Genzyme, who are responsible for taking Ataluren through outside of N America are holding a conference call on Monday 13 September for representatives of patient organizations. If you have any specific questions that you would like asked, please email me at mark@actionduchenne.org as soon as possible
Thanks

Mark

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Hi Mark. Will they publish a summary of this conference call either on this site or in any other place?

Bernardo
Hi Mark, I would lke to know if Genzyme is trying to approve the use of Ataluren in Europe.
Not sure what will be published from the conference call but I will make some notes and circulate the key points.

Ofir - I am assuming Genzyme are still responsible for Ataluren approval outside of N America. I am keen to understand what progress they are making in different countries, including those where the drug has been trialled.



Ofir Arad said:
Hi Mark, I would lke to know if Genzyme is trying to approve the use of Ataluren in Europe.
Great!. Thank you Mark and look forward to your notes.
Hi Mark, do you know something new?
Hi there,

I am hoping that some notes of the meeting are going to be typed up shortly from a transcipt made but in short some of the main points I noted were:

- Still waiting for biopsy data analysis to be completed. Expected to be a matter of 'months' for this to be complete

- Genzyme will be holding discussions with regulatory bodies within EU member states by the end of the year

- At this stage there is no need for patient organisations to join Genzyme in such discussions but when the need arises, involvement of patient organisations will be important

- Eligibility for any Expanded Access Problem would be on the basis of matching the trial population as closely as possible

- Not clear how Ataluren might be made available for those who took part in the trial in a different country from where they live e.g. Swiss citizens enrolled via Italy

Although there was no significant new information revealed, both Genzyme and PTC had a significant number of patient advocacy and clinical research personnel on the conference call reinforcing their commitment to finding a clinical and regulatory path forward for Ataluren. FYI, a representative from Genzyme is scheduled to speak at the Action Duchenne conference - Saturday 13 November (4.30 slot).

As and when the notes of the meeting are typed up, I'll get them posted asap.

best wishes
Mark
I don't exactly understand what happens with Ataluren. It's been so long since they stopped the trial and I do not see any clear path even after so may months. Are they collecting more data now through individual INDs? Do they plan to file for market approval? What is the next step? I am quite confused about this. What happens these months, are they still analyzing data? Haven't they said during the first CC after they stopped the trial that the biopsy data will be available by now? This is really incredible, 150 biopsies take so long, even if they would look at one biopsy per day, they should still be done by now. My friend is a pathologist and does a few biopsies per day as part of her job. What's going on here?

I just hope something like this does not happen with the other drugs tested in the future.
Just my opinion. Unlike many potential Duchenne treatments, Ataluren has clear commercial potential (if it works). The potential patient population is enormous (potential to treat nonsense mutations in numerous genetic diseases). I believe that Genzyme will not risk this drug for Duchenne. As the benefit, even of low dose, was somewhat disappointing and inconsistent, they will push forward with Phase 3 in Cystic Fibrosis (which is now getting a much longer term study-you can go to website). Big potential money is at stake. They will proceed where they feel there is the best chance for initial success. Perhaps the endpoints of CF are easier to measure. My take (I have no specific personal knowledge) is that there is a lot going on behind the scenes that has nothing to do with the analysis of Duchenne patient data (?business considerations). Duchenne is not very amenable to short term studies. One would have to measure slowdown of progression over a very long period of time. If functional endpoints are more important (for trial purposes) than dystrophin expression, proving efficacy for any dystrophin restoration drug may be quite difficult (you need years, not months, to measure success).

Ofelia Marin said:
I don't exactly understand what happens with Ataluren. It's been so long since they stopped the trial and I do not see any clear path even after so may months. Are they collecting more data now through individual INDs? Do they plan to file for market approval? What is the next step? I am quite confused about this. What happens these months, are they still analyzing data? Haven't they said during the first CC after they stopped the trial that the biopsy data will be available by now? This is really incredible, 150 biopsies take so long, even if they would look at one biopsy per day, they should still be done by now. My friend is a pathologist and does a few biopsies per day as part of her job. What's going on here?

I just hope something like this does not happen with the other drugs tested in the future.
I think that maybe the problem is that the results of the biopsies are not good enough. To approve the first drug of the market is easier than to approve the second one. Now the only thing that they have to show is that is better to take ataluren then nothing. But if they see something strange they have to explain it. We know that something strange happens because its not normal that the low dose is better then the high dose. Maybe the results of the biopsies are also "strange" and the FDA will only approve the drug if they find an explanation, and it can take time. But if the drug will be approved for cystic fibrosis I am sure that Duchenne patients will use it too, because PTC demonstrated that the low dose is better than nothing.
Exactly! . That is precisely what I intend to do, no matter what. I live in Argentina and I'm not sure it would be easy to get the drug anyway, but if the FDA approves Ataluren for CF, there's no doubt in my mind that I will make sure my son (wh has DMD) will get the low dose. By the way, does anybody know what exactly this "low dose" was in the clinical trials? (how many mgs per kilo?)

Best regards

Bernardo

Ofir Arad said:
I think that maybe the problem is that the results of the biopsies are not good enough. To approve the first drug of the market is easier than to approve the second one. Now the only thing that they have to show is that is better to take ataluren then nothing. But if they see something strange they have to explain it. We know that something strange happens because its not normal that the low dose is better then the high dose. Maybe the results of the biopsies are also "strange" and the FDA will only approve the drug if they find an explanation, and it can take time. But if the drug will be approved for cystic fibrosis I am sure that Duchenne patients will use it too, because PTC demonstrated that the low dose is better than nothing.
Low-dose PTC124 = 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening
It seems that they have seen this inverted U-curve (or whatever they call it) before with other drugs, when the low dose is more effective than high. Not sure what happens with biopsies but it's hard to believe that they do not have them completed and analyzed by now. Of course, there are many things not available to public as you mentioned.

Ofir Arad said:
I think that maybe the problem is that the results of the biopsies are not good enough. To approve the first drug of the market is easier than to approve the second one. Now the only thing that they have to show is that is better to take ataluren then nothing. But if they see something strange they have to explain it. We know that something strange happens because its not normal that the low dose is better then the high dose. Maybe the results of the biopsies are also "strange" and the FDA will only approve the drug if they find an explanation, and it can take time. But if the drug will be approved for cystic fibrosis I am sure that Duchenne patients will use it too, because PTC demonstrated that the low dose is better than nothing.

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