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The Real Deal: Parents say New Drug Can Save Children but FDA Hasn't Approved It

Updated: Friday, April 11 2014, 11:55 PM EDT
ROUND TOP, N.Y.---Some are calling it a miracle drug, an experimental medication that has slowed or even stopped the progression of duchennes disease, a form of muscular dystrophy, in boys.  A clinical trial has produced groundbreaking results, the maker of the drug has asked for accelerated approval but so far the FDA has not made a decision and in the meantime, local families are in limbo.
 
Duchennes muscular dystrophy is a degenerative disease in boys, most who have it don't make it to the age of 30.  That's why time is of the essence for these local families; their boys are teenagers and while the FDA has told them it's looking at a path to approval, they don't believe the agency is moving fast enough.    
 
Like most 17 year-olds, Westin Alfred likes to play video games and hang out with his friends.  He's also been thinking a lot lately about the future lately, "I want to get into architecture and blue printing design," he says.  His mom is his biggest fan, "he's an amazing boy, he looks forward to going to college, he's got a prom coming up... he keeps everyone in good spirits," Colleen Hill tells CBS6.
 
But the reality is that Westin has been losing control of his muscles.  He was diagnosed with duchennes when he was 6, in a wheelchair by 9 and just a few months ago he underwent spinal surgery. "We've seen boys, we've been at meetings where they're on respirators and they're only able to move their fingers... I don't want that to happen, not when we have a chance to stop that," Hill says. That chance, she is referencing, is a drug called eteplirsen.  In a clinical trial, it appeared to stop the progression of the disease in some boys and in others it actually made them stronger.  The FDA has granted an "orphan drug" designation to eteplirsen which is a special status for drugs that show promise to treat rare and fatal diseases but so far it has not been approved for use.  "If they had children, they'd do anything to save them and to know that there's something out there that can save and they're holding back for reasons that make no sense when the illness is, there's no cure and now there's something that can help and it's safe and effective...I beg them to allow my son to get this," Hill says.
 
The drug is not a cure but it's hope for Westin's family and the Frolish family of Saratoga Springs.  They have two boys with duchennes and have been keeping a close eye on the trial as well, " it would give more time for a cure to be found that maybe someday they could regain the ability to walk or stand," Alex Frolish tells CBS6.
 
In a statement a spokeswoman for the FDA says, "FDA staff are prohibited by law from discussing the status of any investigational drug or any drug application that may under agency review. Under the law that is confidential information between the FDA and the company and we are not permitted to discuss specifics."
 
Jim Baker, A spokesman for Sarepta Therapeutics, the maker of eteplirsen says, " We are currently working with the FDA to come to an agreement on the design of a larger study of eteplirsen that would include more patients at a number of clinical sites. Pending feedback from the FDA, our plan is to initiate this study in the second quarter of this year, with dosing in patients to begin in the months following. In parallel, we are seeking to address the concerns the agency shared with us in November regarding a potential application for an early approval based on the currently available clinical trial data for eteplirsen."
 
Nationwide, parents who have children with duchennes have started a petition (https://petitions.whitehouse.gov/petition/urge-fda-say-yes-accelerated-approval-safe-effective-therapies-children-duchenne/qtRww0rN) which now has more than 100,000 signatures.  They've also put together a website, (http://theracetoyes.org/) with more information on their push to get this drug approved.
The Real Deal: Parents say New Drug Can Save Children but FDA Hasn't Approved It


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