News out. 8:30 am ET March 24, 2015 (Globe Newswire)
Hemispherx Biopharma, Inc. (NYSE MKT:HEB) (the "Company" or "Hemispherx") announces that the European subsidiary, "Hemispherx Biopharma Europe N.V./S.A" gained a positive opinion from the COMP (Committee on Medical Products) regarding its Orphan Medicinal Product Application for Ampligen(R), an experimental therapeutic, to treat Ebola Virus Disease (EVD).
Orphan designation by the European Medicines Agency (EMA) is granted to promote 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. Hemispherx submitted in vitro and in vivo data in appropriate preclinical models relevant to the EVD indication to the European Union (EU) Committee for Orphan Medicinal Products. In addition to the efficacy data, clinical safety information was also included in the comprehensive application.
The dossier submitted to the EU contained an inventory of relevant pre-clinical research in EVD therapeutically-oriented models, as well as, clinical safety information derived from non-EVD clinical studies of Ampligen(R). Of potentially high relevance to the favorable COMP opinion were experiments, conducted in Italy, which showed that, in vitro, Ampligen(R) successfully competed with Ebola viral dsRNA in a manner which may result in loss of virulence by EBOV. Basing experimental design on an earlier report (Prins, et al. J Virol 2010;84(6):3004), independent researchers showed that Ampligen(R), an experimental therapeutic, successfully competed with dsRNA for Ebola VP35 binding with a remarkably low Ampligen(R) concentration reflected by an IC50=1.1 µg/ml. The Italian researchers received a grant-in-aid from Hemispherx Biopharma for the conduct of these experiments.
There are significant benefits for achieving Orphan Designation, including eligibility for grants from 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 European Union with reductions/waivers in the fees and costs of the overall regulatory process. The designation allows companies to make a single application to the European Medicines Agency, 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 application process consists of multiple steps and a final decision from the European Commission normally occurs sometime after the summary report of the COMP. No assurances can be given that the final decision will designate Ampligen as an Orphan Medical Product for treatment of EVD.
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