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FDA declines to approve Hemispherx's Ampligen new drug application

Discussion in 'General ME/CFS News' started by Lynn, Feb 4, 2013.

  1. BEG

    BEG Senior Member

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    Bold is mine. Waverunner, you appear to be in the know about this sort of thing and it bears repeating. Over the last two years, I've watched my husband and a small team bring a patented product to market. For phases I and II, the money came out of their own pockets. The costs, however, for 3 trials in the phase III trial were obscenely exorbitant. Fortunately, a large pharmaceutical company got involved, put up the money, and they are now in trial #3 of Phase III. Without that money from the drug company, obviously this innovation would have been dead in its tracks.

    There seem to be other options for ampligen, so maybe there is hope. It seems that's what sustains us.
  2. Sasha

    Sasha Fine, thank you

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    http://www.fda.gov/Drugs/NewsEvents/ucm337750.htm

    FDA Response Letter Regarding Approval of Ampligen for ME/CFS

    Thank you for sharing your desire for the U.S. Food and Drug Administration (FDA) to approve Ampligen. Please accept this response on behalf of FDA’s leadership, who forwarded your email to the Division of Drug Information for direct reply.

    As evidenced by the hundreds of letters, emails, and testimonies submitted to FDA, Myalgic Encephalomyelitis (ME)/Chronic Fatigue Syndrome (CFS) is a devastating disease with a serious impact on quality of life. We are acutely aware of the seriousness of this disease, that no FDA approved treatments are available, and of the community’s strong desire to see rintatolimod injection (Ampligen) approved.

    For many years FDA has worked with Hemispherx Biopharma, Inc. (Hemispherx) on an approval pathway for Ampligen. Since the time of the original New Drug Application (NDA) submitted by Hemispherx for the use of Ampligen to treat CFS in 2007, FDA’s review division has provided many specific recommendations on how best to address deficiencies in the application. In 2009, the review division asked Hemispherx to conduct at least one additional controlled clinical study showing a convincing effect in the CFS population. No new trials were conducted. Hemispherx conducted additional analyses of their existing data, which FDA agreed to review in a resubmitted NDA.

    A public meeting of FDA’s Arthritis Advisory Committee (AC) on December 20, 2012, was held to provide FDA with independent scientific and clinical expertise regarding the Ampligen NDA. At the meeting, both Hemispherx and FDA reviewers presented assessments and analyses of the NDA data to the experts, including physicians with expertise in CFS, a CFS patient representative, and an industry representative. The majority of AC members were concerned about the lack of consistency within the clinical trial results, as well as the limited size of the database available to evaluate Ampligen. The members shared FDA’s concerns, as well, about how the studies had been conducted, including multiple discrepancies and gaps in the safety data. At the end of a full day of discussion AC members voted 8-5 against the approval of Ampligen for the treatment of patients with CFS because of insufficient safety and efficacy data.

    On Monday, February 4, 2013, Hemispherx announced the receipt of a Complete Response (CR) letter from the FDA for Ampligen. FDA issues a CR letter to convey that our review of an application is complete and we cannot approve the application in its present form. A CR letter describes all of the specific deficiencies that the Agency has identified in an application, allowing the company an opportunity to correct those clearly defined deficiencies in a re-submission. FDA’s decision regarding Ampligen encompassed many factors, including the safety and efficacy data and the advice of the AC. We understand the frustration and pain of ME/CFS patients and their caregivers, and how important it is that we continue to work toward development of treatments.

    We want to emphasize that the CR letter issued for Ampligen is entirely separate and distinct from FDA’s support of drug development pathways for CFS – these initiatives remain unaffected and fully supported. ME/CFS is a serious disease and treatments for it represent an important area of unmet need. We will continue to encourage the pharmaceutical industry to develop new treatments in this area. To assist companies with their development, FDA is sponsoring a workshop in spring 2013 focused specifically on ME/CFS drug development.

    We express our gratitude to you and the ME/CFS community for your unwavering support of the research and care of those with ME/CFS. We join you in this commitment, and we look forward to exploring how best to facilitate and expedite the development of safe and effective drug therapies for the signs and symptoms of this debilitating disease.
    taniaaust1 likes this.
  3. Waverunner

    Waverunner Senior Member

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    I don't know if they have the money or not. The problem is, I'm not even a huge fan of Hemispherex. It's pure hindsight but they should have known, that Ampligen will not get approved, as long as they don't come up with the desired data. Moreover, in my eyes, Ampligen is much too expensive for the fact, that it only helps some people and was first synthesized in the 1970s. Ampligen is 40(!!!) years old and when Hemisphere wanted to conduct large scale studies for AIDS, the FDA declined. The approval of Ampligen is a 30 year old, never ending catastrophe. And when you look at other promising drugs (e.g. Pleconaril), it's the same. If we are extremely lucky, we will have a first broadband antiviral by 2020 but it will probably be more like 2025, so this is the time span, PWCs will continue to suffer.
  4. Waverunner

    Waverunner Senior Member

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    The large pharmaceutical company probably sees potential in your product. I don't know why Ampligen has these problems but I still think, that Phase II is enough. Patients then should decide themselves, if they want to buy a Phase II drug or not but I'm glad that you have this support.
    BEG likes this.
  5. Sasha

    Sasha Fine, thank you

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    Bob just posted on Facebook about this response:

    Here is what being United brings to our illness.
    Thank you All for your Efforts.

    Bob
    BEG and Firestormm like this.
  6. Xandoff

    Xandoff Michael

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    The Beat goes on!
    Sasha likes this.
  7. JohnnyD

    JohnnyD Senior Member

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    And the FDA seems to be aware that they have some deficiencies re: the public hearings held yesterday and today on coming up with a new approval pathway for serious diseases, targeting the most seriously ill.

    I still don't understand why the FDA does not work with Hemispherx through FDASIA. Isn't this situation why FDASIA was created? Patients would get a much needed drug now and the FDA would get a post marketing trial to confirm efficacy. It seemed like an ideal solution. <sigh>
    leela, taniaaust1, Waverunner and 2 others like this.
  8. Firestormm

    Firestormm Senior Member

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    I am of the opine that FDA feel they have worked with Hemispherex but that the data produced for this drug has not been enough. Why try to rush through any further approval when the data won't change? They refused to hold a new clinical trial. They tried rehashing old data. That failed. The ball has ever been in Hemispherex's court. And if they want this drug approved they need once again to take the reins. Streamlining an approval's process in order to expedite a drug from development to market does not mean any old drug. It won't excuse drug manufacturers from demonstrating their product actually works. If any blame should be attached to any of this it is to Hemispherex. Once again patient's hopes are raised and dashed by a commercial enterprise. Where does it leave people now who have seemingly benefitted from Ampligen?
    dannybex and barbc56 like this.
  9. Firestormm

    Firestormm Senior Member

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    This is positive but as I said above it doesn't mean 'any old drug' of course. Every manufacturer will have to demonstrate their product 'does what it says on the tin'. And that includes knowing why their product might be effective given what we do and don't know about the disease. I really cannot stomach any more speculation from promoter's without the data to back up their claims. Bad enough being on this rollercoaster without commercial interests benefiting from my (our) continuing discomfort.
    dannybex likes this.
  10. SOC

    SOC Moderator and Senior Member

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    I think the root of the problem may be that it is simply too expensive for a small pharmaceutical company to get FDA approval of a drug. Whether this is good or bad is a matter of opinion, but the process is set up for the major pharmaceutical companies and what they can do in terms of large-scale studies. Where Hemispherix has done patients a disservice is in thinking that they could push through on a limited budget. Understandably, the Hemispherix management wants to hang onto all the profits it would get selling their product rather than share it with a company like Pfizer whose primary contribution was the money needed to do a proper FDA application. Unfortunately, that may be the only way to get through the process.
    Waverunner and Firestormm like this.
  11. Kati

    Kati Patient in training

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    SOC good point, but I wonder if the company or rights to the drug is sellable at this point? this is a drug that has been on the shelf for 25 years, you are dealing with a populatien of patients that is hard to be defined, no recognized biomarkers, very little research at the NIH and just a handful of experts which are viewed as practicing shoddy medicine by their peers, and difficulty establishing criterias for improvements.

    How attractive is that for a prospective pharma company?
  12. JohnnyD

    JohnnyD Senior Member

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    The company is not for sale. They have 40 million in cash and short term securities, very little debt and a burn rate of about 10 million a year. They have three other major programs besides CFS -- two involving ampligen, with all having the potential to be huge. The Breast Cancer study, which finished last month (we'll see data in a few months), is paid for by the National Cancer Institute. Over the last year and a half they have put 8 million upgrading their FDA approved manufacturing facility to produce Alferon-N (which is FDA approved) and Alferon LDO (a new formualtion that still needs approval) much more efficiently. They are not close to being a take over target.

    You make some good points. It is a very challenging disease - non-homogeneous, no bio marker, a variety of co-morbidities, patients on a variety of drugs, broken immune systems. How do you design a trial? that is why other pharma's big or small have not tried, or have failed. We'll see who shows up this spring for the Stakeholders meetings that is supposed to attract drug makers. HEB has stated in conference calls that they have looked for a partner for CFS - the prospective partners tell them, let's see what the FDA says first, then we'll talk.

    IMO, the FDA has not worked with Hemispherx, HEB applied 4 times for fast track approval, which would have kicked in lots of protocol and endpoint help from the FDA. The FDA turned them down every time based on CFS not being a serious disease. How goes the disease, so goes the company. IMO, the FDA is incompetent, corrupt and complicit. FDASIA was created to address some of these problems at the FDA. Large phase III trials are killing innovation. Average cost to get a new drug to market is 800 million. the FDA has authorized limited use of ampligen for 15 years - limited by a patients willingness to spend the bucks and relocate. Drug access for the privileged or for those willing to go absolutely broke. Shame on them.

    Thanks for the vent.
  13. leela

    leela Slow But Hopeful

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    Yes, BEG, it's a racket. It's designed that way on purpose so only the Big Cats can afford to get drugs on the market.
    BEG likes this.
  14. SOC

    SOC Moderator and Senior Member

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    It depends. Ampligen is not specific to ME/CFS. It's an immune modulator of some kind. It was originally thought to be useful in HIV and cancer. It is/was being investigated in Japan as an adjuvent for vaccines. Hemispherix connected with ME/CFS because they believed they'd have better luck with selling it to the FDA as a drug for a currently unserved illness. Or so I heard.

    The patent (or whatever) might have been marketable decades ago and might still be if it's considered useful for other illnesses. If Hemispherix had been willing to sell out to a big pharmaceutical company decades ago, we might have it available now, even if it was as an off-label immune modulator.

    I doubt anyone wants to buy Hemispherix itself with it's history.
    Firestormm likes this.
  15. Kati

    Kati Patient in training

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    In my opinion, targeted therapies are much more in fashion than general immune modulators. Look at all the biologics (monoclonal antibodies) out there, the existing ones and the ones in clinical trials. This is where science is at with cancer, AIDS, RA, Lupus and the likes.
  16. dannybex

    dannybex Senior Member

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    I agree in general that the FDA is corrupt -- both on the 'Food' and the 'Drug' side. They allow all this processed chemically-laden frankenfood to be manufactured that they KNOW contains many, many synthetic craptastic ingredients in them that have been proven to cause health issues -- and then they approve drugs, many of which, if not the majority of which cause liver problems ("Tell your doctor if you have liver problems". Uh, shouldn't the doctor be telling the patient that?). Even DEATH is an allowed 'side effect'.

    At the same time, Carter and Company are no angels, and you know that, or as a shareholder I hope you would. Hemispherx has a long history of being deceptive and disingenuous, resulting in at least two stockholder class-action lawsuits, not to mention the FDA calling them out on some similar issues during the hearing in December.

    Certainly they must know how much it will cost to do a phase III trial -- as you mentioned they have another drug that has been approved already. That couldn't have cost anywhere near $800 million. If they found the funds to do the trials to get that one approved, I don't see why they can't do the same for Ampligen.

    Thanks for letting me vent. :)
  17. SOC

    SOC Moderator and Senior Member

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    Agreed, which is why I think that while the Ampligen patent might have been salable decades ago, it's probably not today. It might have some use as an adjuvent, but I doubt it's worth the trouble to any pharma company now.
  18. Ember

    Ember Senior Member

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  19. Kati

    Kati Patient in training

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    It is available as a phase 3 clinical trial. Look up Clinicaltrials.gov, search Ampligen.

    Patients need to be screened, not totally sure what the exact criterias are.
  20. JohnnyD

    JohnnyD Senior Member

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    I think deceptive and disingenuous is a bit strong Danny. I think mostly Carter has a big ego - but what M.D. doesn't? but there is no doubt that they would do things differently - plenty of mistakes, plenty of blame to go around for everyone.

    They bought Alferon-N and the manufacturing plant from Interferon Science back around 2003... for a song (28 million if I recall) - so they did not develop alferon. The FDA wants them to do a 600 patient trial. While they could probably pull it off, they then would be so strapped that the company's fate would literally be in the hands of the FDA. They won't let that happen because they have 3 other major programs, all with huge potential, that have well defined endpoints and easy to prove efficacy.

    You're welcome. :)

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