Orphan Drug Designation


LTI-01 Receives Orphan Drug Designation from both Food and Drug Administration (FDA) and European Commission (EC)

Lung Therapeutics has received orphan drug designation from both the FDA and the EC for LTI-01 in the treatment of empyema, a condition resulting from complicated pleural effusion where overt pus (infected fluid) is present in the pleural space.

About Orphan Drug Designation

Orphan drug designation is granted by the FDA or EMA to novel drugs or biologics that demonstrate value in the treatment of rare diseases or conditions affecting fewer than 200,000 patients in the U.S. (for FDA) or fewer than 5 in 10,000 patients in the European Union (for EMA). This designation qualifies a drug manufacturer for certain incentives such as tax credits or market exclusivity for qualified clinical testing to address rare diseases and conditions.

United States: The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA's implementing regulations at 21 CFR Part 316, and the number of people affect by the disease or condition for which the drug product is indicated is fewer than 200,000 persons. The designation provides a manufacturer with a seven-year period of U.S. marketing exclusivity.

European Union: Similar regulations exist in the European Union (EU), with Regulation (EC) No 141/2000 addressing 'orphan medicinal products." The EU definition of the orphan condition is somewhat broader than that of the US FDA, stating that the disease conditions must be life-threatening or very serious conditions affecting not more that five in 10,000 persons in the EU. The designation provides a manufacturer with up to a ten-year period of EU marketing exclusivity.

Japan: Japan has also has a special consideration of orphan diseases and drugs. The Japanese definition of orphan status is that the disease must be incurable without an alternative treatment or the expected efficacy/safety of the new drug far exceeds that of alternatives, and the number of affected patients must be less than 50,000 persons (an incidence of about four in 10,000).