Rintatolimod

Rintatolimod
Clinical data
Trade namesAmpligen
Other namesPolyI:PolyC12U
Routes of
administration		IV
ATC code
  • none
Identifiers
IUPAC name
  • 5'-Inosinic acid, homopolymer, complex with 5'-cytidylic acid polymer with 5'-uridylic acid (1:1)
CAS Number
UNII
KEGG
NIAID ChemDB
CompTox Dashboard (EPA)
Chemical and physical data
3D model (JSmol)
SMILES
  • O=P(O)(O)OC[C@H]3O[C@@H](n1c2N\C=N/C(=O)c2nc1)[C@H](O)[C@@H]3O.O=C1/N=C(/N)\C=C/N1[C@@H]2O[C@H](COP(=O)(O)O)[C@@H](O)[C@H]2O.O=C/1NC(=O)N(\C=C\1)[C@@H]2O[C@@H]([C@@H](O)[C@H]2O)COP(=O)(O)O
InChI
  • InChI=1S/C10H13N4O8P.C9H14N3O8P.C9H13N2O9P/c15-6-4(1-21-23(18,19)20)22-10(7(6)16)14-3-13-5-8(14)11-2-12-9(5)17;10-5-1-2-12(9(15)11-5)8-7(14)6(13)4(20-8)3-19-21(16,17)18;12-5-1-2-11(9(15)10-5)8-7(14)6(13)4(20-8)3-19-21(16,17)18/h2-4,6-7,10,15-16H,1H2,(H,11,12,17)(H2,18,19,20);1-2,4,6-8,13-14H,3H2,(H2,10,11,15)(H2,16,17,18);1-2,4,6-8,13-14H,3H2,(H,10,12,15)(H2,16,17,18)/t4-,6-,7-,10-;2*4-,6-,7-,8-/m111/s1 Y
  • Key:KNUXHTWUIVMBBY-JRJYXWDASA-N Y
  (verify)

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.

  1. ^ a b Cite error: The named reference Smith2015 was invoked but never defined (see the help page).
  2. ^ "Official website of AIM ImmunoTech". Retrieved May 17, 2021.
  3. ^ Ostrom N (1997). "Ampligen Finally Breaks Free of Red Tape – in Canada". Stone-Cold Decade. Retrieved May 17, 2021.
  4. ^ "12 Facts about Ampligen research in CFS". Archived from the original on 2016-10-09. Retrieved 2015-02-08.
  5. ^ a b "Hemispherx Expands Ampligen Early Access Programme to Canada to Treat ME/CFS Patients". Globe News Wire. April 4, 2018. Retrieved May 17, 2021. ... 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.
  6. ^ "Hemispherx Biopharma Files New Drug Application for Ampligen as Treatment of Chronic Fatigue Syndrome NDA of investigational drug includes four well-controlled trials, more than 1,200 trial subjects and 90,000 doses". Archived from the original on 2015-09-24. Retrieved 2007-11-07.
  7. ^ George J (December 3, 2009). "FDA rejects Hemispherx's chronic fatigue drug Ampligen". Philadelphia Business Journal. Retrieved February 12, 2010.
  8. ^ Feuerstein A (December 2009). "Hemispherx's Ampligen Dealt FDA Blow". The Street. Retrieved May 17, 2021.
  9. ^ a b "Drug Development for Myalgic Encephalomyelitis and Chronic Fatigue Syndrome (ME and CFS): Questions and Answers". Food and Drug Administration. February 26, 2016. Archived from the original on January 13, 2017. Retrieved May 17, 2021.
  10. ^ Cite error: The named reference BioSpace2020 was invoked but never defined (see the help page).
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