An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan ...
Approval for a Phase 1/2a start indicates FDA is ready to accelerate approval. The standard starting point is Phase 0 or Phase 0/1.
Orphan drug status applies to diseases for which there are fewer than 200k cases per year making them potentially unprofitable and lower priority for drug discovery and development. Orphan drug status carries with it accelerated approval and automatic unmet need designation. In the case of end-of-life cancer cases, often fewer than 200k cases each year, orphan drug becomes a matter of simply focusing trials on those patients and then filing a request for orphan drug status after successful Phase 2a completion.
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