Hemispherx Biopharma. Inc, an advanced biopharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of seriously debilitating disorders, announced that it has filed a New Drug Application (NDA) with the US Food and Drug Administration (FDA) for its experimental therapeutic Ampligen, also known as Poly I : Poly C12U, to treat Chronic Fatigue Syndrome ("CFS").
The company is also moving forward as planned with filings in countries outside the United States. According to data from the Centres for Disease Control ("CDC"), Chronic Fatigue Syndrome may now affect more than 1 million people in the United States alone, causing an economic impact estimated at more than $9 billion annually. The Centres for Disease Control refers to CFS as a "… disabling disease, as disabling as other severe chronic conditions like multiple sclerosis, COPD [Chronic Obstructive Pulmonary Disease] and rheumatoid arthritis" in their ongoing public awareness campaign launched in 2006. The condition may also be associated with early death due to secondary depression/suicide and cancer.
Over its developmental history, the experimental therapeutic Ampligen has received various designations, including Orphan Drug Product Designation (FDA), Emergency (compassionate) Cost Recovery Sales Authorization (FDA) and "promising" designation by the Agency on Health Research Quality (AHRQ). Four well controlled, or pivotal, trials are now being reported in their entirety within Hemispherx Biopharma's NDA submission, which covers more than 1,200 subjects evaluated with approximately 90,000 dose administrations. The experimental therapeutic was originally discovered and developed at The Johns Hopkins University and thereafter licensed to Hemispherx.
On December 29, 2006, the FDA received the company's pre-submission of pre-clinical information and assigned an NDA number. The company will now move forward promptly with a series of scientific and medical peer-reviewed publications on the audited database contained within the NDA application.
"The comprehensive data package being submitted has been possible because of the guidance, and scientific support, of the FDA, the CDC and CFS Advisory Committees to both the government and the private sector. Our profound gratitude goes out to the teams of independent clinical investigators around the country and especially, to the individuals afflicted with CFS who supplied the courage and will to sustain the research, clinical and regulatory communities in their continuing search for better understanding and management of CFS," said, William A. Carter, M.D., chief executive officer and chairman, Hemispherx.
The bioactivity of the experimental therapeutic, Ampligen, as noted in a peer-reviewed Journal of Immunology study and reported by Hemispherx on April 30, 2007, may be based on its binding to the human toll-like receptor 3 (TLR3) and activating subsequent signalling cascades. Toll-like receptors such as TLR-3 serve as "pattern recognition" receptors in the early detection of pathogens and the establishment of early defense mechanisms (innate immunity). As such, they may be critical to the first line of immunological defense against a broad range of pathogens, such as otherwise lethal viruses and various forms of cancer, the company said.
Chronic Fatigue Syndrome (CFS) has a top priority status at the Centres for Disease Control and Prevention, the governmental agency responsible for disease surveillance, as a serious and debilitating disease in the US. The disorder reflects a major unmet medical need. There are currently no approved therapies for CFS. The Agency for Research and Quality, a health service arm of the US Department of Health and Human Services, reported that Ampligen, an experimental therapeutic, yielded the most promising clinical results to date related to CFS. It should be noted that only federal regulatory agencies can determine whether any experimental therapeutic is "safe and effective" for wider distribution outside of the existing authorized clinical trials.
Hemispherx's flagship products include Alferon N Injection (FDA approved for a category of sexually transmitted diseases) and the experimental therapeutics Ampligen and Oragens. Ampligen and Oragens represent experimental RNA nucleic acids being developed for globally important debilitating diseases and disorders of the immune system. Hemispherx's platform technology includes large and small agent components for potential treatment of various severely debilitating and life threatening diseases. Hemispherx has in excess of 100 patents comprising its core intellectual