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 Keith Van Houten on May 31, 2010 at 10:15pm
Would a drug provided under an IND typically be provided by the company at no charge?
Comment by Ofelia Marin on May 30, 2010 at 10:15am
This is very good indeed!

I am looking forward to see the biopsy data. Not much more wait at this point.

Comment by Pat Furlong on May 30, 2010 at 7:58am
Here's the thing. The access program will be for those who were involved in the trials. While it would be possible to open to individuals with stops, there has to be a regulatory path forward - approval. This is not a unilateral decision, rather a thoughtful decision in terms of the path forward. Please keep in mind this is ONE step forward in terms of an access program and in parallel preparing the regulatory package. Also keep in mind, it is a difficult and fine line. While you are suggesting they open to all eligible dmd boys, a percentage of all genetic disease (10-30% or 6000 rare diseases) is caused by a premature stop. So, where do you draw the line? As you can imagine, they receive calls from people with genetic disease requesting access. They have to have to proceed on this regulatory path forward, as difficult and frustrating as it feels. Approval is the only way for sustained access for all.

I also understand 3 months feels very long in terms of analysis, but actually analysis is a very complex process with many moving parts. PTC has consulted with leaders in statistical analysis and it has been a difficult and painful process. They are working night and day to make sense of the data. The regulatory package has to be RIGHT, has to meet statistical relevance and sufficiently compellling for the regulatory agencies to approve Ataluren.
During the call you should have heard that the low dose was statistically significant. And you are correct Ofir, the regulatory agency may require a new trial in order for approval. That is yet to be determined. .
And Tracy, your son will have the opportunity if this drug is approved and we are talking about a time-frame of months at this moment. I understand your frustration, but cross fingers/pray, Ataluren is approved and your son will be among the first to have a treatment. From my heart to yours, this would be amazing. Count your blessings.
Comment by Tracey Hartz on May 29, 2010 at 9:15pm
So there's no chance of a boy who wasn't in 2a or 2b to get an IND? That is so extremely frustrating. My son just got diagnosed last summer, it was already closed.
Comment by Ofir Arad on May 29, 2010 at 8:43pm
It seems that the problem now is to interpret the results of the trial. Almost three months after the trial was suspended we don't have the conclusions of this trial. I think that to make individual trials can be positive to this 170 boys but the results may be even more confusing that the results that we have now. I don't know if it's possible but maybe it would be more logic to begin a new coordinated trial to find out what is exactly the optimum dose.
Comment by Michelle on May 29, 2010 at 7:56pm
Why limit IND access to 170 boys? This is a very, very small group of boys we're talking about. Only 10-13% of the whole DMD population. Would it really be that unmanageable to have double that number via expanded access? The individual site physicans are taking responsibility for administration of the drug anyway. If PTC is pursing the regulatory path forward, they must believe there is some merit to the time, trouble and most importantly, expense, involved. They must believe this drug will work at the low dose to even consider making it available via IND. Ask them to collect data on how many others (non trial boys) would file an IND if allowed. At least know what sort of demand you're looking at before making a unilateral decision to provide the drug subjectively. Maybe it isn't that many extra as they think.
Comment by Susan Rathfelder on May 28, 2010 at 6:37pm
Comment by Ofir Arad on May 28, 2010 at 5:16pm
What is an individual IND?

Comment by Pat Furlong on May 28, 2010 at 5:04pm
CLARIFICATION!!! - this is ONLY for families who have participated in the Phase 2a, 2b and those who participated in the non-ambulatory trial. PTC has to focus on these individuals first and completing the analysis/package for the regulatory path forward

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