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Orphan medicines

A designation given to medicines during development which allows the sponsor to access incentives to encourage the development of medicines for rare diseases. This designation applies specifically to medicines for very rare, life-threatening or debilitating diseases, for which there is no satisfactory treatment available or for which the new medicine would provide a significant benefit.

Read related content in the entries on therapy classification, specifically in the entry on ATMPs and Orphan medicines of the research pathways.

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