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Major Move From FDA Puts Ampligen Back on Track

Reversing a 2009 decision some commentators felt was potentially crippling to Hemispherx BioPharma, Ampligen's producer, the FDA today stated they would not, after all, require a expensive 300 person study to assess Ampligen's effectiveness in chronic fatigue syndrome (ME/CFS). (Safety concerns appear to have been taken care of.) Instead they would allow Hemispherx to use new analyses of data it's already collected. Hemispherx must be breathing a huge sigh of relief.

A 300 person, double-blinded, placebo controlled drug trial was far beyond Hemispherx financial capacity, whose assets, Reuter's reported, fell from 58.1 million dollars in late 2009 to just 3.6 million dollars in March of this year.

The FDA imposed some major conditions on Hemispherx in 2009 but it could have rejected the application outright. Instead it chose to give the company more and more and more time; essentially keeping the door open. With this decision, which occurred after a recent meeting between the company and the FDA, the FDA opened the door a bit wider. Reuter's reported that Hemipherx's stock price soared 50% on the news.

It's not clear what circumstances prompted the FDA to change its mind but its been under fire for low rates of drug approvals over the past five years; in particular for drugs to treat chronic illnesses. With about a million people with ME/CFS in the US and government-funded studies suggesting as much as 20 billion dollars a year in economic losses, ME/CFS may be the only 'major' disorder without an FDA approved drug. In its report MarketWatch called ME/CFS a 'chronic, serious debilitating disorder". (The FDA recently agreed to a Stakeholder's Meeting to discuss its approach to chronic fatigue syndrome.)

In March an analyst noted that although the economics of ME/CFS treatment are unclear Hemispherx could be sitting on a multi-billion dollar chronic fatigue syndrome market worldwide. Even if just a portion of ME/CFS sufferers were treated with Ampligen the potential upside for the company and its shareholders is large indeed.

Marketwatch reported that upon news of the FDA's decision Hemispherx immediately began hiring more staff, consultants and independent contractors to prepare for the work ahead.

A 2010 study suggested Ampligen could take the place of drugs that expose some ME/CFS patients to cardiac toxicity. Other studies have shown Ampligen can significantly increase exercise tolerance.

The best drug never to get approved for ME/CFS suddenly appears to have a real chance. Hemispherx reported it will file its response in the fall of this year. The FDA typically takes up to six months to produce a decision.ar

Nothing is assured but with Hemispherx finally getting its day 'in court' and Rituximab trials ramping up the treatment possibilities for ME/CFS appear quite a bit brighter.

Tomorrow a blog reporting on Dr. Peterson's take on the drug with three of his Ampligen treated patient will appear (See Dr. Peterson Talks!)


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Comments

Awesome!

I bet our recent emails to FDA helped in part to motivate this positive decision. Sometimes it feels like we're banging our head against the wall with advocacy, at least to me, but I think this shows that our efforts will make a real, positive difference to millions of pwME in the coming years. Great job everyone! Let's keep up the good work!
 
Awesome!

I bet our recent emails to FDA helped in part to motivate this positive decision. Sometimes it feels like we're banging our head against the wall with advocacy, at least to me, but I think this shows that our efforts will make a real, positive difference to millions of pwME in the coming years. Great job everyone! Let's keep up the good work!
It does feel like we're banging our heads on the wall alot! I think its very possible we did make a difference. We were talking to Dr. Woodcock, after all - she is a top official at the FDA! However it happened this is Ampligen getting resurrected...there's no way Hemispherx would have been able to fund that big study....(Hemispherx did get its other product, Alferon approved for use in the US - that will come on the market later this year and that will help but they were still very low on cash...)..

Its a great decision! Good for the FDA - quickly becoming my favorite agency in the govt :)
 
This is really great news, Cort. The more potential treatment options we have the better. It kind of sounds like their safety concerns might have been addressed, so let's hope it goes through. Thx for posting this.
 
Do we have any idea of what percentage of us it helps, and how much? The history of chronic disease is painted with immoderate hopes applied to scraps of leftover cure.

Not to be the Debbie Downer here ;-)
 
Approving this drug will set a strong precedent. Here is hoping it happens!
 
Awesome!

I bet our recent emails to FDA helped in part to motivate this positive decision. Sometimes it feels like we're banging our head against the wall with advocacy, at least to me, but I think this shows that our efforts will make a real, positive difference to millions of pwME in the coming years. Great job everyone! Let's keep up the good work!
Is it all this banging on the head that is causing all my headaches? :)
I hope this happens. Not a day too soon.
 
Do we have any idea of what percentage of us it helps, and how much? The history of chronic disease is painted with immoderate hopes applied to scraps of leftover cure.

Not to be the Debbie Downer here ;-)
That was very poetically said How To Escape :). We're a big group and its only going to help some people; some people do very, very well - have you read Kelvin Lord's blogs on this site?, others do moderately better - and are quite grateful for that level of improvement and some it doesn't work for at all. I don't know the percentages, though....
 
That was very poetically said How To Escape :). We're a big group and its only going to help some people; some people do very, very well - have you read Kelvin Lord's blogs on this site?, others do moderately better - and are quite grateful for that level of improvement and some it doesn't work for at all. I don't know the percentages, though....
Its not just the treatment end that's important; getting Ampligen approved would put more focus on the immune side of this illness and should prompt studies to learn why it works at all in the people it does work for. Throw Rituximab in the mix and I imagine you'd have a disorder some top immunologists would start to get interested in...Ampligen approval could be catalyst for more treatment options, more interest in immune drugs in ME/CFS and more research...
 
Does anyone maintain their improvement once they're off Ampligen?
 
Hi. Interesting development, but I thought it wasn't quite so clear cut from the FDA - that they have agreed to put a reanalysis of the original data out for review which will make the decision, not that a further study won't be needed:
the FDA agreed to accept, for review, new analyses of data from Hemispherx's AMP-516 Phase III Clinical Trial ("AMP-516 Trial") in support of its New Drug Application ("NDA") for Ampligen® (Poly I : Poly C12U). If found sufficient to support approval of the drug, these new analyses will be in lieu of an additional confirmatory Phase III study called for in the Agency's November 25, 2009, Complete Response Letter ("CRL") . The FDA has advised that whether the new analyses provide adequate evidence of Ampligen® 's efficacy in treating Chronic Fatigue Syndrome ("CFS") will ultimately be a review issue...
From Yahoo finance
http://finance.yahoo.com/news/hemispherx-biopharma-fda-reach-agreement-120000248.html
 
Hi. Interesting development, but I thought it wasn't quite so clear cut from the FDA - that they have agreed to put a reanalysis of the original data out for review which will make the decision, not that a further study won't be needed:
Right, the main thing is that Hemispherx has the possibility of gaining approval for Ampligen without undertaking an expensive study which they don't have the money for. It doesn't mean that the FDA will not, after they review the data, ask for more studies.... - I imagine the options are a) could approve the application (b) ask for more data (c) reject the application in a way that would make it difficult for Hemispherx to get more money from investors...( the FDA does do this at times; they have not done that with Ampligen).

Hemispherx does have some rodent studies the FDA wants which it stated will not be done by the third quarter...
 
Does anyone maintain their improvement once they're off Ampligen?
Bob Miller, in a blog that's coming up, told me Dr. Peterson told him that 1/3 of the patients on Ampligen get better, a third maintain their improvements and a third eventually relapse...It can take several years for someone to relapse.
 
Is that a third recover fully and stay recovered, a third improve somewhat and maintain the improvement and a third improve at least somewhat and then relapse? Sorry to nitpick, just really interested!
 
Is that a third recover fully and stay recovered, a third improve somewhat and maintain the improvement and a third improve at least somewhat and then relapse? Sorry to nitpick, just really interested!

:)...about a third show further improvement after they get off the drug....I don't know if anyone has recovered fully using it....my guess, based on what Dr. Peterson said, that he doesn't know any patients that are 'fully' recovered...patients now leading normal lives - yes, but apparently with some restrictions...
 
Is that a third recover fully and stay recovered, a third improve somewhat and maintain the improvement and a third improve at least somewhat and then relapse? Sorry to nitpick, just really interested!

:)...about a third show further improvement after they get off the drug....I don't know if anyone has recovered fully using it....my guess, based on what Dr. Peterson said, that he doesn't know any patients that are 'fully' recovered...patients now leading normal lives - yes, but apparently with some restrictions...
Thanks, Cort - I see what you meant now!
 
Do we have any idea of what percentage of us it helps, and how much? The history of chronic disease is painted with immoderate hopes applied to scraps of leftover cure.

Not to be the Debbie Downer here ;-)
For me, Ampligen helped cognitive function the most, improved physical functioning moderately, and didn't improve pain or sleep.

There are some patients, a minority, who get a nearly complete remission of all their symptoms on Ampligen. I was not one of those ones, but I found the drug to be very useful and worth the cost.
 
Does anyone maintain their improvement once they're off Ampligen?
Bob Miller, in a blog that's coming up, told me Dr. Peterson told him that 1/3 of the patients on Ampligen get better, a third maintain their improvements and a third eventually relapse...It can take several years for someone to relapse.
Most people maintain the improvements after stopping Ampligen, but it usually isn't permanent. For example, if you take the drug for a year and respond, you can expect to feel better for another year or two after you stop taking it. Most will eventually relapse if they are off it long enough.
 
Most people maintain the improvements after stopping Ampligen, but it usually isn't permanent. For example, if you take the drug for a year and respond, you can expect to feel better for another year or two after you stop taking it. Most will eventually relapse if they are off it long enough.
Why do you think that is?