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European Union

Hemispherx Biopharma Receives Orphan Drug Designation for Ampligen(R) to Treat Ebola Virus Disease

Strategic Insights

— 2 min read

Hemispherx Biopharma, Inc. has announced that its European subsidiary, Hemispherx Biopharma Europe N.V./S.A., received formal notification from the European Commission approving its Orphan Medicinal Product Application for Ampligen(R), an experimental therapeutic, to treat Ebola Virus Disease (EVD). This designation is a crucial step toward commercial approval, which will require clinical trials before a decision on commercial approval can be obtained from the European Commission.

Key Takeaways:

  • Hemispherx Biopharma's European subsidiary received formal notification from the European Commission approving its Orphan Medicinal Product Application for Ampligen(R) to treat Ebola Virus Disease (EVD).
  • The Orphan Medicinal Product Application is a crucial step toward commercial approval, which will require clinical trials before a decision on commercial approval can be obtained from the European Commission.
  • The European Medicines Agency (EMA) has designated Ampligen(R)/rintatolimod as an orphan drug, which includes eligibility for grants from European Union (EU) and Member State programs as well as initiatives supporting research and development.
  • 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.
  • Authorized orphan medications, once commercially approved, receive benefits including ten years of complete protection from market competition with similar medicines.
  • Ampligen(R), an experimental RNA nucleic acid, is being developed for globally important debilitating diseases and disorders of the immune system, including Chronic Fatigue Syndrome and Ebola Virus Disease.
  • 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(R) in patients with 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.

Statistics:

  • 10 years of complete protection from market competition with similar medicines for authorized orphan medications
  • 2 years of additional protection for medicines that have complied with an agreed pediatric investigation plan
  • 50% reduction or waiver of fees and costs for the overall regulatory process for designated orphan medicines
  • Estimated number of patients affected by Ebola Virus Disease worldwide: over 28,000

Sources:

  • GlobeNewswire via COMTEX (2015-05-11)
  • European Commission website (ec.europa.eu)
  • Hemispherx Biopharma's website (www.hemispherx.net)
  • United States Army Medical Research Institute of Infectious Diseases (USAMRIID)

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