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Panel rejects experimental chronic fatigue syndrome drug

A drug for chronic fatigue syndrome that spent decades in clinical development and won fervent patient support has been turned down for approval by a committee of advisers to the US Food and Drug Administration (FDA) who voted 9–4 against it. The drug, named Ampligen (rintatolimod), has not been shown to be effective or safe, the committee determined on 20 December.

Ampligen is a double-stranded RNA molecule (called poly I:poly C) which is thought to stimulate the immune system. The FDA does not have to follow the recommendations of its advisers, but briefing documents released by the agency strongly suggest that it will. The FDA’s list of deficiencies in the drug application submitted by Hemispherx Biopharma is stunning and encompasses nearly every aspect of clinical testing.

“Key deficiencies included inadequate evidence of effectiveness or safety, inadequacy of drug–drug interaction studies, lack of carcinogenicity assessment, lack of anti-drug antibody determination, and inadequate analytical methods and drug product specifications,” the agency wrote in one of several sections listing problems associated with the application.

FDA representatives also expressed concern over inconsistencies in the data and statistical analyses. An agency representative says of the data: “It raises our eyebrows and makes us wonder what else is in the database that we’re not seeing.”

Despite these weighty caveats and the drug’s middling performance in the clinic when averaged across all patients, several people with chronic fatigue syndrome (CFS; also called myalgic encephalomyelitis) testified that Ampligen yielded dramatic improvements in their symptoms. Some moved to Reno, Nevada, to be near the clinical-testing site and to receive infusions of the drug.

Alaine Perry, who served as a patient representative on the advisory committee, acknowledged that the Ampligen data are slim, but noted that CFS symptoms can be so severe that some patients would willingly take on a significant mortality risk for the promise of even minor relief of their symptoms. “A very small improvement in a disease like this is life altering,” she said. There are no other approved treatments specifically for CFS.

Accelerated approvals are sometimes used in cases of serious unmet medical need, and there was speculation ahead of Thursday’s meeting that recent legislation charging the FDA with expanding its use of accelerated approvals may come to bear on Hemispherx’s application. Ultimately, however, the FDA is bound by requirements that drug-makers convincingly demonstrate efficacy before approval, representatives said. In fact, according to the FDA briefing documents, the agency made it clear to Hemispherx in June that its application would be unlikely to win approval without additional clinical trials. That revelation probably came as a surprise to many Hemispherx investors, noted The Street’s Adam Feuerstein.


  1. Report this comment

    Trevor Marshall said:

    It is clear that Patient Reported Outcomes, even in debilitating diseases, no longer hold any significant weight in the FDA approval process. When taken with the agency’s grass-roots rejection of (former) Commissioner Von Eschenbach’s “Science of Safety,” it would seem that FDA is moving away from science, away from exploration, towards some concept of ‘efficacy’ and ‘safety’ which will certainly terminate the delivery of new interventions for Rare and/or Idiopathic diseases.

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    Kelly Latta said:

    An issue behind the “middling” performance across all patients is that ME and CFS are likely multiple discrete entities with different pathophysiology, etiology and thus different treatment requirements. A drug trial specifically for osteoarthritis for example would not include patients with RA or juvenile arthritis all in the same arm. Doing so would most likely dilute the efficacy data.

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    CFS Boston said:

    Why would a government that mindfully belittles and neglects CFS & ME, while broadcasting to the world that we are a bunch of psycho-lazya$ses (who just need therapy and more exercise) want to help treat us?

    I mean, did people really believe that Ampligen’s FDA approval was even a possibility?

    w m

  4. Report this comment

    CFS Boston said:

    Chapter 33 of Hillary Johnson’s: Osler’s Web: Inside the Labyrinth of the Chronic Fatigue Syndrome (CFS) Epidemic is entitled “HIV-NEGATIVE AIDS.”

    Neenyah Ostrom’s book “America’s Biggest Cover-up: 50 More Things…CFS and Its Link To AIDS” cites as # 1 thing: “Some CFS Patients May Be Non-HIV AIDS Cases.”

    NON HIV AIDS cases are cited in medical journals since 1992.

    NON HIV AIDS cases are ICD-coded “Chronic Fatigue Syndrome,” where they are hidden in plain sight.

    Will CFS ever make any progress unless we acknowledge this reality?

    My life w/ NON HIV AIDS (including my recent 5-minute federal testimony):
    w ww.cfsstraighttalk.blogspot.c om

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    Lawrence Winkler said:

    This brief summary seems to strongly indicate that Hemispherx did a poor job of research. It seems just as obvious is investors want to see their money and they could see a profit if the FDA would approve. For investors, it doesn’t matter if the drug works as long as they get their cut.

    And Patient Reported Outcomes should have little importance to final approval. It could be useful if patient reports are used to define a population where the treatment is effective and then prove it with trials. Short of good research, of course, drugs should not be approved.

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