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I am not seeing any posts or comments about the devastating news that the FDA has again chosen to sabotage Ataluren, by issuing a "Refusal to File" letter to PTC, which then led to the company announcing that it is laying off 18% of its workforce in the US, plus a huge fall of the price of its shares. The news is more than one month old, and yet I only learned about it by chance, by checking a financial news website. 

In the past I submitted many comments about this key drug and the idiotic way in which the FDA has treated this very promising (so far unique) treatment for nonsense mutation DMD,which HAS DEFINITELY SHOWN A MARKED IMPROVEMENT FOR PATIENTS AT LOW DOSES OVER THOSE ON PLACEBO.

In Europe, PTC was granted conditional approval more than 15 months ago. Many patients and parents around the world were eagerly waiting for the FDA to follow the same path.

What is this? Some kind of jingoistic "war" between the FDA and the EMA?. Are the Americans trying to make the point that they are reeeaaaaally careful and responsible, while the Europeans are a bunch of happy-go-lucky bums?

I've been following the story of PTC124 since early 2003 (THIRTEEN YEARS!!) My son is now almost 26 years old. Everybody knows that Ataluren WORKS. There is a 47 meter difference in the 6MWT in favor of Ataluren treated boys against those on placebo. I've had enough, really.

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Hi Cinni! 

PPMD has been in communication with PTC since they received the Refusal to File letter from the FDA. It is our understanding that PTC is putting together a strategy so that they can go back to the agency in order to understand a path forward.  We have let Stu and the entire team at PTC know that the community is ready and willing to do whatever we can to make the case for this therapy. We hear you and remain frustrated by the lack of urgency by FDA regulators when it comes to rare disease. We promise to be in touch with more once we are clear about what regulatory options exist.  Please contact me if you want to discuss or have any questions.Ryan@parentprojectmd.org 


Cinni said:

What did Ryan post?
Never mind I read it :)
Thanks Ryan :)
Any update on Ataluren. It's been approved in the UK now! Can we please do something about it, how do we raise our voices and be heard on this drug. It's torture to know that there is a drug out there than can help but access to it is denied. Will they file again with the FDA. I tried reaching out to PTC therapeutics but they wouldn't provide me with a solid answer on what they are planning here in the US.

Dear Mr. Iriberri,

I have been trying unsuccessfully to learn how well European boys have done with the Ataluren.

I have contacted PTC Therapeutics but am told to have the doctor phone.

I only know that the FDA would not review the drug because it crosses the blood brain barrier

but my grandson has a nonsense point mutation and none of the exon-skipping trials addresses

his particular mutation.  He is six years old.

Both doctors he has seen have recommended Ataluren but no one can tell

me how to obtain it.

Since NICE approved the drug, i guess we could establish residency in England.

As Ever,

Frances Maher



Frances Maher said:

Dear Mr. Iriberri,

I have been trying unsuccessfully to learn how well European boys have done with the Ataluren.

I have contacted PTC Therapeutics but am told to have the doctor phone.

I only know that the FDA would not review the drug because it crosses the blood brain barrier

but my grandson has a nonsense point mutation and none of the exon-skipping trials addresses

his particular mutation.  He is six years old.

Both doctors he has seen have recommended Ataluren but no one can tell

me how to obtain it.

Since NICE approved the drug, i guess we could establish residency in England.

As Ever,

Frances Maher

Thank you very much for your post Frances. I sincerely hope that your grandson can access Translarna soon. My son will turn 26 in August and here we are, still evaluating the prospects of applying for an import permit from Europe (we live in Argentina), but it is very hard. I don't understand the FDA' position at all. But I am sure that eventually they will come to their senses. Your grandson is young and hopefully things will improve in time for him to benefit from the treatment. As far as I know the drug may slow down the progress of the disease, although it is not a cure, but information is still sketchy. Best of lucks!. Bernardo



Bernardo A. Iriberri said:

Thank you very much for your post Frances. I sincerely hope that your grandson can access Translarna soon. My son will turn 26 in August and here we are, still evaluating the prospects of applying for an import permit from Europe (we live in Argentina), but it is very hard. I don't understand the FDA' position at all. But I am sure that eventually they will come to their senses. Your grandson is young and hopefully things will improve in time for him to benefit from the treatment. As far as I know the drug may slow down the progress of the disease, although it is not a cure, but information is still sketchy. Best of lucks!. Bernardo



Frances Maher said:



Bernardo A. Iriberri said:

Thank you very much for your post Frances. I sincerely hope that your grandson can access Translarna soon. My son will turn 26 in August and here we are, still evaluating the prospects of applying for an import permit from Europe (we live in Argentina), but it is very hard. I don't understand the FDA' position at all. But I am sure that eventually they will come to their senses. Your grandson is young and hopefully things will improve in time for him to benefit from the treatment. As far as I know the drug may slow down the progress of the disease, although it is not a cure, but information is still sketchy. Best of lucks!. Bernardo

pls let me know if we can do something to accelerate FDA's action and respond, it is nonsense that FDA slow down or ignore decisions that make life saving difference!

i am from Iraq and nothing available here neither community for DMD nor centers or even hospitals to take care of  information about my son's disease DMD!

imagine how i feel, i feel like i am useless for my son, i am trying to search by myself but still don't know how to make right decision?!

All,

Two things:

If you have been involved in a clinical trial with ataluren or are on translarna currently. please take the following survey:

http://www.parentprojectmd.org/translarnaexperience

Also, for those interested, we are holding a webinar with PTC to discuss the path forward and the data - the webinar is happening on Monday August 1st http://community.parentprojectmd.org/events/upcoming-webinar-ptc-th...

I wish we could do something, but I am affraid that especially for those of us who do not live in the US there's nothing realistically available. Personally I am just hoping that the application in Canada is successful, and then I will apply here in Argentina for coverage and importation of the drug. The FDA is playing some sort of political game as far as I can tell.

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