Orphan drug propranolol for infantile hemangioma: ten-year real-world safety data from the FAERS database

Propranolol, a non-selective β-adrenergic receptor blocker, is the first FDA-approved orphan drug for the treatment of infantile hemangioma (IH). While it has demonstrated substantial therapeutic benefits, the safety profile of propranolol in infants remains insufficiently characterized, particularly in real-world settings. A retrospective pharmacovigilance analysis of FAERS reports from Q1 2015 to Q4 2024 was conducted, applying four signal detection algorithms (ROR, PRR, BCPNN, MGPS) to identify disproportional AE signals related to propranolol in IH. A total of 1615 AE reports were included. Signal detection identified events across 20 System Organ Classes (SOCs), with vascular, psychiatric, and metabolism and nutrition disorders being most prominent. At the Preferred Term (PT) level, the most frequently reported AEs were sleep disorder (n = 149, ROR = 32.67), peripheral coldness (n = 87, ROR = 108.73), and hypoglycaemia (n = 62, ROR = 22.95), which were pharmacologically plausible and clinically relevant. This study identified safety signals associated with propranolol in infants with IH, highlighting risks across organ systems. These findings support the need for vigilant monitoring, individualized risk assessment, and refinement of labeling recommendations to optimize safety in this vulnerable population.