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FDA considering new drug that helps Spinal Muscular Atrophy patients for approval

Posted: 11:37 PM, Sep 26, 2016
Updated: 2016-09-27 11:15:18-04

This is a story of two children with an identical genetic condition: Spinal Muscular Atrophy, which robs children of their muscles.

Three-year-old Jace Dorer of Royal Oak is one of those children; Channel 7 anchor Stephen Clark’s granddaughter Scarlet is the other.

But while Scarlet continues to lose strength every day, typical for SMA children, Jace is getting stronger.

The difference is a drug called Nusinersen, which Monday submitted an application for FDA approval. For the last 15 months Jace has been part of clinical trials for the drug.

“Within a year we may have the very first approved treatment for this disease,” says Kenneth Hobby, CEO of CureSMA.org. “We believe this is a treatment that will slow things down, buy us more time. Hopefully its really good to stop the further progression that’s going to give us the ability to bring new drugs to bear that can hopefully work out to be a cure.”

Even though this is great news for families of SMA children there is still frustration that Nusinersen won’t be available for at least a year as it winds through the FDA approval process.

“The average lifespan for somebody that is affected by SMA type one,” Hobby points out, “is just two years. So you can see the race we have for families that are getting diagnosed today that we need to move through this as quick as we can.”

Even though the typical time for FDA approval is nearly a year, Biogen Pharmaceuticals has applied for Priority Review, which if granted, would shorten this review time to 6 months in the U.S.