Orphan Designation

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CHORD Therapeutics

Drug development for rare neurological diseases


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ORPHAN DESIGNATION IN DRUG DEVELOPMENT



Orphan drug (US) and orphan medicinal product (EU) designations are part of regulatory frameworks designed to provide incentives for the development of drugs to meet important unmet needs in rare indications.  Some of the advantages of orphan designation are:


  • Market exclusivity for a specific drug in a specific indication, where patent protection may not be possible
  • Improved access to regulatory guidance from health authorities, to maximize impact and efficiency of investment in drug development
  • Grants for research and development
  • Accelerated reviews
  • Reduced or waived fees


There is today a trend towards investment in orphan development programs in both small and large companies – the search for “niche busters” as an alternative to “block busters”.


Diseases with fewer than 200,000 patients in the US, or with a prevalence of fewer than 50 per 100,000 in the EU, are considered rare by the corresponding health authorities.  Japan, Australia and several other countries today have similar regulatory frameworks, supporting broad application of an orphan strategy.