Rare Disease Report

Statement from the Jett Foundation About FDA Extension To Review Eteplirsen

MAY 25, 2016
Guest post by Christine McSherry

“This morning we learned that FDA will will not be able to complete their work on the review of eteplirsen by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016

"While we are disappointed that FDA will not meet it’s deadline, we are hopeful that this additional time will provide the agency with further clarity and comfort around approving eteplirsen. I am optimistic that ongoing conversations within the agency around the approvability of eteplirsen means that there is still a viable path forward for eteplirsen and the rest of the exon-skipping therapies in the pipeline.

"Eteplirsen’s strong clinical efficacy and dystrophin data, clean safety profile, and scientific, medical, and patient support is unprecedented in Duchenne. For these reasons, we remain confident that science and process will win out, and that the next announcement will provide the answer we have been hoping for.

"I remain confident that these internal discussions will allow FDA to do the right thing, and grant eteplirsen approval. In the meantime, we will continue to wait, as we have been for 4 years, in hopes that eteplirsen will soon be the first FDA approved therapy to treat Duchenne and that there will be a feasible way forward for the development of future candidate treatments for Duchenne.”

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 dollars 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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