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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Send your email to: PCNS@fda.hhs.gov

Email subject line: Written testimony submission for FDA Advisory Committee Meeting to consider Eteplirsen 

Attn: Moon Hee V. Choi

Please be sure to indicate "The following is my written testimony submission for the FDA Advisory Committee Meeting to consider Eteplirsen - April 25, 2016"

Details and Guidance on Submitting Written Testimony to the FDA Adv...

Thanks Andrea!

Hi All,

I am frustrated as well to see what is going on here.  My 4 year old son son was recently diagnosed in August with a duplication of 46 and none of the exon skipping drugs in the pipeline will help him, but I can understand your frustration.  I find it even more ridiculous that the FDA is not consistent within each division.  I am a nurse by background and have been working in Medical Affairs at Novartis Oncology/Rare Disease for the past 8 years and the FDA has approved many drugs (conditional approval) with really small populations with side effects.  I don't understand how is this possible on the oncology side, but not on the neurology side.  There needs to be more education within the FDA to look at what others are doing.  Not only has the oncology side used Accelerated Approval, but the newly created Breakthrough status has also been given to some drugs which really shortens the timeline to approval.  I pray for each of our sons each day and hope that treatments are forthcoming.  Are any of you going to the conference in June.  Would love to meet you guys.  We live in NJ.  


Hi Limca
Really good to see everyone raising a voice. Expecting more responses. Thanks !

I hope somebody is listening at the FDA, although in the case of Ataluren they have plenty of information and evidence supporting the usefulness of the drug. I cannot find the right way to describe their conduct. Criminal negligence perhaps?

PPMD is working on our behalf. Let's see what FDA does on Eteplirsen!

Well, I hope PPMD can somehow explain to the FDA for a thousandth time that Ataluren works. I am sceptical, given the harsh terms of the FDA's Refusal to File letter addressed to PTC in late February. I do not see any reaction from PPMD about this, even though more than 40 days have gone by,  

Dear All,

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 


Thanks Ryan!

Thank you Ryan.

What did Ryan post?

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