Thomas K. Equels, Executive Vice Chairman, states, "European Medicines Agency designation of Ampligen(R)/rintatolimod as an orphan drug is an important achievement in our path seeking commercial approval of Ampligen(R) as a prophylactic and early onset therapeutic for Ebola and other lethal viral diseases."
The formal European Medicines Agency (EMA) orphan drug designation will be posted on the Company website www.hemispherx.net and will also be posted on the European Commission website in the near future at ec.europa.eu.
Orphan drug designation promotes the clinical development of drugs that target rare life-threatening conditions and which are expected to provide significant therapeutic advantage over any existing treatments and includes some tropical diseases primarily found in developing nations as is the case for EVD.
There are significant benefits for achieving orphan drug designation, including eligibility for grants from European Union (EU) and Member State programs as well as initiatives supporting research and development encompassing clinical protocol design assistance. Designated orphan medicines are assessed for marketing authorization centrally in the EU with reductions/waivers in the fees and costs of the overall regulatory process.
The designation allows companies to make a single application to the EMA, resulting in a single opinion and a single decision from the European Commission, valid in all EU Member States. Authorized orphan medications, once commercially approved, receive benefits including ten years of complete protection from market competition with similar medicines.
This period of protection from competition is extended by two years for medicines that also have complied with an agreed pediatric investigation plan. Sponsors may also have access via orphan designation to conditional approval, which is also conducted under the centralized procedure.
The EU orphan drug designation is an important preliminary step toward commercial approval, which will require clinical trials before a decision on commercial approval can be obtained from the European Commission. No assurances can be given that the EMA will approve Ampligen(R)/rintatolimod for treatment of EVD.
Ampligen, an experimental therapeutic, is a new class of specifically-configured ribonucleic acid (RNA) compounds targeted as potential treatment of diseases with immunologic defects and/or viral causation. Ebola virus specifically inhibits the dsRNA within cells via a sequestration process.
Such RNA would otherwise cause a robust antiviral response to be mounted: Ampligen may be able to overcome this deficiency in host response. Positive results against Ebola in vitro, and/or in an animal model, have been reported to the Company by the United States Army Medical Research Institute of Infectious Diseases (USAMRIID) and other research/academic institutions in the US and abroad.
However, in vitro and animal testing is no assurance of human safety or efficacy for EVD, positive results from a non-human primate (NHP) study may be required before initiation of human clinical testing of Ampligen in patients with EVD.
Clinical studies would also be necessary to establish human efficacy and safety of Ampligen for either treatment and/or prevention of EVD. Clinical safety and tolerability data obtained for one indication, for example, CFS, may be different for another disorder like EVD. Clinical protocol development is being pursued.
Clinical trial data will be necessary to establish the human safety and efficacy, if any, of Ampligen as a commercially approved drug for Ebola viruses.