FDA Approval of Exon Skipping
I realize that this is a little premature, but since I have a 10 year old who is predicted to benefit from skipping either exon 43 or 45, but not 51, I am more than a little concerned that he'll be on a ventilator and gi tube before completion of clinical trials for a compound that will skip exon 43 or 44. I spent a godawful amount of money on a book on FDA law, and reached the conclusion that the FDA probably has discretion to treat the Prosensa compounds as one drug, even where they are designed to skip different exons, and the would apply to AviBioPharma's compounds. However, there is nothing in the law that would require the FDA to do so. Does anyone know whether these two companies or any of the patient advocacy organizations have begun talking to the FDA about whether they will require separate trials for each compound? I have heard a great deal of rumor and speculation, but I really want to know whether there is any reason to be optimistic, short of going to Congress for a legislative change. If we need a legislative change, then that ball needs to get rolling next year, assuming that the Phase I/II's for ProSensa and AviBioPharma come in as expected in December and are positive.
Replies to This Discussion
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Permalink Reply by MicahsDaddy on
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"but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room..."
Hi Cheryl,
I dunno...
If in trial stages, and deathly ill, then Compassionate Use can be done...as long as the 3 requirements are met.
If FDA approved (no longer in trials), then whoever is in charge would possibly make the pharma's lower the cost of production and get insurance companies to accept exon skipping as a treatment. I don't know how all that works, but I did ask about this and was assured that the cost would not be $250,000 per treatment if exon skipping was FDA approved.
Need someone else whose smart on those sides of the lines to tell us how that works.
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Permalink Reply by Ofelia Marin on
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Cheryl,
What I meant is that the cost of the treatment should be a lot LOWER otherwise the pharma companies will not make profits. I am confident that when a treatment becomes reality all the boys will have access to it. Keep in mind that this is a fatal disease (w/o any treatment or cure at this point) so insurance should cover it/part of it.
I also think that they will not be able to approve it only in EU; they need more patients for exon skipping trials, there aren't as many as they need for phase III in EU alone.
Ofelia
cheryl cliff said:Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
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"Ofelia and Christian-
You guys are extremely smart"
I dunno about Ofelia, but as for me, I did stay in a Holiday Inn Express last night...I dunno about anyone else, but i crack myself up...ugh, too much TV.
I am no different than you. I used to ask all the same questions you do. I used to believe 100% in exon skipping.
Where I still believe in it, I believe more in others right now because IF they work, then it would be ready sooner for all DMD patients, not selective percentages. At the rate exon specific treatment is going, it will take what seems like forever to get to all possible skips....and still be leaving 2 behind.
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Hehe. You're too funny Christian! :-)
I agree with Christian. I too have tons of questions and no answers. The sad part is that even the big names in this field, Wilton, Mendell did not have a lot of answers for my questions at this point... I got a "we are on a very steep learning curve right now" from Mendell though. :-)
To be honest, based on what we know at this point, I think that it will take a cocktail (and a good one!) to treat DMD. We should learn more about exon skipping during the next 1-2 years or so.
MicahsDaddy said:"Ofelia and Christian-
You guys are extremely smart"
I dunno about Ofelia, but as for me, I did stay in a Holiday Inn Express last night...
I am no different than you. I used to ask all the same questions you do. I used to believe 100% in exon skipping.
Where I still believe in it, I believe more in others right now because IF they work, then it would be ready sooner for all DMD patients, not selective percentages. At the rate exon specific treatment is going, it will take what seems like forever to get to all possible skips....and still be leaving 2 behind.
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Permalink Reply by cheryl cliff on
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Hi Ofelia,
I agree with everything you wrote. I wasn't expecting exon skipping to only happen in the UK. If FDA takes another 5-10 years to get "our" version thru I can't wait that long, Alexander can't wait that long. He just turned 10 and by good standards his life is half over. He is not on his death bed right now so if it takes a little more time we might be ok. How many patients do they need for phase III trials - would you happen to know?
cheryl
Ofelia Marin said:Cheryl,
What I meant is that the cost of the treatment should be a lot LOWER otherwise the pharma companies will not make profits. I am confident that when a treatment becomes reality all the boys will have access to it. Keep in mind that this is a fatal disease (w/o any treatment or cure at this point) so insurance should cover it/part of it.
I also think that they will not be able to approve it only in EU; they need more patients for exon skipping trials, there aren't as many as they need for phase III in EU alone.
Ofelia
cheryl cliff said:Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
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Permalink Reply by Paul Cliff on
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Thanks, I had remembered seeing it in that article. I think that's just an indirect way to fund the R&D by making the people with research grants participate in the cost of validating the compound. AviBIOpHarma, or however the bleep they spell their name, has annual revenue in the $20 Million Dollars range and has only a relatively small amount of VC and grant money to pursue this, and has never shown a profit. I'm sure that they justify charging this guy up the wazoo in some way.
MicahsDaddy said:"Where does the $250k/treatment number come from."
Paul, I read it as a quote from someone significant to this type of research. I can't recall who, but it was last year when I thought I had what it took to become a researcher (LOL) that I came across it.
It was not just someone on a message board that pulled it out of their woo hoo and threw it up.
I will try to find it again and send you the link....
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I'm big on cocktails. I don't question that they're needed, though ethanol works better for me than these weird long chain organic compounds. Like Cheryl said, I'm just trying to gauge Alexander's future prospects. It's a lot like a mutual fund, where you spread your risk among various investments on the belief that the odds of them all tanking at the same time is diminished.
Ofelia Marin said:Hehe. You're too funny Christian! :-)
I agree with Christian. I too have tons of questions and no answers. The sad part is that even the big names in this field, Wilton, Mendell did not have a lot of answers for my questions at this point... I got a "we are on a very steep learning curve right now" from Mendell though. :-)
To be honest, based on what we know at this point, I think that it will take a cocktail (and a good one!) to treat DMD. We should learn more about exon skipping during the next 1-2 years or so.
MicahsDaddy said:"Ofelia and Christian-
You guys are extremely smart"
I dunno about Ofelia, but as for me, I did stay in a Holiday Inn Express last night...
I am no different than you. I used to ask all the same questions you do. I used to believe 100% in exon skipping.
Where I still believe in it, I believe more in others right now because IF they work, then it would be ready sooner for all DMD patients, not selective percentages. At the rate exon specific treatment is going, it will take what seems like forever to get to all possible skips....and still be leaving 2 behind.
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Hi Christian-
It's ok not to believe in exon skipping 100%, I don't believe in it 100% either. It is just the next one coming up on the menu, albeit in the UK, and might possibly buy Alexander enough time until the something better comes along. Personally I think most of these medications/treatments have a high likelyhood to be as toxic as 20mg prednisone daily or worse. Probably one treatment won't fit everyone and we should have many more options to select from.
In the meantime, I gotta keep Alexander alive
cheryl
MicahsDaddy said:"Ofelia and Christian-
You guys are extremely smart"
I dunno about Ofelia, but as for me, I did stay in a Holiday Inn Express last night...
I am no different than you. I used to ask all the same questions you do. I used to believe 100% in exon skipping.
Where I still believe in it, I believe more in others right now because IF they work, then it would be ready sooner for all DMD patients, not selective percentages. At the rate exon specific treatment is going, it will take what seems like forever to get to all possible skips....and still be leaving 2 behind.
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Hi Cheryl,
I think they need a number in the 100's since DMD is orphan disease...something similar with what they have for PTC124 Phase 2b. Actually I think that they might not even need a Phase 3 if they see significant benefit in boys, Phase 2b would be as far as they need to go in that case.
I too hope they come up with something (anything!) that will buy time for our boys soon. We'll also go to EU or anywhere else if something is approved there faster.
I have some hope in exon skipping since it was applied systemically in dogs and monkeys and they did see improvements and no immune response. I whish they would start this UK trial faster to get some answers.
cheryl cliff said:Hi Ofelia,
I agree with everything you wrote. I wasn't expecting exon skipping to only happen in the UK. If FDA takes another 5-10 years to get "our" version thru I can't wait that long, Alexander can't wait that long. He just turned 10 and by good standards his life is half over. He is not on his death bed right now so if it takes a little more time we might be ok. How many patients do they need for phase III trials - would you happen to know?
cheryl
Ofelia Marin said:Cheryl,
What I meant is that the cost of the treatment should be a lot LOWER otherwise the pharma companies will not make profits. I am confident that when a treatment becomes reality all the boys will have access to it. Keep in mind that this is a fatal disease (w/o any treatment or cure at this point) so insurance should cover it/part of it.
I also think that they will not be able to approve it only in EU; they need more patients for exon skipping trials, there aren't as many as they need for phase III in EU alone.
Ofelia
cheryl cliff said:Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
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I think we're back full circle here. I am going to sniff around and find out what is publicly available concerning the NDA applications on file with the FDA for everything that's in the pipeline and figure out whose desk it's sitting on and send him or her a little reminder of what's at stake.
Ofelia Marin said:Hi Cheryl,
I think they need a number in the 100's since DMD is orphan disease...something similar with what they have for PTC124 Phase 2b. Actually I think that they might not even need a Phase 3 if they see significant benefit in boys, Phase 2b would be as far as they need to go in that case.
I too hope they come up with something (anything!) that will buy time for our boys soon. We'll also go to EU or anywhere else if something is approved there faster.
I have some hope in exon skipping since it was applied systemically in dogs and monkeys and they did see improvements and no immune response. I whish they would start this UK trial faster to get some answers.
cheryl cliff said:Hi Ofelia,
I agree with everything you wrote. I wasn't expecting exon skipping to only happen in the UK. If FDA takes another 5-10 years to get "our" version thru I can't wait that long, Alexander can't wait that long. He just turned 10 and by good standards his life is half over. He is not on his death bed right now so if it takes a little more time we might be ok. How many patients do they need for phase III trials - would you happen to know?
cheryl
Ofelia Marin said:Cheryl,
What I meant is that the cost of the treatment should be a lot LOWER otherwise the pharma companies will not make profits. I am confident that when a treatment becomes reality all the boys will have access to it. Keep in mind that this is a fatal disease (w/o any treatment or cure at this point) so insurance should cover it/part of it.
I also think that they will not be able to approve it only in EU; they need more patients for exon skipping trials, there aren't as many as they need for phase III in EU alone.
Ofelia
cheryl cliff said:Ofelia and Christian-
You guys are extremely smart but do you really believe that a treatment, regardless of who own's it, that will snatch a child from certain death would be denied because of inability to pay? Yea, drug companies are in this for profit, but they can't make much if their customers die. I think they know the moral issues at hand. Besides, if they did behave badly I'd like to see them handle a mob of angry parents in a court room...
We've already seen what a judge will do with this issue and PTC124.
cheryl
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Permalink Reply by Kelvinsmom - Michelle on
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After asking questions this year of both Dr. Mendell and Dr. Wong, I understand that Exon Skipping is actually doing very well and that most likely, each different exon skipping # will not take a separate clinicial trial, meaning that if exon 51 works great, then they can move on (well, this is what doctors think if they did the preclinical testing proper enough, but you never know the FDA). Even though each exon skipping is different, the process is still the same. I know for a fact that there are a few other exon skipping #'s already done and ready, but that they can't move on until exon 51 clinical trial is finished. I don't think it will be very long, maybe within 2 years, but, again, as I understand, some deletions will fair much better than others with this process and possibly some won't fair much better (per the doctors). I believe they'll move on with creating the exon skipping for those that will work the best and will benefit the most kids, then continue down the line. There are 79 exons, so this will take a lot of different Exon Skipping. Michelle
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Please listen/read AVI's presentation. There are lots of answers to our questions!!!
Transitioning an Antisense Pioneer into a Leading RNA–based Drug Discovery and Development Company
September 10, 2008
http://www.avibio.com/pr/pr390.php
Leslie Hudson, President and CEO, AVI BioPharma mentions exons 51, 45, 44, 53, 46 and 50.
Ofelia
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