Rintatolimod

Rintatolimod
Clinical data
Trade names	Ampligen
Other names	PolyI: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	38640-92-5;
UNII	94325AJ25N;
KEGG	D09661;
NIAID ChemDB	000136;
CompTox Dashboard (EPA)	DTXSID50191952 ;
Chemical and physical data
3D model (JSmol)	Interactive image;
	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 ; Key:KNUXHTWUIVMBBY-JRJYXWDASA-N ;
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Rintatolimod, sold under the tradename Ampligen, is a medication intended for treatment of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). There is some evidence it may improve some ME/CFS symptoms.

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).

Although Ampligen was initially cleared for use in Canada in 1997, and obtained orphan drug status for treatment of ME/CFS in the European Union in 2000, it is approved for use only in Argentina. Its status in Canada, per later information, is as a Special Use Program. 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, but this was rejected in December 2009, because the FDA concluded that the two randomized controlled trials "did not provide credible evidence of efficacy" and "because of clinical, statistical, clinical pharmacology, nonclinical, product quality, and facilities inspection deficiencies." 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. There are two open-label uses in the US, under Dr. Dan Peterson in Nevada and Dr. Charles Lapp in North Carolina.

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.