Is the FDA Taking Its Time While Children Die of Muscular Dystrophy?

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By USDR

Parents of children facing devastating muscle wasting and premature death from Duchenne Muscular Dystrophy are calling on the U.S. Food and Drug Administration to accelerate approval of a new drug that leading scientists say is safe and effective.

Through their campaign “The Race to Yes,” the families need 100,000 signatures by March 29 on a petition to guarantee a White House response. As of March 18, they have 43,750 signatures.

The parents believe the FDA is discounting clear evidence that a drug – eteplirsen – can safely and effectively treat the world’s leading genetic killer of children. Long-term testing in boys with Duchenne amenable to this drug shows it halts progression of the disease and has no side effects.

“Every day that the FDA stalls, Duchenne takes children’s lives,” said Tracy Seckler whose son, Charley, has the disease. “The FDA claims this is a priority, but they’ve been sending us the same bureaucratic form letter for several months. If they grant accelerated approval, our children could be the first generation of Duchenne patients to survive.”

Marissa Penrod, whose son Joseph also has Duchenne, said, “I’ve been praying for a miracle, and now that miracle is actually here in the form of a treatment that could pave the way to help all children with Duchenne. I’m begging President Obama to step in and hold the FDA accountable to follow the law and approve this drug.”

More than 20 Duchenne-related organizations have joined the national grassroots campaign to get 100,000 signatures by the deadline.

Last month, seven of the world’s leading Duchenne scientists and researchers met with FDA officials in Washington, D.C. to urge approval of the drug. Thirty-eight members of the U.S. House and Senate have written the FDA urging it to make greater use of a 2012 law that allows for accelerated approval for drugs to treat rare and fatal diseases that have no other treatments.

Australian professor Steve Wilton, a neuromuscular researcher and a pioneer in this science, told the FDA “in Australia we’d say it’s bleeding obvious” that the drug works.

Celebrities such as Olympic gold medalist Aly Raisman, Dallas Mavericks owner Mark Cuban and actor/rapper LL Cool J have tweeted asking their followers to sign the petition.