Ampligen On the Clock: Hemispherx's 'Complete Response' Means Ball is Now in FDA's Court Now

[caption id="attachment_12929" align="alignright" width="325" caption="By end the January, 2013, at the latest,we will know if the FDA will approve Ampligen for ME/CFS..."]
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Moving quickly, Ampligen's producer, Hemispherx Biopharma, filed its 'complete response' to the FDA's request for data just 53 days after the agency agreed to relax its requirements for review of the drug. Hemispherx's 'complete response' means we will know by the end of January and perhaps sooner whether Ampligen becomes the first FDA approved drug for chronic fatigue syndrome (ME/CFS).

New Data

In its response Hemispherx provided new data derived from a 24 week extension period occurring after the 40 week original trial. Hemispherx stated the data will show that patients who remained on the drug for a further 24 weeks showed further improvements in endurance (23% - 39% increase in treadmill duration) suggesting that the drug's effectiveness increases over time.

Similarly, Hemipherx reported that patients who responded well to Ampligen showed significantly greater improvement relative to non-responders in areas such as vitality, general health, Karnofsky performance (functionality) and activities of daily living the longer they were on the drug. This is important because the original study did not highlight improvements in functionality and well-being. This data suggests Ampligen works 'globally' to improve both endurance and functionality.

FDA and Ampligen

It appears that the FDA has at least several possibilities;
  • it can approve the drug
  • it can reject the drug
  • it can give the drug 'accelerated approval' status - in this scenario Ampligen is not approved but Hemispherx is allowed to market the drug and gather more data for a final attempt at approval
  • it can ask Hemispherx for more data - without the income derived from 'accelerated approval' this outcome would be difficult for Hemispherx.
Hemispherx's Press Release suggests the company is gunning for accelerated approval status. While the FDA did recently substantially relax its requirements to consider Ampligen for approval its possible, perhaps even probable, they still want that large, multi-dose, placebo-controlled, double-blinded treatment trial originally required in their 2009 report. Hemispherx didn't come anywhere near providing that kind of data but getting accelerated approval would give them the means to do so.

The FDA has increasingly come under fire to approve more drugs and, in particular, to approve more drugs for chronic illnesses and the new law provided drug companies an opening to do just that.

New Law Presents Opportunity for Hemispherx and Ampligen

In their press release Hemispherx highlighted the fact that the 2012 Food and Drug Safety and Innovation Act (FDSIA) law encourages the FDA to take 'innovative and flexible approaches' to assessing treatment for 'serious' disorders with 'unmet' needs.

In section 901 from the FDASIA Act below note the emphases on 'expedited...review' and the need to target subpopulations.

"the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions... This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation
Patients benefit from expedited access to safe and effective innovative therapies to treat unmet medical needs for serious or life-threatening diseases or conditions.​

Indeed, recent history suggests a drug need be effective in only a small group of patients if if few treatment options are present. With FDA analyses suggesting that 11 lupus patients needed to be treated on Benylstra to find one the drug helped, and with the drug showing marginal effectiveness overall, but with the last FDA approved drug for lupus dating back 50 years, Benylstra was easily approved. (Benlystra significant helped those few patients which responded.)

Hemispherx's production facility at New Brunswick will be undergoing 'pre-approval inspection' as part of the FDA review process. If the drug is approved or granted accelerated approval status Hemispherx should, hopefully, be able to hit the ground running. Could the drug be widely available to ME/CFS patients sometime next year? Time will tell.

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Comments

I'm not optimistic about Ampligen, but if it is approved by the FDA does it then mean that any physician is able to prescribe it or will this also depend on subsequent approval by someone else? Not sure how it works over the pond.

Of course in the UK we'd need NICE to also agree that it was an effective treatment option for CFS patients I would suspect before any doctor could prescribe it specifically.

Thus far it hasn't to my knowledge attracted comment from the UK authorities. I guess like in many other things we're awaiting the USA to take the plunge.
 
Great news! Very glad to see they are on the ball and there is new data showing such effectiveness! What a godsend it would be if it were approved, both for treatment and for our 'credibility.'

Yes, once FDA approval is granted then any doctor can prescribe it for any condition he sees fit. If it is approved, it will be approved specifically for ME, meaning that it can be advertised and marketed as treating ME (they won't be able to do so for any other condition, though a doctor would be free to prescribe it for another condition). And it will be possible, but not a sure thing i think, that insurance companies will cover it (they will not cover medicines for conditions for which the drug has not been specifically approved).

Firestormm, why are you not optimistic (about the drug itself or just approval?) about ampligen? i will probably start taking it in about a year with Enlander or Cheney once i'm done with gcMAF.

The stars seem to me to be fairly well aligned for approval, but i don't know a lot about ampligen.

Thanks, Cort for the info.
 
Great news! Very glad to see they are on the ball and there is new data showing such effectiveness! What a godsend it would be if it were approved, both for treatment and for our 'credibility.' knock on wood!

Firestormm, Yes, once FDA approval is granted then any doctor can prescribe it for any condition he sees fit. If it is approved, it will be approved specifically for ME, meaning that it can be advertised and marketed as treating ME (they won't be able to do so for any other condition, though a doctor would be free to prescribe it for another condition). And it will be possible, but not a sure thing i think, that insurance companies will cover it (they will not cover medicines for conditions for which the drug has not been specifically approved).

Firestormm, why are you not optimistic (about the drug itself or just approval?) about ampligen? i will probably start taking it in about a year with Enlander or Cheney once i'm done with gcMAF.

The stars seem to me to be fairly well aligned for approval, but i don't know a lot about ampligen.

Thanks, Cort for the info.
 
If it is FDA approved then any doctor in the US, to my knowledge, can prescribe it for ME/CFS. Whether the insurance companies will pay for it is another story. Cost of scale factors should, I would think, push the price down, though, I would think and approval would mean more money for studies which should make it harder for insurance companies not to cover.
 
Thanks for the info Cort.
 
Hemispherix makes Immunovir. It was prescribed to me. It isn't covered by any insurance, you have to order it from Canada, and I didn't notice any benefit after taking it for a long time. I hope Ampligen works better.
 
Great news! Very glad to see they are on the ball and there is new data showing such effectiveness! What a godsend it would be if it were approved, both for treatment and for our 'credibility.' knock on wood!

Firestormm, Yes, once FDA approval is granted then any doctor can prescribe it for any condition he sees fit. If it is approved, it will be approved specifically for ME, meaning that it can be advertised and marketed as treating ME (they won't be able to do so for any other condition, though a doctor would be free to prescribe it for another condition). And it will be possible, but not a sure thing i think, that insurance companies will cover it (they will not cover medicines for conditions for which the drug has not been specifically approved).

Firestormm, why are you not optimistic (about the drug itself or just approval?) about ampligen? i will probably start taking it in about a year with Enlander or Cheney once i'm done with gcMAF.

The stars seem to me to be fairly well aligned for approval, but i don't know a lot about ampligen.

Thanks, Cort for the info.
Morning Justin,

Good to see you posting. Been a long while.

Thanks for that (and from Cort too). I wasn't sure of the process over there.

I think we have had several threads on Ampligen now and I expressed my concerns again I think when this last study was published. Those were based on that study, but I guess I am still feeling that Ampligen like (what I know of) GcMAF was something that searched for something to treat and I'm still not convinced of it's ability to do whatever it now says on the tin in the way that it says it. But if it's approved then we can all give it a whirl I suppose. Whether or not that approval would extend to the UK I really don't know. We'll see I guess.

Apologies for not being as enthused. I hope it lives up to all expectations :)
 
Does anyone know if there are any simple stats to read on the results of any Ampligen studies that I can have a look at without having to read through whole papers?
 
Hemispherix makes Immunovir. It was prescribed to me. It isn't covered by any insurance, you have to order it from Canada, and I didn't notice any benefit after taking it for a long time. I hope Ampligen works better.

Revex makes Immunovir - http://www.rivexpharma.com/products_imunovir.html - Ampligen has undergone quite a bit of study...It's Peterson's best drug at the moment...I would be surprised if its not more effective than Immunovir...
 
Thanks for clearing that up Cort. I didn't think Immunovir was made by Hemispherx .

Ampligen has undergone quite a bit of study...It's Peterson's best drug at the moment...I would be surprised if its not more effective than Immunovir...
But we need to see proper blinded medical trial data so we know what to expect from Ampligen.
Your recent long article said: "Dr. Peterson said that after going off Ampligen about a third of patients actually get better, a third maintain their improvement and a third eventually slide back."
This is good news, but it's not a controlled trial.

And does Peterson use it on all of his patients, or only a subset? (i.e. does it only work for a certain subset of CFS/ME patients?) I thought I read that Peterson only uses Ampligen on a small subset of his patients, but I just had a look for that info and couldn't find it, so maybe I've got that wrong.
 
Ampligen's effects:
-improvement in VO2 max and increased ability to exercise on a treadmill
-ability to improve daily functioning, particularly in the most severely disabled and bedridden patients who do not respond well to symptomatic therapy or cognitive therapy
-reduced usage of medications that treat individual symptoms
-improved cognitive function- in the severely ill, who may have a form of dementia, this includes both improved working memory and normalization of performance IQ
-improved natural killer cell function
-control of herpesvirus reactivation- patients who have consistent positive PCRs for HHV-6 or EBV will have these tests come up negative shortly after starting Ampligen
-complete elimination of the defective 37kd form of Rnase-L and normalization of the overall level of Rnase-L in patients who have abnormal Rnase-L levels. (Rnase-L is part of the interferon pathway, our body's natural antiviral control system)
-some improvement is noticeable after 6 months, but it can take up to 18 months for full benefit
-dosage can be very tricky
-some patients do not respond to Ampligen
-Ampligen can cause flu-like symptoms like chills, headache, muscle pain, and increased fatigue for up to 24 hours after each infusion, but Ampligen appears to be safer than other drugs being considered to treat CFS, such as rituximab and valcyte
 
Thanks for clearing that up Cort. I didn't think Immunovir was made by Hemispherx .

Ampligen has undergone quite a bit of study...It's Peterson's best drug at the moment...I would be surprised if its not more effective than Immunovir...
But we need to see proper blinded medical trial data so we know what to expect from Ampligen.
Your recent long article said: "Dr. Peterson said that after going off Ampligen about a third of patients actually get better, a third maintain their improvement and a third eventually slide back."
This is good news, but it's not a controlled trial.

And does Peterson use it on all of his patients, or only a subset? (i.e. does it only work for a certain subset of CFS/ME patients?) I thought I read that Peterson only uses Ampligen on a small subset of his patients, but I just had a look for that info and couldn't find it, so maybe I've got that wrong.
The original trial was blinded but it used one dose throughout which sounds like it was a mistake. Nevertheless positive findings regarding endurance and functionality were found. I think a study with multiple doses - each tailored to the patient - as Dr. Peterson does - would get much better results. They also need to figure out who really benefits; its going to be a subset - identifying that subset would be very helpful.
 
If it is FDA approved then any doctor in the US, to my knowledge, can prescribe it for ME/CFS. Whether the insurance companies will pay for it is another story. Cost of scale factors should, I would think, push the price down, though, I would think and approval would mean more money for studies which should make it harder for insurance companies not to cover.
I believe any doctor could prescribe it, but I wonder in practice how many would when they don't understand the illness at all. Would they be comfortable prescribling something with potentially serious side-effects for an illness that is basically unbeleiveable? I think that will be an obstacle.........but the fact that it is approved by the FDA would lend a ton of credibility to this illness, and I think over time, physicians will learn to take it seriously. Wouldn't it be fantastic if Hemispherx started running commercials for Ampligen on TV, CFS/ME would become household words just like has happened with Fibromyalgia.

Ampligen is administered by IV twice a week, so I'm not sure how a run-of-the mill Family Doc would administer without an IV suite. Maybe they could prescribe and then patients could go to their local hospital for infusion??

Also, as I understand it, Ampligen currently is made by hand in small batches. Hemispherx was having a significant problem (as of a couple of years ago) converting the process into larger scale production. I don't know that they have solved these issues........let's hope so.

On the insurance front, surprisingly, I know of one big insurance company that was planning on covering Ampligen at the beginning of 2011. I happened to carry that insurance but I stopped infusions right before that time so I don't know for sure that it happened, I still think there were some issues being ironed out. I know that Hemispherx is pushing the insurance industry on it.
 
Ampligen is administered by IV twice a week, so I'm not sure how a run-of-the mill Family Doc would administer without an IV suite. Maybe they could prescribe and then patients could go to their local hospital for infusion??
Thanks. I hadn't remembered/didn't know that. Yes that would be a considerable problem should this drug find it's way across to the UK I should think for General Practitioners anyway.
 
Years ago I tried Vit C infusions and had to pay privately to go to some local place - a nursing home? Can't actually remember - to have it done several days in a row. Just needed a bed to lie down on (because I was bedbound) and that metal stand thing that you see on TV for the drip to hang on, and the bag of drip, and a nurse to stick the needle in. It didn't seem very complicated or to involve any big-deal equipment but maybe I just wasn't noticing. I'd be surprised if this wasn't something that could be done easily in a GP's treatment sideroom but perhaps that's just my ignorance speaking!

And if it was on the NHS (unlike my Vit C) we shouldn't have to pay privately.
 
As far as I know, Immunovir is not manufactured by Hemispherix but by Rivex, a Canadian company that is urelated. They do sound alike though.

My take based on reading and talking to people is that Hemispherix probably works for subset of CFS patients but many will likely need to take it regularly and continuously for a benefit. But what that subset is is not known publicly and is not in the papers; perhaps some docs have a sense of who would benefit. The docs that I know who use it do not suggest it for everyone.

More generally, I have problems with the outcome measures that Hermispherix uses (statistically significant does not equal clinically significant) and safety issues. The company is not as forthcoming with the latter as they could be; there may be more behind the story than what is out in the public eye. Regardless of efficacy, safety does need to be considered.

Overall, I think there will be better drugs than Ampligen, safety and efficacy-wise, in the near future and I would caution people to not push for a "political victory" without regard to safety.
 
As far as I know, Immunovir is not manufactured by Hemispherix but by Rivex, a Canadian company that is urelated. They do sound alike though.

My take based on reading and talking to people is that Hemispherix probably works for subset of CFS patients but many will likely need to take it regularly and continuously for a benefit. But what that subset is is not known publicly and is not in the papers; perhaps some docs have a sense of who would benefit. The docs that I know who use it do not suggest it for everyone.

More generally, I have problems with the outcome measures that Hermispherix uses (statistically significant does not equal clinically significant) and safety issues. The company is not as forthcoming with the latter as they could be; there may be more behind the story than what is out in the public eye. Regardless of efficacy, safety does need to be considered.

Overall, I think there will be better drugs than Ampligen, safety and efficacy-wise, in the near future and I would caution people to not push for a "political victory" without regard to safety.
I hope there are better drugs out in the near future but I would be surprised if there are significant safety issues with Ampligen as Dr. Peterson said in a recent interview with Llewelyn King, I think, it was...that it is safe and effective. He's been giving it to patients for over 10 years...

Some people seem to do well after Ampligen - they stay at their level of wellness - but others relapse. It can take a couple of years for them to relapse or it can happen more quickly...

I see Ampligen as a bridge to something better; once they can start studying it in more detail they'll know better why it works in some patients and not others; that will give researchers a leg up on the next drug...but its got to get in there first....
 
I agree with Cort, it would be really nice if they could figure out who would benefit from the drug. But if it was covered by insurance, a lot more people would be able to try it and find out, in the absence of defining biomarkers.

Infusing Ampligen is no big deal, like Sasha said. You just need an IV pole, a nurse to do the infusion, etc. The most complicated part is that it has to be kept refrigerated until use and taken out an hour or so beforehand so it's not too cold. And a good IV nurse is pretty indispensable, but people can be trained to do that by taking a short course and practicing.

I also agree that Ampligen could be a bridge to something more effective. It works for me, but it takes a long time, and although the side effects aren't serious, they are like worsening CFS in the beginning, which usually wears off eventually. For me it takes about four months. And although I got a lot better last time I was on it and went back to my normal life, I never completely got rid of the post-exertional malaise, although it took a lot more to trigger it. The increase in energy and disappearing of other symptoms, such as pain and brain fog, made it totally worth it.

Does anyone know if, in the case of accelerated approval, the patients would still have to pay for the drug like they do now? Would it be under the auspices of a drug trial such as the current Amp-511? What would be the difference between accelerated approval and the current Amp-511?

Thanks.
 
It's not my area of expertise but my feeling is approval of Ampligen by FDA would not necessarily mean approval for reimbursement by US insurance companies. Private insurance companies are given leeway in whether they want to pay for something on not given their own review of the evidence. Most of the time, it takes more than one successful trial (and those trials often have to be larger, multi-center, etc.) for them to consider a drug effective and worth covering. Government insurance like Medicare are mandated by law if I remember right to cover at a least one drug for every medical condition but even then, they might require more evidence before coverage. But coverage by Medicare does influence private insurance coverage since private companies which have a contract with Medicare are required to provide AT LEAST what Medicare does in their plans (companies can choose to provide more).

I hope it does not happen but one of my concerns if if Ampligen is approved whether the idea would then spread among physicians, government, insurance companies, etc. that all people with CFS should be on Ampligen or have a trial of Ampligen. That would be dangerous for some people with CFS.

RE: Peterson. I don't know Peterson personally nor am I a patient of his and he doesn't talk much publicly so I only see/ hear what most people hear/ see. Outside of studies, many people who have side effects or aren't improving on a drug are lost to clinic follow-up (they stop going to the doc) so if they don't do well, their physicians will not know what happened unless the physician makes an extra effort to reach that person. So I believe what he sees/ hears is by nature limited through no fault of his. Also, drug companies are not bound to report all their information to clinicians. And finally, as I mentioned, I doubt he (or any ME/CFS specialist) recommends Ampligen to every CFS patient.

I also think if Ampligen were the true answer Peterson and some of the other docs that have been in this for a while and are dedicated to caring for patients would be much more outspoken and encouraging about the drug.