GSK trial of exon 51 skipping on Ambulatory Boys
Does anyone know what is going on with GSK and the trial on exon 51 skipping? They said the trial will not be conducted this year. Does that mean next year, or not at all? Have they pulled the plug on this trial? If they have, why the heck are they moving forward with the non-ambulatory trial which the FDA has green lighted.
Insight????
Replies to This Discussion
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Permalink Reply by Mindy on
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I don't think AVI is stringing anyone along. They are very open and honest about the fact that they are trying to figure out why some boys are responding more robustly to the drug than others. I think that once they have some ideas on that, and a protocol designed to test those ideas, they will move forward.
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Permalink Reply by Ofelia Marin on
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GSK's trial is Phase III hence closer to approval as opposed to AVI's trial. We do not have time.
arnold said:Avii is our best chance right now but folks seem to fixated on GSK
irishgirl said:That is what is so surprising. The FDA has already approved the trial for the non-ambulatory boys and that trial is already enrolling participants. GSK has said NOTHING other than the ambulatory trial with the same drug will not happen this year. Since the FDA has approved the one trial, it seems to make sense that they are waiting on that data before giving final approval to GSK for the Phase III we are all waiting for. The FDA is VERY wary about approving trial on humans. The FDA has neither approved or flatly denied anything yet - other than this non ambulatory trial. That is a start. We all know Canada and Europe are far more lax in their requirements for new drugs. The US healthcare is primarily run by the greedy pharmaceutical companies and they want to make money. How much do they stand to make on this new drug???
Mindy said:FDA turned them down. The FDA wants more data. GSK is responding by moving forward in the EU, and possibly Canada, only, rather than providing the FDA with what they want.
I'm so glad we have a government agency that will defend our right to die of a terminal disease so vehemently.
And yet I can still eat BPA in my food with no complaints from them at all...
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Sure, if you can afford you can have access to the treatment when approved in the EU or Canada or other country.
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Permalink Reply by Tonya on
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Does anyone have any facts what the FDA is basing their decision on? I swear I had heard that GSK/Prosensa may have had some toxicity issues that they couldn't go to any higher doses than 6mg. Had anyone else heard that? As far as I'm aware AVI didn't have any toxicity issues therefore they are approved to go as high at 100mg. It just seems AVI has something really good here. I would rather make sure there are no toxicity issues before having my son in a trial. I absolutely agree we don't have a lot of time here, then what can we do. I don't want to look at it like the FDA is willing to kill our boys. Statements like this I really find sickening, maybe you feel better throwing that out, but I want to know the facts. Is there something we don't know here about Prosensa's drug? why are they postponing? Let's find out.
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Permalink Reply by irishgirl on
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We can get the drug, if approved elsewhere like Deflazacort, but no insurance will cover it in the US and it could be upwards of hundreds of thousands of dollars each year. Compared to that, Deflazacort is super cheap!
AVI's trial is certainly not approved by the FDA either. AVI has begun connecting with people about the trial, but say they do not have FDA approval. The same thing with GSK could happen.
jenn said:will americans still have access to the drug if it is approved outside the US, like we do deflazacort?
Ofelia Marin said:Isn't this just disgusting? I wasn't this upset in a LONG time. I'm looking into moving out of this country. I am now convinced that any DMD medication will be approved here years after EU or Canada which should not be allowed! Why the h@#%^% doesn't my son have the same right to live as any other boy in other countries?
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And be ruled by the FDA. No FDA approval - no trial.
Mindy said:I don't think AVI is stringing anyone along. They are very open and honest about the fact that they are trying to figure out why some boys are responding more robustly to the drug than others. I think that once they have some ideas on that, and a protocol designed to test those ideas, they will move forward.
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There was a bit about toxicity and the kidneys, but they said it was no big deal. FDA asked GSK to provide more data - they did not possibly. AVI did provide more data. GSK was at the end of the trial line - much further along than GSK.
At this point, AVI is making moves calling possible participants ensuring folks have their ducks in a row. We were called by Dr. Violet (sp?) because our son is on the list. But they make no bones about the fact that AVI DOES NOT have FDA approval yet. THey can line kids up a mile long, but nothing will happen without the FDA approval.
Tonya said:Does anyone have any facts what the FDA is basing their decision on? I swear I had heard that GSK/Prosensa may have had some toxicity issues that they couldn't go to any higher doses than 6mg. Had anyone else heard that? As far as I'm aware AVI didn't have any toxicity issues therefore they are approved to go as high at 100mg. It just seems AVI has something really good here. I would rather make sure there are no toxicity issues before having my son in a trial. I absolutely agree we don't have a lot of time here, then what can we do. I don't want to look at it like the FDA is willing to kill our boys. Statements like this I really find sickening, maybe you feel better throwing that out, but I want to know the facts. Is there something we don't know here about Prosensa's drug? why are they postponing? Let's find out.
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Hey Tonya,
I don't think the FDA is willing to kill our boys - that's not what I'm trying to say. What I'm trying to say is that their model for risk assumption is inconsistent.
I would much prefer being fully informed about toxicity risks and allowed to make my own decision about participation in a trial for a product designed to treat a terminal disease. It's my understanding that they do that with cancer medication all the time. But I'm not allowed to assume risk on my son's behalf until it's absoutely proven to be safe.
In contrast, there are many chemicals and food products with long records of harm in both people and animals. BPA is a great example - you ingest it every time you have a soda or eat canned food. But the FDA will not pull it from the market until they have absolute, solid evidence of harm. So the public is forced to assume risk on behalf of the chemical industry until it's absolutely proven to be dangerous.
It's inconsistent at best.
I hope I'm explaining my objection properly. Let me know if I'm not...
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Permalink Reply by jenn on
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i agree mindy, when it comes to a terminal illness any possible treatment ought to be available to patients along with their doctors to choose to try...for example, fenergan is not approved during pregnancy but they didnt hesitate to prescribe it to me for nausea ( i didnt take it after looking up side effects online) we all should be in control over whet we decide to do, when provided the information on side effects. the side effects for birth control, diabetes drugs , steroids,and many others seen on tv are terrible, why are they FDA approved??!
Mindy said:Hey Tonya,
I don't think the FDA is willing to kill our boys - that's not what I'm trying to say. What I'm trying to say is that their model for risk assumption is inconsistent.
I would much prefer being fully informed about toxicity risks and allowed to make my own decision about participation in a trial for a product designed to treat a terminal disease. It's my understanding that they do that with cancer medication all the time. But I'm not allowed to assume risk on my son's behalf until it's absoutely proven to be safe.
In contrast, there are many chemicals and food products with long records of harm in both people and animals. BPA is a great example - you ingest it every time you have a soda or eat canned food. But the FDA will not pull it from the market until they have absolute, solid evidence of harm. So the public is forced to assume risk on behalf of the chemical industry until it's absolutely proven to be dangerous.
It's inconsistent at best.
I hope I'm explaining my objection properly. Let me know if I'm not...
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This is not akin to BPA. Avoid sodas and canned products and you avoid the risks that are associated. We are not even given the chance to avoid a certain drug here. Yes, we all want non toxic results and for the most part, this drug was clean up to a certain dosage. But positive results were achieved with small doses. At higher doses, there was something with the kidneys, but that was brushed off as safe and easy to deal with. We cannot compare apples to oranges. Feels like we are not being told the truth. Prosensa was extremely tight lipped about all the trial results from EU - while Mutoni reported dramatic results with no side effects. If the FDA wants more data - GSK should give it to them as AVI has done.
I apologize is I am coming across as harsh, but it is so hard to stay on this roller coaster from HELL. All of this is just that - a roller coaster ride from HELL. We are already in HELL because our children have been diagnosed with DMD.
Mindy said:Hey Tonya,
I don't think the FDA is willing to kill our boys - that's not what I'm trying to say. What I'm trying to say is that their model for risk assumption is inconsistent.
I would much prefer being fully informed about toxicity risks and allowed to make my own decision about participation in a trial for a product designed to treat a terminal disease. It's my understanding that they do that with cancer medication all the time. But I'm not allowed to assume risk on my son's behalf until it's absoutely proven to be safe.
In contrast, there are many chemicals and food products with long records of harm in both people and animals. BPA is a great example - you ingest it every time you have a soda or eat canned food. But the FDA will not pull it from the market until they have absolute, solid evidence of harm. So the public is forced to assume risk on behalf of the chemical industry until it's absolutely proven to be dangerous.
It's inconsistent at best.
I hope I'm explaining my objection properly. Let me know if I'm not...
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I agree we all should be able to make the choice, what I am saying is if we can get the information from either the FDA or GSK/Prosensa then we would be able to make an informed choice. Yes, it is a Hell we live in, I live it too. I believe we all deserve to know the truth about what the hold up is. Not to jump to a conclusion that the FDA is willing to let us die. I also agree that when it comes to terminal illness we should have the choice, but we want to know the facts about the choice we will make. I understand your point of view Irish girl!! I agree with you we should have the truth and have a choice to decide.
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This drug is safe enough that EU or Canada allow a clinical trial in children. If it would not be safe, they would not allow a Phase III trial. Let's be honest here, they would not allow to test a toxic drung in children period.
I understand GSK not wanting to gather more data in animals. They are starting a phase III drung in humans, why would they? Why would they want to collect 6 month toxicity data in mice and primates when they start a 2-year trial in kids?
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