Inspire Pharmaceuticals' enufosol tetrasodium has been granted orphan drug designation for the treatment of cystic fibrosis (CF) by the European Medicines Agency (EMEA).

"We are pleased to make continued progress in our CF programme and we look forward to using the assistance available through the EMEA's orphan drug programme. We believe that achieving European orphan drug status represents an important development as we move forward in securing a corporate partner to develop and commercialise this product candidate outside of North America," said Christy L. Shaffer, president and CEO of Inspire.

According to the EMEA, orphan medicinal products are for diagnosing, preventing or treating life-threatening or very serious conditions that are rare and affect not more than five in 10,000 persons in the European Union (EU). An orphan drug designation provides ten years of potential market exclusivity if the product candidate is approved for marketing in the EU, regulatory assistance in preparing the marketing application, free protocol assistance to optimise clinical development, reduced regulatory fees associated with applying for marketing approval and direct access to the centralised procedure for marketing authorisation application through the EMEA

In March 2001, the US Food and Drug Administration granted orphan drug status for denufosol for the treatment of CF in the United States.

Cystic fibrosis is a fatal disease involving a genetic mutation that disrupts the cystic fibrosis transmembrane regulator (CFTR) protein. This protein acts as an ion-specific channel that moves salt and water to the surface of the airways.

Denufosol tetrasodium is a selective P2Y2 agonist designed to enhance the lung's innate mucosal hydration and mucociliary clearance mechanisms by activating an alternative ion channel that acts in the same way as the defective ion channel in moving salt and water to the surface of the airways.

Inspire is a biopharmaceutical company that discovers, develops and commercialises prescription pharmaceutical products in disease areas with significant commercial potential and unmet medical needs.