FDA details potential plan to grant accelerated approval to Duchenne treatment

Rebecca Savransky, Campus Editor

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The Food and Drug Administration outlined a possible pathway that would grant accelerated approval to a drug with the potential to treat Duchenne muscular dystrophy, the disease Dance Marathon raised more than $1.3 million to fund research for last quarter.

The FDA Safety and Innovation Act passed by Congress in 2012 gives the FDA the ability to approve therapies at a faster rate for rare diseases with “unmet medical need,” including Duchenne, the most common degenerative genetic muscle disease. 

Team Joseph, the primary beneficiary for the 2014 Dance Marathon, is a part of a larger network of Duchenne foundations responsible for creating a website called The Race to Yes, which includes a link to a White House petition created last February asking the government to grant this specialized approval of the drug, eteplirsen. The petition received more than 100,000 signatures in 30 days, the requirement for it to be looked at by the government.

Sarepta Therapeutics, a Massachusetts-based biopharmaceutical company, announced its plans to move forward in submitting a new drug application to the FDA for eteplirsen in a news release Monday, after the FDA advised them of a way to submit the proposal under a “potential accelerated approval pathway.”

“We are excited to have guidance from the FDA that allows us to move quickly into additional clinical trials with eteplirsen to confirm our current understanding of eteplirsen’s safety profile, its effect on dystrophin production and its impact on clinical outcomes in DMD patients,” said Edward Kaye, M.D., senior vice president and chief medical officer of Sarepta Therapeutics.

Last month, DM public relations co-chairs Summer Delaney and David Harris sent out an email from the 2014 Executive Board with a message urging students to sign the petition in support of finding a treatment for children who suffer from Duchenne.

“A pending FDA decision could determine whether this generation of children is the last to die from Duchenne or the first generation to live,” the executive board wrote in the email.

Email: rebeccasavransky2015@u.northwestern.edu
Twitter: @beccasavransky

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