval and to take the patient perspective into consideration when reviewing drugs to treat Duchenne. On February 18, 2016, 109 Members of of the United States House of Representatives, including Representatives Keating, Lynch, Capuano, Tsongas, and McGovern sent a letter to FDA expressing similar sentiments.

"I look forward to joining researchers, scientists and Duchenne families in Maryland this week," said Basile. "I share their hope that the FDA will act quickly to approve a safe and effective treatment for Duchenne, and I am proud and grateful to be representing Governor Baker and all Massachusetts Duchenne families at this important meeting."

About Duchenne muscular dystrophy: Duchenne is a progressive neuromuscular disorder that causes a loss of motor, pulmonary, and cardiac function, and premature death. It is the most common lethal pediatric genetic disorder, and it affects every 1 in 3,500 live male births and some females. Duchenne is caused by the body's inability to create dystrophin, a large protein found in muscle cells. Duchenne has no FDA approved treatment or cure and is 100% fatal. Children with Duchenne are born normal and decline over time, usually losing their ability to walk around the age of 12 and succumbing to the disease in their early to mid-twenties.

About the Jett Foundation: Since 2001, Jett Foundation, located in Kingston, MA, has worked to find treatments and a cure for Duchenne muscular dystrophy (DMD) while improving the lives of those affected by DMD. Over the past decade, Jett Foundation has raised over $15 million for Duchenne research and programs to enhance the lives of those living with Duchenne. The Jett Foundation also provides resources for treatments and adaptive activities, serves as an educational resource, and provides camp opportunities through Camp Promise. The Jett Foundation is a registered charity with 501(c)(3) status from the IRS; all donations are tax deductible. www.jettfoundation.org.

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SOURCE Jett Foundation