An FDA status for drugs targeting rare diseases — comes with development incentives and market exclusivity, but is not itself an approval.For diseases affecting fewer than 200,000 people in the US. Incentives include 50% tax credit on clinical trial costs, waived FDA fees, and 7 years of market exclusivity after approval. Afamelanotide (Scenesse for EPP) and setmelanotide (Imcivree for POMC/PCSK1/LEPR-deficiency obesity) both received orphan drug designation before approval. Designation is granted based on disease prevalence and unmet need — it does not indicate the drug works.