Inspire's cystic fibrosis treatment candidate denufosol tetrasodium gets orphan drug status in Europe

By E. Janene Geiss

Philadelphia, Jan. 10 - Inspire Pharmaceuticals, Inc. announced Tuesday that denufosol tetrasodium has been granted orphan drug designation for the treatment of cystic fibrosis by the European Medicines Agency.

According to the agency, 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.

An orphan drug designation provides 10 years of potential market exclusivity if the product candidate is approved for marketing in the European Union, regulatory assistance in preparing the marketing application, free protocol assistance to optimize clinical development, reduced regulatory fees associated with applying for marketing approval and direct access to the centralized procedure for Marketing Authorization Application through the agency.

"We are pleased to make continued progress in our [cystic fibrosis] program and we look forward to using the assistance available through the EMEA's orphan drug program," said Christy L. Shaffer, president and chief executive officer of Inspire, in the release.

The company said it will move forward in securing a corporate partner to develop and commercialize the drug candidate outside of North America.

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

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 Durham, N.C., biopharmaceutical company dedicated to developing and commercializing prescription pharmaceutical products in disease areas with significant commercial potential and unmet medical needs.

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