Safety Outcomes from a Phase 3, Randomised, Double-Blind, Placebo-Controlled Study of Fremanezumab for the Preventive Treatment of Episodic Migraine in Children and Adolescents

Background/Purpose: There are limited well-tolerated migraine preventives approved for children and adolescents. Monoclonal antibodies (mAbs) targeting the calcitonin gene-related peptide (CGRP) pathway, such as fremanezumab, are approved for preventive migraine treatment in adults. There are currently limited data on the safety of these mAbs in children and adolescents. This study (NCT04458857) aimed to evaluate the efficacy, safety, and tolerability of fremanezumab in children and adolescents with episodic migraine (EM). Here, we present the safety outcomes.

Methods: Participants were 6 to 17 years old and had been diagnosed with EM prior to screening. Randomisation: 1:1 monthly fremanezumab s.c. or matched monthly placebo (PBO). Primary endpoint: the least-squares mean change from baseline in monthly average number of migraine days (MMD) during the 12-week double-blind period. Secondary endpoint: safety and tolerability assessed through adverse event (AE) reporting, clinical laboratory test results, and electrocardiogram findings.

Results: In total, 235 participants were available for analysis (fremanezumab, n = 123; PBO, n = 112). In total, 97% of participants in the fremanezumab and 95% in the PBO group completed the 12-week double blind period. The proportion of participants reporting any AE was generally consistent across groups. Injection site reactions were the most common AEs reported more frequently with fremanezumab versus PBO. The proportion of participants with serious AEs and AEs leading to discontinuation was low, and no deaths were reported. In addition, MMDs were significantly reduced for fremanezumab versus PBO (−2.5 vs. −1.4; p = 0.0210).

Conclusion: The safety and tolerability profile of fremanezumab in children and adolescents with EM was consistent with previous RCTs in adults, with no new emerging safety signals observed. These findings suggest fremanezumab provides an effective and well-tolerated preventive migraine treatment option in this patient population.

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Artikel online veröffentlicht:
26. September 2025