The Jett Foundation Fighting Duchenne Muscular Dystrophy

Scientific Letter in Support of eteplirsen signed by 36 Researchers/Physicians

March 21, 2016

lou kunkel photo

Scientific Letter in Support of Eteplirsen Signed by 36 World Renowned Duchenne Clinicians and Researchers.

A scientific letter that underscores the strength of Sarepta Therapeutics’ data on eteplirsen, authored by Drs. Stanley Nelson and Carrie Miceli from the Center for Duchenne Muscular Dystrophy at UCLA, and signed by 36 world renowned Duchenne clinicians and researchers, was released publicly this morning, and was recently presented to Dr. Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research and sent to DNP at FDA.

This remarkable letter supports eteplirsen’s clinical efficacy, dystrophin analysis, historical control, and its legal standing for accelerated approval according to FDASIA. These 36 experts collectively treat over 5,000 patients with Duchenne including many enrolled in eteplirsen clinical trials. This letter is the strongest show of collective scientific support to date for the accelerated approval of a potential Duchenne therapy.

This letter reinforces the public testimony of advocates, parents, and patients since week 48 of study 201 in October 2012. The data is there: eteplirsen works. Eteplirsen defies natural history of Duchenne; clinicians are eager to prescribe it, the law supports its accelerated approval and, most importantly, the drug is safe.

Duchenne experts are as rare as the disease in which they specialize, and each has their own unique history, experience, and perspective. Creating a product specific letter that 36 Duchenne experts agreed upon – was an incredible achievement by Drs. Nelson and Miceli. And it was science, not sympathy, that convinced them do so. We applaud and thank every single one of them for their unfailing commitment to their patients and to the Duchenne community. Your work makes you our super heroes.

Signatories include:

  • Jeffrey Chamberlain, a professor and researcher at the University of Washington who studies dystrophies;
  • Sue Apkon, a clinician at Seattle Children’s Hospital who treats Duchenne patients;
  • Lou Kunkel, Harvard Professor and discoverer of the dystrophin gene;
  • The foremost Duchenne experts in the world, doctors and researchers who dedicated their careers to treating and researching an impossible disease that was thought to be too difficult to study and too sad to treat;

Those who signed this letter are trusted by families and patients, globally – to make educated, scientifically sound decisions that affect the lives of those with Duchenne. These experts support eteplirsen, and therefore, this letter deserves the serious consideration of the the FDA.

We implore the FDA to apply the views of the patients who will testify at the Open Public Hearing, and the expertise of the clinicians and researchers who study and treat Duchenne during their review of eteplirsen. There is scientific consensus among the leading Duchenne experts in the world. Eteplirsen is safe, it has the potential to save lives, it must be approved.





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