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Trade names | Ampligen |
Other names | PolyI:PolyC12U |
Routes of administration | IV |
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Rintatolimod, sold under the tradename Ampligen, is a medication intended for treatment of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS).[1] There is some evidence it may improve some ME/CFS symptoms.[1]
It is an immunomodulatory double-stranded RNA drug similar to the prototypical RNA poly I:C. It was first synthesized in the 1970s and is manufactured by AIM ImmunoTech (formerly known as Hemispherx Biopharma).[2]
Although Ampligen was initially cleared for use in Canada in 1997,[3] and obtained orphan drug status for treatment of ME/CFS in the European Union in 2000, it is approved for use only in Argentina.[4][5] Its status in Canada, per later information, is as a Special Use Program.[5] Rintatolimod has not yet been approved as a legally-prescriptible medication to treat any formally-defined health conditions, diseases, or symptoms in the United States of America; it is still classified by the U.S. Food and Drug Administration (FDA) as an experimental drug.
In 2007, Hemispherx filed a new drug application with the U.S. Food and Drug Administration (FDA) to market and sell rintatolimod for the treatment of CFS,[6] but this was rejected in December 2009, because the FDA concluded that the two randomized controlled trials "did not provide credible evidence of efficacy"[7][8] and "because of clinical, statistical, clinical pharmacology, nonclinical, product quality, and facilities inspection deficiencies."[9] The FDA requested Hemispherx conduct at least one additional controlled trial to demonstrate efficacy in treating ME/CFS. In August 2012, Hemispherx submitted further analyses of the original clinical trial data, but did not submit additional trials for review. Four months later, a committee of the FDA voted 8–5 against approval for rintatolimod, again citing insufficient data.[9] There are two open-label uses in the US, under Dr. Dan Peterson in Nevada and Dr. Charles Lapp in North Carolina.[10]
Ampligen continues to be evaluated, and as of May 2021 is the subject of phase 2 and phase 3 trials to potentially treat myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and several cancers.
Smith2015
was invoked but never defined (see the help page).... special access activities in Canada to include managing the supply of Ampligen® for the treatment of ME/CFS, for which there is currently no approved product in Canada. Ampligen is approved only in Argentina for severe ME/CFS.
BioSpace2020
was invoked but never defined (see the help page).