Orphan drugs
Opportunities in the
biotechnology sector
 
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Orphan drugs


  • In order to search for new opportunities in the biotech sector Genetrix aims to reconcile economic interest with social commitment within new market niches.
  • Our group develops clinical research programs for the treatment of rare (orphan) diseases, which affect no more than five in every 10,000 people in European Union (EU).


Orphan drug designation provides the following advantages:

 

  • Market exclusivity for up to ten years in the given indication.

  • Better options for achieving a high price from the Regulatory Authorities.

  • Reduction in fees associated with various aspects of the regulatory process, including the application for marketing approval

  • The possibility to bring forward revenue generation. Traditionally, 40% of orphan drugs have been approved based on only Phase II clinical trial data.

Cellerix, a Genetrix company, has succeeded in the orphan drug designation of Ontaril© (treatment of complex anal fistula) and Cx501 (treatment of epidermolysis bullosa).


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