ATALUREN - confused about the information given from PTC Therapeutics and Genzyme

 

Hi Sharon and Pat

or anyone else who can help

 

Can someone PLEASE help me understand the information given from PTC Therapeutics and Genzyme, regards to Ataluren. Our son can use Ataluren, and he is about to stop walking. He didn’t participate in the
previous clinical trials, because the enrollment was closed before our son
could enter the trial. I know there are many other boys ”out there” in the same
position.

I am asking for several other parents coming from Norway,but also parents coming from other contries.

 

First we had this article from the Annual Meeting of the American Academy of Neurology, published

April 13th. This article didn’t say anything that this is going to be a follow-on clinical study of Ataluren, so I thought ohhhhh finally Ataluren will be on the market in the USA (if approved by the FDA):

 

 

AAN 2011: Ataluren Reduces Decline in Ambulation in Duchenne Muscular Dystrophy

Based on these results, PTC Therapeutics, the company that developed ataluren, is planning to seek approval for its use in DMD from the United States Food and Drug Administration. Because of
the mechanism by which the drug overcomes the effects of the mutation, it would
be appropriate for only about 13% of DMD patients, according to study presenter
Ted Abresch, Research Associate in Neuromuscular Disease at the University of
California at Davis.

 

The whole article published April 13th 2011

http://www.hcplive.com/conferences/aan_2011/Ataluren-Reduces-Declin...

 

Then today, this article was publised by Genzyme. They are talking about a follow-on open label clinical study for previous trial participants, in Europe.

 

Ataluren update from Genzyme

Follow-on open label clinical study announced for previous trial participants

As part of our continuing assessment, Genzyme plans to initiate a follow-on clinical study of ataluren in nmDBMD patients who previously participated in the clinical trials in Europe, Israel and Australia.
All patients who have previously participated in the clinical trials with
ataluren in nmDBMD, irrespective of their current clinical status, will be
eligible to participate.

We believe that this clinical study will allow for the collection of additional information on ataluren in nmDBMD while providing access to ataluren to all patients who have been involved in earlier clinical trials.
Currently we are working to develop the timelines and design for this clinical
study and we plan to update the community, with these additional details in the
very near future. We are reaching out to the original trial investigators to
make them aware of this new development and they may be in touch with eligible
families directly as well.

The whole article posted May 2nd 2011: http://www.actionduchenne.org/viewarticle?news=175

 

When/if Ataluren is approved by the FDA in the USA, is this an approval for all Stop-codon-boys in the USA ? Also those who didn’t participate in the previous clinical trials ?

When/if Ataluren is approvad by the European regulatory authorities, is this an approval for all Stop-codon-boys in Europe, also those who didn’t participate in the previous clinical trials ?

 

Can someone please help.

 

BeritSofie

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Stem cell trial: 

 

Unless this trial is being conducted, at least in part, in the US and has been reviewed by the FDA, I would be surprised if the FDA will accept the results (trials on clinicaltrials.gov do not have to be FDA-approved).  It looks like it is sponsored by a Chinese company and the only site listed is in China, but clearly based on your post they seem to be recruiting in other countries.   Just because something gets approved in another country does not mean it will be approved in the US automatically (for better or worse!).  But, in the defense of the investigators, sometimes placebos are not used if the risk is judged to be too great with no hope of benefit or the number of participants is so low that it's not practical--for example, gene therapy studies may inject a "placebo" into one muscle and the gene therapy vector into another in the same person, but they almost never have people who only receive a placebo injection--that may change if we ever get to phase II or III studies with gene therapy and there are larger numbers of participants.

 

Sharon

 

Hello Pat,

Thank you for your input. However, unless I have it seriously wrong, the trial that was called off two years ago was NOT supposed to be supported only by data coming from the high dose group. I rembember clearly that the trial was at all times presented as one involving a high dose, a low dose and a placebo. Moreover, the data coming out of the low dose group proved from the outset (they did not have to wait to find this out), that the low dose boys had "failed" to meet the 30m declination target by only 30 centimeters, i.e. a 1% (ONE PERCENT) shortfall. The trial would have been described as a great success by anybody endowed with common sense. Science should not be divorced from common sense. It is simply incredible that we are even discussing this. All of the other potential drugs and treatments are light years away. Only Ataluren is capable to slow down DMD or even improve the boys' condition here and now. 

Dear Pat,

Thank you for your through-full explanation.

I would like to comment on the issue of taking and accepting risks. Accepting all you have said, undoubtedly the risk acceptance varies widely from one individual to another. It is highly subjective issue and as you said can not be standardized. 

You are asking what risk we as parents and DMD boy themselves are accepting. Basing on what I have learned so far, the risk of taking  Ataluren against the risk of taking steroids is balanced. Furthermore, we as parents of DMD boys, are taking many risks: administering our children supplement, steroids. Question arise when to start, what dosage to use - it's our responsibility to decide. Some accept the risk other don't. but the risk is taken.

Thanks to this website we know more, eg. the last webinar "Identification of Efficient Therapies and Observation of Disease Severity" which sheds light on effects of use of supplements in DMD.

Yesterday were presented top line data from PTC. Ataluren in CF - success! http://ptct.client.shareholder.com/ReleaseDetail.cfm?ReleaseID=681445

Sharon - I hope that it's understood how much we all appreciate Pat. None of us have access to the FDA, so I think that we're all attempting to express thoughts that Pat will then put together into a coherent argument and champion for us in her conversations with them.

 

The one comment I want to make about the question of whether our sons are dying is this: drugs like exon skipping and ataluren (and many other drugs that aim to slow the rate of degeneration) seem to be only effective if given early enough in the progression. So what the FDA needs to understand is that while ambulatory boys who are candidates for these trials are not dying in this moment, if they don't get on the drugs before their tipping point, the end result in the same. These decision are indeed life and death...

 

And these are those of us who are desperately trying to keep our sons on their feet. Surely that is a worthy goal as well.

 

Mindy--good point. 

One of the things we've been pushing for is to allow parents better direct access to the FDA.  They absolutely need to hear from you and the way the system is set up now, although there are "patient advocates" in the system, they are not necessarily allowed to weigh in on the diseases by which their families are affected.  There is nothing in the code that says "we must have a patient affected by X disease involved in the decision making process for a drug designed to combat X disease."  I think that's nuts.   There was language to this effect in the Expert Act, but it was removed by either the House or the Senate, I forget which (Ryan would know).  It's very frustrating.

Can anyone explain why a patient in PTC trial could not request access to the low-dose Ataluren under compassionate use guidelines?

I realize the FDA cannot force a company to manufacture more drug for compassionate use, but if patient/advocacy organizations stepped up with $ to fund the manufacturing costs, would this be allowed under those rules?

-David

Is pulmonary function an endpoint (secondary) in the Duchenne study or are we just looking at 6 mwt and other skeletal muscle strength measures?

Blazena Kaderabkova said:

Yesterday were presented top line data from PTC. Ataluren in CF - success! http://ptct.client.shareholder.com/ReleaseDetail.cfm?ReleaseID=681445

Hi Ofelia, pulmonary function was a secondary endpoint in the original study--I don't know if it's being considered in extended access program.

How did the pulmonary results look, although 48 weeks might not be long enough. Wondering if they collected more data during the open access here in the US. 

Sharon Hesterlee said:

Hi Ofelia, pulmonary function was a secondary endpoint in the original study--I don't know if it's being considered in extended access program.

Ofelia--let me see if I can find any of my notes from presentations they've given at past meetings.  I'll get back to you on that.

David,

Anyone who was in the Ataluren trial in the US or Europe can request access to the drug and PPMD helped support the US program financially.

 

Sharon

 



David said:

Can anyone explain why a patient in PTC trial could not request access to the low-dose Ataluren under compassionate use guidelines?

I realize the FDA cannot force a company to manufacture more drug for compassionate use, but if patient/advocacy organizations stepped up with $ to fund the manufacturing costs, would this be allowed under those rules?

-David

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