Evaluating the impact of the FDA's orphan drug designation on share prices of biotechnology companies.

• Unveiling the impact of the FDA's orphan drug designation (ODD) on small pharma: a tale of significant short-term gains and complex long-term performance. • This study provides insight in-to how regulatory milestones can affect the financial landscapes of the small pharmaceutical companies. This analysis explores the impact of the FDA's orphan drug designation (ODD) on biotechnology companies' share prices in the short and long term. Our analysis reveals that there is a significant immediate increase in share prices following an ODD announcement, which underscores its potential as a robust indicator of short-term investor returns. However, the long-term findings present a more complex picture, with a sustained impact within the biotech industry but a significant depreciation against the broader market over time. These findings elucidate the nuanced effects of regulatory milestones on the financial performance of pharmaceutical companies and highlight the need for more comprehensive studies to further unravel these complex dynamics. [ABSTRACT FROM AUTHOR]