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Whitehouse petition: Urge the FDA to Say YES to Accelerated Approval for children with Duchenne.

WE PETITION THE OBAMA ADMINISTRATION TO:

Urge the FDA to Say YES to Accelerated Approval for safe, effective therapies for children with Duchenne.

We urge the Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy - the leading genetic killer of children that impacts 1 out of every 3,500 boys born in the United States. It's time for the FDA to Say Yes and make this the first generation of Duchenne survivors.

 

https://petitions.whitehouse.gov/petition/urge-fda-say-yes-accelera...

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Besides signing the petition, please ask your spouse, adult kids, friends, co workers, or others to sign the petition.  Post it on Facebook, Twitter, Linked In, other social networks.

https://petitions.whitehouse.gov/petition/urge-fda-say-yes-accelera...

Every year this approval is delayed about 90 boys in the USA will die from DMD that could be saved by skipping exon 51. Another 90 will take their last step, and another 90 will take their last unassisted sip of water.

Approving Eteplirsen will open the floodgates for skipping other exons.  It won't be instantaneous.  But it will happen sooner than it would if Eteplirsen is further delayed.  The FDA needs to recognize 6MWT and dystrophin as a biomarker

Key scientists have testified to the FDA and Congress the drug works and appears very safe.  Even, if this doesn't help your boy, it will help directly some boys with DMD and start the long road to helping many others where exon skipping technology will work.

Ideally the FDA will give this a full approval with monitoring and recognize dystrophin as a biomarker.  The FDA should provide assistance in writing the NDA as has happened for other rare diseases

.

Ask your congress people to push for approval! Ask others.  You can make a difference NOW!

Signatures needed by March 29, 2014 to reach goal of 100,000

80,000

Total signatures on this petition

20,000
Keep up the good work and open the floodgates get everyone you know to sign

how soon could the skipping of other exons be realized after FDA approves exon 51? and which ones are likely to be next?

Next exon's are 53, 44, 45 will need to do Phase 1 & 2 to apply for Accelerated Approval about 2 half 3 years.   Accelerated Approval can also be applied to all DMD drugs like utrophin up regulation and others. 

Hi Tommy i am actually Jason's brother.  We need exon 44 skipped because we have a exon 45 deletion so how fast things move after exon 51 is something i have looked into.  The way to think of Sarepta is the drug is like a drill and each exon just needs a different drill bit attached to the drill.  So right now with no revenue from Eteplirsen the company can only put limited funds towards producing the drug for other exons.   If Eteplirsen were approved now Sarepta is very close to going into full production.  They then could immediately start accelerating production for the other exons, i believe exon 44 is next.  So Sarepta has said if Eteplirsen was approved now they could have it ready by the end of this year.  

Another way to speed this up is getting the FDA to approve dystrophin as a biomarker. (wouldn't need 6-minute walk any more for exon skipping drugs)

Hi Richard, my son Max has exon 43 deletion. Am I right in thinking exon 44 skipped would work with that as well?

Richard Darienzo said:

Hi Tommy i am actually Jason's brother.  We need exon 44 skipped because we have a exon 45 deletion so how fast things move after exon 51 is something i have looked into.  The way to think of Sarepta is the drug is like a drill and each exon just needs a different drill bit attached to the drill.  So right now with no revenue from Eteplirsen the company can only put limited funds towards producing the drug for other exons.   If Eteplirsen were approved now Sarepta is very close to going into full production.  They then could immediately start accelerating production for the other exons, i believe exon 44 is next.  So Sarepta has said if Eteplirsen was approved now they could have it ready by the end of this year.  

Drugs approved under accelerated approval with a dystrophin surrogate endpoint would still require a confirmatory trial with a clinical endpoint, like the 6MWT, to receive full FDA approval.  If no clinical efficacy is shown in the confirmatory trial, the drug would be removed from the market.       

Yes, Phil.  An exon 43 deletion would require exon 44 to be skipped to get back in frame.

You are incorrect Keith If dystrophin was confirmed as a biomarker it can be used as primary endpoint.

The FDA did not feel that the reduction in decline of 6MWT was due to increases dystrophin therefor not a biomarker. 

They also said the way researchers have been measuring dystrophin for 25years+ is inaccurate  

 

Phill I'll answer for my brother. Your right Max need to skip exon 44

Thanks Keith & Jason.


Sorry Keith your right about surrogate endpoint but what a biomarker does do is allow smaller studies (less patients)  this is very important for the rare exon.

This is another reason I think the FDA has it backwards. It should be a primary endpoint.

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