Hi--Just wanted to introduce myself.  I joined PPMD in January as the Research and Advocacy Director after 11 years at MDA running their translational research and drug development programs.  At PPMD I'll be further developing work with drug companies, reviewing and growing the main research program and participating in advocacy intiatives.  I will also try to jump in as much as possible to answer questions about research and track down information about new treatments/therapies that anyone hears about.  One of my first projects along this line is to get to the bottom of this stem cell report from Costa Rica.  If anyone posts something that needs a response from me and you don't hear from me, you can always send me a nudge at sharon@parentprojectmd.org.  I'm really excited about the chance to focus exclusively on Duchenne and am looking forward to hearing from all of you.

 

Best Wishes,

Sharon Hesterlee

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Sharon, Do you have any idea what happened at the TACT meeting?

TACT will identify the first drugs for review by the end of 2009, and as a next step will call for applications to be submitted so that possible candidates may be discussed at the first drug review meeting on the 6th-7th February 2010.
The applications to be considered during the TACT review meeting scheduled for 6-7 February 2010 in Rome are:

1.Losartan
2.Isosorbide and NSAID
3.Flavocoxid.
All three drugs will be considered for indication in Duchenne Muscular Dystrophy.

Hi Ofelia--the TACT has met and is now putting together its recommendations to the applicants. I don't know what the time line will be like for that feedback, but TREAT-NMD is going to cover the meeting in detail in the next issue of their newsletter. I can probably find out and get back to you.
Thanks Sharon. I will read the next Treat-NMD issue.

Please let us know how the FDA call goes today and if there are any news about exon skipping approvals.


Sharon Hesterlee said:
Hi Ofelia--the TACT has met and is now putting together its recommendations to the applicants. I don't know what the time line will be like for that feedback, but TREAT-NMD is going to cover the meeting in detail in the next issue of their newsletter. I can probably find out and get back to you.
Hi--So I did learn that a workshop is in the planning phases for early September. It may end up being a collaboration between FDA and NIH, but it's not clear yet if there is funding for it. If NIH doesn't want to fund it, then the advocacy groups may be able to help fund it (not a problem). The time line can't be helped because of the red tape and approvals required. FDA can't tell me anything about exon-skipping approvals because they are not allowed to by law. They can only discuss specific applications with the applicants (ie, Prosensa/GSK and AVI). I'm working to set up a call with AVI and a follow up call with Prosensa to try to get those details.
What is this workshop supposed to do? Why is that needed? Does it have anything to do with the actual trials, except presenting the research performed so far, results obtained, discussing exon skipping as a "platform" drug etc.; similar to the one in EU? To be honest I am more interested in when these trials are starting. Discussing about "platform" drug approval will not speed up the approval if the trials are not starting. Let's be honest here, the FDA will not approve this as a platform until a number of exon skipping trials are performed. There are 2 going on in EU, 51 and 44, but none in US. So what is the point of discussing this in the Fall?

Sharon Hesterlee said:
Hi--So I did learn that a workshop is in the planning phases for early September. It may end up being a collaboration between FDA and NIH, but it's not clear yet if there is funding for it. If NIH doesn't want to fund it, then the advocacy groups may be able to help fund it (not a problem). The time line can't be helped because of the red tape and approvals required. FDA can't tell me anything about exon-skipping approvals because they are not allowed to by law. They can only discuss specific applications with the applicants (ie, Prosensa/GSK and AVI). I'm working to set up a call with AVI and a follow up call with Prosensa to try to get those details.
I'm sure the parents whose children do not have exon 51 or 44 deletions think speeding the approval process from a platform perspective is important. Not to downplay the importance of getting the current studies moving...
But you are correct that the workshop in the Fall is not intended to deal directly with the approval process for the trials planned by AVI and Prosensa. There is no public mechanism for dealing with those individual applications. The FDA isn't allowed to say anything even if all the delays are compeletely the fault of the applicant company. The only information comes from the companies and what they are willing to share (with their interpretation of events).
I am ALL for platform approval. What I meant is that this workshop will not help at all if a number of trials are not completed. So for me and other parents of boys needing 51, 44 or ANY other exon skipped, the actual start of the clinical trials is more important. There have been a number of workshops/meetings about exon skipping during the last two years and that did not help speeding up the approval process, which is what us (parents/patients) are interested in. So I lost my interest in them unfortunately.

It makes sense that only the FDA and the company applying for trial have access to that info. We all wait until AVI, Prosensa/GSK are ready to make an announcement, but the reality is that time goes a lot faster when you live with a progressive fatal disease.


Sharon Hesterlee said:
I'm sure the parents whose children do not have exon 51 or 44 deletions think speeding the approval process from a platform perspective is important. Not to downplay the importance of getting the current studies moving...
But you are correct that the workshop in the Fall is not intended to deal directly with the approval process for the trials planned by AVI and Prosensa. There is no public mechanism for dealing with those individual applications. The FDA isn't allowed to say anything even if all the delays are compeletely the fault of the applicant company. The only information comes from the companies and what they are willing to share (with their interpretation of events).
Hence the need to closely follow the current and future trials in the UK and other countries. The EMEA will open the floodgates for exon skipping and utrophin. At least we have them for hope.

The FDA only leaves us puzzled about why they choose to remain in the dark while science advances light years ahead. And we watch our children die. With their inaction they have written off thousands of American Duchenne patients who want to live. Its too late for the FDA to jump into the game now.

Plan on having many communications abroad, trips and class-action compassionate use appeals.
FDA won't save this generation so I say-
God Save the Queen !!
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
how long does a drug take to get from phase 3 to available? wont it just make sense to order pro051 when it hits the UK? we still have to order deflazacort outside of the US and US doctors have been prescribing it for years. i want to know who is responsible for delaying my son's treatment, he became a full time wheelchair user a few weeks ago... in october steve wilton said at a conference in London that he could treat ANY deletion in his lab RIGHT now!...WTF!

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
Hi Jenn,

My son isn't far behind yours, his walking is almost over. I think that any medication for a life threatening condition can be administered under compassionate use in either Phase I, II or III Clinical trials. But the catch is BioPharms have to agree to allow compassionate use. If you can get Prosensa or AVI to agree to administer exon skipping to your sons then you don 't have to wait out the clinical trial phases.

The tough part is getting them to agree before their trials are completed because the FDA requires them to tell all, every result good and bad. If, during a clinical trial, they agree to let you or anyone else use their chemestry set, and something goes wrong, it could kill their entire trial process and end potential use for everyone. But, I really don't know how it works with the EMEA or other regulatory agencies. I think you have a better chance of obtaining compassionate use during a Phase II or certainly phase III, when risks are diminished. I understand your anger, being not far behind you. But I wouldn't worry so much right now about who is to blame (save for later?), but perhaps instead spend energy finding ways to obtain comapssionate use.

My son has to wait longer, needing to skip 44 & 45 which means utrophin will be here before they mix exon skipping compounds. I would congratulate any family that can escape the torture of DMD with or without compassionate use.



jenn said:
how long does a drug take to get from phase 3 to available? wont it just make sense to order pro051 when it hits the UK? we still have to order deflazacort outside of the US and US doctors have been prescribing it for years. i want to know who is responsible for delaying my son's treatment, he became a full time wheelchair user a few weeks ago... in october steve wilton said at a conference in London that he could treat ANY deletion in his lab RIGHT now!...WTF!

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.

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