Efficacy 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: Newer migraine-specific preventive treatments targeting the calcitonin gene-related peptide (CGRP) pathway, such as fremanezumab, are approved for adults with chronic and episodic migraine (EM). However, data are lacking on the efficacy of CGRP pathway mAbs in children and adolescents. This study (NCT04458857) aimed to evaluate the efficacy, safety, and tolerability of fremanezumab for the preventive treatment of EM in children and adolescents. Here, we present efficacy 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: change in monthly average number of migraine days (MMD) during 12-week double-blind period. Secondary endpoints: change in monthly headache days of at least moderate severity (MHD), proportion of participants achieving ≥50% reduction in MMD, change in days with acute medication use, and changes in PedMIDAS and PedsQL scores at week 12.

Results: Two hundred thirty-four participants (fremanezumab, n = 123; PBO, n = 111) were included in the efficacy analysis. Over 12 weeks, the change from BL in MMD was significantly greater with fremanezumab versus PBO (−2.5 vs. −1.4; p = 0.0210), as was the reduction in MHD (−2.6 vs. −1.5; p = 0.0172), and the proportion of participants achieving ≥50% reduction in MMD (47.2% vs. 27.0%; p = 0.0016). Change in monthly average number of days with acute medication use was −2.1 for fremanezumab and −1.0 for PBO (p = 0.0016). At week 12, the PedMIDAS score was numerically improved for fremanezumab versus PBO (−21.6 vs. −15.3), and the PedsQL score had a similar improvement in both groups (+5.7 vs. +6.2).

Conclusion: In this study, fremanezumab demonstrated significantly superior efficacy over PBO in children and adolescents with EM. These findings add to the limited data on the efficacy of CGRP pathway mAbs in these patients and suggest fremanezumab may provide an effective migraine prevention in this patient population.