Orphan Drug Status Orphan status does not mean that FDA has approved the drug. Orphan status and FDA approval are not the same. The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Sponsors must establish safety and efficacy of a compound to treat a rare disease through adequate and well-controlled studies.
The FDA noted in its press release: "Orphan drug designation provides incentives such as clinical trial tax credits, user fee waiver and eligibility for orphan drug exclusivity to assist and encourage the development of drugs for rare diseases."
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