Pharmacokinetics and safety of erenumab in pediatric patients with migraine: A phase I, randomized, open-label, multiple-dose study.

Erenumab, a fully human monoclonal antibody targeting the calcitonin gene-related peptide receptor, is efficacious and safe for prevention of attacks of migraine in adults. This phase I, randomized, open-label, multiple-dose study evaluated the safety, tolerability, and pharmacokinetics (PK) of erenumab in children and adolescents with migraine. The initial treatment phase lasted 12 weeks, followed by an optional 40-week extension phase for adolescents. Primary end points were PK of erenumab, incidence of treatment-emergent adverse events (TEAEs), and changes in clinical and laboratory assessments. Participants received erenumab 35 mg (n = 4), 70 mg (n = 17), or 140 mg (n = 32) q4w. The mean age was 14.1 years. Of the 53 participants, 48 (90.6%) completed the initial treatment phase and 36 (67.9%) received erenumab during the extension phase. Mean exposures to erenumab based on the maximum observed concentration and the area under the drug concentration-time curve during the dosing interval increased approximately dose-proportionally. A total of 42 participants (79.2%) reported TEAEs (307.2 per 100 participant-years); and four (7.5%) reported serious TEAEs not considered treatment-related. The most common TEAEs were upper respiratory tract infection, headache, and vomiting. No clinically significant changes were reported in vital signs, electrocardiograms, and laboratory and neurological assessments. Overall, the observed PK profile of erenumab in children and adolescents with migraine is consistent with that in adults when body weight differences are taken into consideration. The safety profile of erenumab in children and adolescents is consistent with that in adults.