Hemispherx Biopharma, Inc., an advanced specialty pharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of seriously debilitating disorders, has submitted a new drug application to ANMAT (Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica), the agency responsible for the national regulation of drugs, foods and medical technology in Argentina, under the ANMAT’s Orphan Drug regulations.
Ampligen is an experimental drug currently undergoing clinical development for the treatment of Chronic Fatigue Syndrome (CFS) in the United States. Over its developmental history, Ampligen has received designation by the US Food and Drug Administration (FDA) as an Orphan Drug, and has received authorization to distribute the drug under a Treatment IND. Ampligen represents the first drug in the class of large (macromolecular) RNA (nucleic acid) molecules to apply for New Drug Application (NDA) review in the United States. Over 1,000 patients have participated in Ampligen clinical trials representing the administration of more than 90,000 doses.
Jorge Braver, president of GP Pharm SA said, “We are pleased that this Orphan Drug application has been filed with ANMAT and we hope for a speedy approval because of the significant need here in Argentina for a drug to treat chronic fatigue syndrome. Approval of Ampligen as an Orphan Drug in Argentina should make Ampligen more widely available since, upon approval, we believe it will be reimbursed by the Health Services Authority (SSS), the central health authority in Argentina, for many patients who seek a treatment for CFS.” GP Pharm is Hemispherx’s partner in Argentina and responsible for the efforts toward commercialization there of both Ampligen and Alferon N Injection.
Hemispherx had previously provided GP Pharm an option to market Alferon N Injection, its FDA-approved natural interferon, and Ampligen in Argentina and other Latin America countries. Thomas Equels, executive vice chairman of Hemispherx, stated: “This application is the result of Hemispherx’s ongoing business development initiative in Latin America for both Ampligen and Alferon N Injection.”
In March 2012, Hemispherx announced the publication of a peer-reviewed article in PLoS One which provided additional analyses of the results from the AMP-516 phase III Clinical Trial of Ampligen [rintatolimod, Poly (I) • (C12U)]. The report is entitled “A Double-Blind, Placebo-Controlled, Randomized, Clinical Trial of the TLR-3 Agonist Rintatolimod in Severe Cases of Chronic Fatigue Syndrome”. Recently, researchers from the Centers for Disease Control and Prevention (“CDC”) and Harvard School of Public Health published new data showing the profound economic impact of CFS on increasing healthcare costs of $452 million and decreasing CFS patient productivity by $1.2 billion in Georgia, a state with a population of approximately 5.5 million between the ages of 18-59 (Cost Effectiveness and Resource Allocation, 9:1, 2011).
In the study described in the PLoS One publication, analyses of the exercise tolerance data, showed an average improvement of 21 per cent in subjects receiving Ampligen compared to placebo. In addition, the proportions of patients with exercise improvements of at least 25 per cent and 50 per cent were 1.7 and 1.9-fold greater for the Ampligen group versus placebo (p<0.05). A continuous responder analysis, which examined response improvements from 25 per cent to 50 per cent in five per cent increments, showed a greater improvement in exercise tolerance for patients receiving Ampligen versus placebo at every five per cent increment above 25 per cent.
New analyses of data from the AMP-516 Trial also showed that patients on Ampligen reduced their use of concomitant medications compared to patients receiving placebo. In particular, Ampligen patients reduced their use of medications which may prolong the QT interval. Prolongation of the QT interval is a known risk factor for sudden cardiac death and arrhythmias. A greater portion of the placebo patients were found to have a significant prolongation of the QT interval compared to patients who had received Ampligen®, thereby creating a cardiac risk situation in the CFS patients. Cardiac death is one of three major causes of premature death in CFS, which affects predominantly women in their 40s.