The FDA has approved to review the medication, Ampligen, as potentially the first prescription drug to treat ME/CFS. The drug is being reviewed in particular to treat patients who have severe ME/CFS. Ampligen has been in development and testing now for 30 years as a treatment for Chronic Fatigue Syndrome.
Ampligen, which is used intravenously, has been approved in Canada and Belgium for ME/CFS treatment since 1996. The drug is meant to be used twice a week for at least a year.
AmpligenR (polyI:polyC12U) – still allowed only in specific clinical trial settings conducted under U.S. governmental authorization – is termed a nucleic acid drug, designed to modulate the body’s immune system. Its mechanism of action in ME/CFS is not entirely clear, but it is thought to act on two enzyme systems so as to help the immune system destroy viral RNA and speed the death of virus-affected cells. In particular it may downregulate an anti-viral pathway which research suggests has become upregulated in certain ME/CFS patients (the 2-5 Synthetase/RNase L anti-viral pathway).
The drug’s maker, Philadelphia-based Hemispherx Biopharma, submitted a New Drug Application to the FDA in 2007, and had been asked to answer a series of questions. The FDA’s acceptance of the drug for safety/efficacy review was based on receipt of the requested data. The maker reportedly suggests it is also researching oral delivery of the drug. A “Who’s-Who” of the world’s leading ME/CFS specialists have participated in AmpligenR trials over the years.