FDA Approval for Ampligen Sought

On October 11, 2007, Hemispherx Biopharma, Inc., filed a new drug application with the Food and Drug Administration seeking to market Ampligen as a treatment for chronic fatigue syndrome (CFS). The company is also moving forward with filings in countries outside the United States.

The Philadelphia biotechnology company has been developing and testing Ampligen for more than 30 years. Before being explored as an experimental therapeutic for CFS, Ampligen was considered a potential treatment for AIDS and cancer. The drug was discovered and developed at Johns Hopkins University and it was later licensed to Hemispherx.

Over its developmental history, Ampligen has received various designations from the FDA, including Orphan Drug Product Designation and Emergency (compassionate) Cost Recovery Sales Authorization (FDA). Four pivotal trials—with data on more than 1,200 subjects evaluated with approximately 90,000 dose administrations—are being reported in the new drug application to the FDA.

Late in 2006 the FDA received Hemispherx's presubmission of preclinical information and assigned an application number. Hemispherx will now move forward promptly with a series of scientific and medical peer-reviewed publications on the audited database contained within the new drug application.

The bioactivity of Ampligen (as noted in a study published in Journal of Immunology on April 30, 2007) may be based on its binding to something called the human toll-like receptor 3 (TLR-3). Toll-like receptors such as TLR-3 serve in the early detection of pathogens and the establishment of early defense mechanisms. As such, they may be critical to the first line of immunological defense against a broad range of pathogens.