As many of you know, because you participated, PTC held an open conference call yesterday to update the community about ataluren and future plans. The discussion included analysis of data from the ataluren trial and a summary of regulatory challenges. Langdon Miller (Chief Medical Officer for the trial) described PTC’s plan for regulatory interactions relating to ataluren and the effects of these interactions on potential patient access to ataluren.

And when the call was over, like many of you, I walked away from the call still frustrated, still hurting for our boys.

I understand the urgency to complete the sub-analysis and to prepare the package to establish the path forward. Like you, I worried we would again be waiting until the presentation in Naples and another call.

Time. And like you, I have watched my own two sons lose strength and felt my heart break over and over again for them and for all of you.

I reviewed this conversation in my head throughout the night and today. I wanted to have something concrete to hold onto and step-by-step I went through the call. And I made some calls.

Here is what I understand:

By the Naples meeting PTC will have completed the analysis and prepared the package for the regulatory path ahead. Keep in mind, while on the call, PTC talked about an access program and mentioned Individual IND’s. I think the risk/benefit of the individual IND is worth pursuing as each day these boys lose function. You already know, I think in increments of 5 minutes: treasuring every one of those moments and hoping to buy more. I know all of you feel the same.

This morning I spoke with PTC and asked that question in order to get some details and clarification. PTC confirmed that they are planning for individual IND’s. Planning is being done in parallel to data analysis and preparing the regulatory package. PTC is interested in understanding who/how many of the families are interested in securing an individual IND. Please contact your site coordinator and ask if they would be willing to participate in an individual IND.

I know what you are thinking, that you may have another willing physician. At the moment, PTC wants to understand:
  1. Who is interested in an individual IND, and
  2. If the physician from your clinical site would be willing to act as the sponsor.
Please send this information to Diane Goetz.

From the FDA SITE: The sponsor is the person who takes responsibility for and initiates a clinical investigation. The sponsor may be a pharmaceutical company, a private or academic organization, or an individual. A Sponsor-Investigator is an individual who both initiates and conducts a clinical investigation and under whose immediate direction the investigational drug is being administered or dispensed. For administrative reasons, only one individual should be designated as sponsor.

I feel – PPMD feels – that it is our job to tell your stories to PTC and any other company working in Duchenne. And we will.

I have said it before – I continue to believe in the work of PTC. I believe they are committed to helping our sons and I truly credit them with being trailblazers in Duchenne treatment. I also commend them for their communication with us. Most pharmaceutical companies would remain silent in a situation like this and while we may be frustrated by the information we receive or the amount of information we have received, at least we are not listening to silence.

But we won’t sit still and we won’t sit quietly.

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Comment by Blazena Kaderabkova on June 6, 2010 at 3:55pm
do you know how it would be in Europe?
Comment by ferre on June 4, 2010 at 7:15am

i would like to contact parents whiose kids were on ataluren and who didn't live en USA or canada

could you please contact me on my e mail address

Comment by celia garcia gomez on June 3, 2010 at 4:51pm
Comment by celia garcia gomez Delete Comment 23 hours ago are my PTC Participate in the Phase 2b trial and the three-month extension 2b taking the high dose, Were Maintained, Since the trial ended, You Have less strength, more fatigue, a large muscle loss in the lower Extremities very fast "Casula" What is it and it is Called an individual index
Comment by Melanie Sunny on June 3, 2010 at 11:12am
Thanks Pat. Melanie:)

Comment by Pat Furlong on June 2, 2010 at 9:40pm
yes, it is the package that will be used for the regulatory path (fda).
I'm sorry Celia. I do not speak Spanish.
Comment by Melanie Sunny on June 2, 2010 at 6:11pm
Dear Pat,
Thank you very so much!
Is the package which PTC is preparing for FDA drug approval?
Kindly Regards,
Comment by celia garcia gomez on June 2, 2010 at 5:07pm
Mi hijo participo en el ensayo PTC fase 2b y los tres meses de 2b extensión tomando la dosis alta, se mantenia ,desde que termino el ensayo, tiene menos fuerza,se fatiga más, una gran perdida muscular en las extremidades inferiores muy rápida ¿Casulidad? ¿ Qué es y a que se llama un IND individual ?

Comment by Pat Furlong on June 2, 2010 at 4:10pm
Dear Friends,
to clarify - ptc is finalizing the analysis and preparing the package. They are, in parallel, considering the acess program - how it would work. They have asked the patients /families who participated in the clinical trials to contact their sites and to notify ptc about their interest. In July, ptc presents during the Naples meeting, at which time the package will be finalized. In JULY, after the NAPLES meeting, there will be additional information/details forthcoming about the access program.

There is no access to Ataluren at this time.

Comment by Pat Furlong on June 2, 2010 at 12:52pm
Sharon, PTC is competing the analysis. They are preparing the package required for regulatory agencies. In parallel they are working on an access program. They will present final data in July/Naples meeting and shortly thereafter (with approval for the regulatory path forward) they will open the access program.

Keith - sorry I do not have the answer to your question as the program is in development. I think all of us would be willing to step up to the plate if needed.
Comment by sharon Christiansen on June 2, 2010 at 12:48pm
Pat I had some therapy to attend to with my boy and therefore missed the conference call. Joey was in the 2b trial out of Vancouver, he was on the low dose and we did see some improvement. I'm wondering if we are able to receive the drugs through this IND? Please any info to enlighten me would be very much appreciated. Thank you

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