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Review Article
- Gepants for the Treatment of Migraine in Children and Adolescents: Acute and Preventive Roles, Current Evidence, and Ongoing Trials
- Ji-Hoon Na
- Received July 20, 2026 Accepted September 12, 2026 Published online October 19, 2026
- DOI: https://doi.org/10.62087/hpr.2026.0034 [Epub ahead of print]
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Abstract
PDF - Migraine affects approximately 10% of children and adolescents and is often disabling, but evidence-based options for acute treatment and prevention remain limited. A high placebo response has also complicated pivotal pediatric trials. Therapies targeting the calcitonin gene-related peptide (CGRP) pathway have reshaped adult migraine care, and the approval of one CGRP monoclonal antibody for pediatric prevention has validated this target in pediatric patients. Gepants, small-molecule oral or intranasal CGRP receptor antagonists, are of particular interest because, unlike injectable antibodies, they offer acute or preventive treatment without vasoconstrictive activity. This review summarizes the pharmacology and adult evidence for the four approved gepants, their emerging pediatric use, and ongoing clinical trials. Depending on the agent, gepants have established efficacy for acute treatment or prevention in adults, with favorable long-term safety and no new hepatic or cardiovascular safety signals. Pediatric evidence is limited to small retrospective studies of off-label use in patients whose migraine is refractory to standard therapy. These reports suggest acceptable tolerability and possible benefit in some patients, but controlled trials are needed to determine efficacy. Phase 3 randomized trials are evaluating ubrogepant for acute treatment and atogepant and rimegepant for the prevention of episodic and chronic migraine in pediatric patients aged 6–17 years for acute treatment and episodic migraine prevention, and 12–17 years for chronic migraine prevention, with estimated completion expected between 2027 and 2032. Until controlled data become available, gepant use should be individualized and restricted to patients who have not responded to standard therapies. Pediatric dosing, long-term safety, and the lower age limit of benefit remain key questions.
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