Characterization of Safety Signals for Spinal Muscular Atrophy Treatments in Children Aged 0 to 3 Years Potential Safety Patterns for Further Investigation.

BACKGROUND

Spinal muscular atrophy (SMA) has become a manageable chronic condition with the advent of three major disease-modifying therapies. However, real-world safety data in children aged 0-3 years remain limited. This study aimed to characterize safety signals using long-term pharmacovigilance data.

METHODS

Adverse drug event reports related to SMA therapies submitted to the FDA Adverse Event Reporting System were extracted from the first quarter of 2015 to the third quarter of 2025. Disproportionality analyses were conducted to detect signals.

RESULTS

A total of 1,155; 408; and 957 Adverse drug event reports were identified for onasemnogene abeparvovec (OA), risdiplam, and nusinersen as the primary suspected drugs, respectively, with balanced sex distributions across the three therapies. Reports involving OA more frequently included abnormalities in liver function-related parameters. Signals related to respiratory failure, atelectasis, and pneumothorax were identified across all three therapies. Reports involving OA also showed a broader spectrum of renal and urinary preferred terms, including proteinuria, hematuria, and glycosuria.

CONCLUSIONS

Distinct safety signal patterns were identified among the three SMA disease-modifying therapies in children 0-3 years. These findings provide postmarketing insights into their safety profiles. Further prospective studies are warranted to validate these findings.