Last night I watched the recording of the Ataluren webinar, held on January 17, 2013. Excuse me for not sounding particularly upbeat about it. I know that PTC and everyone at PPMD are doing as best they can in order to finally get approval for this very successful drug, but I could not avoid a sense of deep frustration and anger, which was triggered about mid-way into the webinar recording, when in one of the slides it became clear for everyone to see that the primary endpoint in the Phase 2B trial that was stupidly cancelled three years ago (a Delta of 30 meters in the 6 minute walk test) had not only been ACHIEVED, but that it had actually been SURPASSED.

While the preliminary reports that led to the cancellation of the trial in March 2010 talked about a Delta of "only" 29.7 meters in the 6MWT between the placebo cohort and the boys who had been on low dose Ataluren (a shortfall of just 1% that for anyone possessing common sense would habe been touted as a resounding success), it now turns out (THREE YEARS LATER!!!), that the Delta was actually 31.3 meters. This means that rather than a 1% shortfall in the expected results of this particular primary endpoint, the boys on low dose Ataluren had actually beaten the goal.

I am simply outraged to learn that at least five precious years have been wasted because of the defective funtioning of the brains of those who gave the order to stop that 2010 trial. I say five years because, as the webinar informs us, it won't be until mid 2014 that PTC will be able to recruit the 220 boys that are needed for the Phase III Trial. Add to that the year it takes to conduct the trial and you are already in 2015. By then, many boys with DMD who could be benefitting right now from Ataluren will be dead.

At the end of 2015 we will attend another webinar where they will tell us that indeed the earlier results were correct, and that the low dose regimen shows remarkable attenuating results. In short: the trials were stopped in March 2010 because of the actions of stupid and incompetent people. Is there anybody being held responsible for this inexcusable waste of time?

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Dear Sharon

You said that you have been to Europe. Do you think it would be possible to apply to EMA for compassionate use of Ataluren? And in case of positive response (hopefully) would there be a chance to get the drug from PTC? We live in Europe, my son has a stop codon.

Hi Peter, I think the issue is just the same as in the US--even if the EMA granted access through some kind of compassionate use program, PTC would be prohibited from selling the drug and would have to provide it for free (and monitor for adverse events etc).  I just don't think they have the financial resources to give away the drug to everyone who could potentially benefit while they are also trying to conduct a big and expensive phase III study, which will be necessary to gain approval both in the US and Europe.  Could they do it for a few people?  Sure, but who would those people be and who would decide?  That's why right now they are only providing expanded access to boys who participated in one of the earlier trials.  It truly is an awful situation, but I think the best route to getting access to the drug will be for it to be approved as soon as possible, preferably on both sides of the Atlantic.

Sharon

I think that the problem lies with the totally unnecessary Phase III Trial, and the two years minimum that will be wasted as a result of this. The re-assessed results of the Phase IIb trial are clear: the low dose regimen works. It could be improved and fine-tuned, yes, but it woks and that was clear already in 2010. The Phase III trial (or whatever other proper name they can come up with) could perhaps be used for purposes of fine-tuning the correct dose, but in the meantime the FDA and the EMA should grant immediate approval for stop codon DMD.

I have to agree with you here Bernardo if the FDA was truly interested they could do this via some funding. After all the USA is giving 17 billion dollars to Egypt's new government, the Muslim brotherhood plus a few dozen tanks and F-18s. If they gave 1 less plane and 1 less tank, that would provide and extra 43 million dollars for critical "people" help.

But that would make too much sense in this country.  The USA must be the butt of jokes across the whole planet. Any other rationalization of the FDA and it's accursed rule book insults intelligence with double talk and obfuscation of what can be done, with more reasons of why it cannot be done.... all wasted drivel from smiling fools running interference for sycophant morons.

If this were for AIDS - they wouldn't even waste time testing it on mice, before releasing it to everyone, WHY???,  because AIDS patients pay taxes and vote - kids with Duchenne don't.

Hi Sharon

Thank you for your reply. This paper you are working on, will it adress Ataluren specific or trials in genereal ? You mentioned earlier that having the parents voice up front when it comes to what we are willing to risk contra benefit could be the right way to go. Will this be included in this paper to the FDA/EMA ? I strongly suggest that you also send the same paper to the EMA. I have learned after many years with DMD, you can not take anything for granted. 

Berit 

Hi Berit--My colleague Holly Peay is doing the majority of the work on the risk/benefit survey (along with all of the parents who have been willing to complete this hour and a half long survey!).  We are analyzing the preliminary results now, but yes, we absolutely plan to provide the study results to the FDA.  We can make it available to the EMA as well, but we may need to do a parallel study in Europe to take into account cultural differences and we are happy to partner with groups in Europe to adapt the survey.  Sharon

Thanks Sharon

Although we live in different contries around the world, I don't think there are differences in how parents relate to the risk/benefit issue. We all have one thing in common, not matter where in the world you live. We want the best available treatment for our kids - as soon as possible. If a survey should take place in Europe too, we have to spend even more time. My suggestion is to use the same survey done in the US, and try to influence the EMA with this. I don't think any parents would disagree in this.

Berit

I agree with Berit 100%. Human nature is the same all over the world. We are one species and cultural differences only play a role in less important matters, not in love between parents and children or in life-death situations like this. Besides, almost three years of continuous in-take of Ataluren by dozens of boys under trials and extended trials have shown that THERE IS NO RISK. So I imagine that the question in the survey could go like this: "Do you want your son to receive NOW a drug that has proven to have benefficial effects by at least slowing down the progression of DMD, or would you rather wait for five or ten more years until we tell you what we have known since 2010, i.e. that the drug is riskless?

We can try...and will try.  If we have to do a European survey, though, we will do it (partnering with colleagues in other organizations in Europe).  One way or another it will get done. 

Thank you Sharon. Please keep us posted  - good or bad news. Would also like to hear what PTC says, when you have spoken to them about the time line.

Needless to say, all boys with a stop codon not included in one of the previous Ataluren trials, are in a hurry to get this drug. 

Berit

Hi Pat and Sharon

Very important recommendations: 

http://www.parentprojectmd.org/site/DocServer/WhitePaper_2013FINAL.pdf?docID=1...

Will this help speed up approval for Ataluren ? Will the European Medical Agency also have recommendations like this ?

Berit

 

Berit, we hope that this document will help speed the approval of all therapies.  We will coordinate with our colleagues in Europe on an EMA effort (although in some key ways the EMA is more progressive than the FDA already)....all of these therapies are going to have to be global otherwise the economics won't work so a European strategy is important.

 

Sharon

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