Ataluren Update

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.