Major Move From FDA Puts Ampligen Back on Track

July 11, 2012

Posted by Cort Johnson

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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29 comments

{ 29 comments… read them below or add one }

justinreilly July 11, 2012 at 6:47 pm

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!

Cort July 11, 2012 at 6:55 pm
justinreilly

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 :)

jeffrez July 11, 2012 at 7:26 pm

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.

HowToEscape? July 11, 2012 at 8:48 pm

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 ;-)

Snow Leopard July 12, 2012 at 12:58 am

Approving this drug will set a strong precedent. Here is hoping it happens!

Nielk July 12, 2012 at 4:00 am
justinreilly

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.

Cort July 12, 2012 at 5:47 am
HowToEscape?

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….

Cort July 12, 2012 at 5:50 am
Cort

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…

Sasha July 12, 2012 at 5:51 am

Does anyone maintain their improvement once they're off Ampligen?

Simon July 12, 2012 at 5:54 am

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

Cort July 12, 2012 at 6:51 am
Simon

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…

Cort July 12, 2012 at 6:52 am
Sasha

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.

Sasha July 12, 2012 at 7:08 am

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!

Cort July 12, 2012 at 9:09 am
Sasha

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…

Cort July 12, 2012 at 9:11 am

Check out Phoenix Rising's new Ampligen Resource Center

Sasha July 12, 2012 at 9:51 am
Cort
Sasha

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!

satoshikasumi July 12, 2012 at 9:58 am
HowToEscape?

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.

satoshikasumi July 12, 2012 at 10:00 am
Cort
Sasha

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.

Nielk July 12, 2012 at 10:05 am
satoshikasumi

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?

satoshikasumi July 12, 2012 at 10:07 am
Simon

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

The FDA ought to have fast-tracked Ampligen for approval based on the phase II results back in 1998. They didn't. This was partly due to the perception that CFS was not a serious disease.

Fifteen years later, CFS remains a crushing burden on the economy and no other drug companies have even gotten through a phase II study. There are no prospects for a treatment being approved for CFS in the next five years other than Ampligen.
Other treatments being considered are much more dangerous than Ampligen.

The agency said no in 2009 but they have a lot of discretion in how strictly they scrutinize drugs. They should consider the burden of CFS on society and the failure of any other drug company to make progress and give Ampligen the benefit of the doubt.

5150 July 12, 2012 at 7:06 pm
HowToEscape?

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 ;-)

how to avoid being debbie downer in our position? a difficult assignment, at which i am largely failing. my main roadblock is knowing that many people who have our damnable illness do not have the kind of medical coverage required to pay for Ampligen, so what's the difference anyway? after all, almost nobody has the kind of money that the Whittemores spent on their daughter. after 30 years of fighting, i am very tired indeed… and it is a big job to remain positive after seeing all the BS that comes with our trying so valiantly to get some help.

best wishes to the younger ones. that's about as positive as i can muster up for now.

Snow Leopard July 12, 2012 at 9:39 pm

It's all about building momentum. Approving a drug specific to ME/CFS is a big hurdle that will be very positive if achieved.

MishMash July 13, 2012 at 1:14 am

This is definitely good news. I won't be able to afford it, but it'll definitely be judged in the court of patient opinion.

I'm hoping Dr. Peterson's subjects were long-term, intractably ill patients. Because the CFIDS Assoc stated on their web page (http://www.cfids.org/resources/newly-diagnosed-faq.asp) that a third of all persons who become ill recover completely: "…About one-third of persons with CFS recover almost fully, but no one knows why. Some people recover in small to moderate degrees, while others recover minimally, if at all…."

Cort July 13, 2012 at 6:59 am

That may be true but, on the other hand, some do – perhaps many – and an Ampligen approval provides them with a new treatment option. If you're low income the Affordable Health Care Act should provide you with insurance at very low or no prices beginning in 2014 providing its not repealed.

satoshikasumi July 13, 2012 at 7:38 am
5150
HowToEscape?

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 ;-)

how to avoid being debbie downer in our position? a difficult assignment, at which i am largely failing. my main roadblock is knowing that many people who have our damnable illness do not have the kind of medical coverage required to pay for Ampligen, so what's the difference anyway? after all, almost nobody has the kind of money that the Whittemores spent on their daughter. after 30 years of fighting, i am very tired indeed… and it is a big job to remain positive after seeing all the BS that comes with our trying so valiantly to get some help.

best wishes to the younger ones. that's about as positive as i can muster up for now.

Most drug companies will have a patient assistance program to give the drug free to uninsured, poor patients after the drug is approved. If Ampligen is approved, it will be very profitable and it is likely that Hemispherx would create such an assistance program.

Second, if Obamacare is not repealed, you will be able to buy health insurance that will cover Ampligen in 2014. Please don't give up hope!

The main barrier is the FDA's approval process. If it can pass that hurdle, you'll almost certainly be able to get it one way or another.

satoshikasumi July 13, 2012 at 7:41 am
Cort

That may be true but, on the other hand, some do – perhaps many – and an Ampligen approval provides them with a new treatment option. If you're low income the Affordable Health Care Act should provide you with insurance at very low or no prices beginning in 2014 providing its not repealed.

Not to mention that most approved drugs for serious conditions have a patient assistance program to help people who cannot afford the medicine.

This is how some patients are getting access to drugs like Valcyte now.

Hemispherx would be able to create a patient assistance program if their drug was approved and profitable.

Cort July 13, 2012 at 10:29 am

Dr. Peterson talks on Ampligen and we have stories from three Ampligen patients on the latest article here.

snowathlete July 13, 2012 at 10:40 am

i guess its still three years from being available in the UK on the NHS, if everything pans out…
Still, better than nothing.

satoshikasumi August 3, 2012 at 6:34 am
Nielk

Why do you think that is?

Almost no drug for chronic diseases has permanent effects. Immune-modulating drugs are almost always reversible (which is a very good thing for people who have bad side effects). Ampligen changes the functioning of the rnase-l system and improves natural killer cell function. If the virus theory is correct, this allows the patient's immune system to reduce herpesvirus levels over time. When treatment is stopped, the immune system is no longer being modified by Ampligen and the viruses can begin reproducing again. If the autoimmune theory is correct, ampligen shifts the immune system into a mode where it produces fewer autoantibodies. They are gradually depleted during treatment. When treatment is stopped, the body starts gradually producing the bad stuff again until the patient gets sick.

There is a long delay both in responding to Ampligen and in relapsing after you stop it.

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