Abstract Objective This study aims to evaluate the tolerability and effectiveness of the off‐label use of rimegepant as acute migraine treatment in adolescents. Background Calcitonin gene‐related peptide (CGRP) levels are elevated during migraine attacks. Gepants, oral CGRP receptor antagonists, have demonstrated effectiveness as acute and preventive migraine treatment in adults. They are generally well tolerated with limited side effects. Their use for migraine is approved by the United States Food and Drug Administration (FDA) in adults; however, data on their use in adolescents remain limited. Methods This is a retrospective chart review of pediatric patients <18 years of age who received at least one dose of rimegepant as acute migraine treatment at Dell Children's Medical Center between 2020 and 2023. Effectiveness was evaluated using patient‐reported outcomes during clinic visits. Tolerability was assessed through vital signs, laboratory data, and patient‐reported side effects pre‐ and post‐rimegepant use. Data were analyzed using descriptive and inferential statistics. Results Twelve adolescents who received at least one dose of rimegepant were identified. The mean age at first dose was 15.3 years (SD ± 1.5); nine (75%) were female. Migraine types included episodic migraine without aura ( n = 4, 33%), episodic migraine with aura ( n = 1, 8%), chronic migraine without aura ( n = 4, 33%), and hemiplegic migraine ( n = 3, 25%). The mean number of headache days per month at baseline was 11.5 (SD ± 11.8). Patients tried a mean of 3.9 (SD ± 2.4) acute medications prior to rimegepant use. During follow‐up, 10 of 12 (83%) patients reported either resolution or improvement of their migraine attacks with rimegepant use. There were no statistically significant height, weight, blood pressure, or laboratory changes after rimegepant use, and no patients reported side effects. Conclusion Rimegepant may be a safe and effective acute migraine treatment in adolescents. No adverse events occurred, and the medication appears to be safe and well‐tolerated. These findings support the need for larger prospective and randomized controlled trials to further evaluate the tolerability and effectiveness of gepants in the pediatric population.
Tourlas et al. (2026) studied this question.