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FDA approves fremanezumab for the preventive treatment of pediatric episodic migraine

On August 6, 2025, the U.S. Food and Drug Administration (FDA) approved fremanezumab for the preventive treatment of episodic migraine in children and adolescents aged six to 17 years weighing at least 45 kg. It was previously approved in adults for the preventive treatment of both episodic and chronic migraine and marks the first anti-calcitonin gene-related peptide (CGRP) monoclonal antibody approved for use in the pediatric population.¹

Unmet need in pediatric episodic migraine

Migraine is a highly prevalent and disabling neurological condition in children and adolescents, affecting approximately 7.7% of this population. For those living with episodic migraine, defined as fewer than 15 headache days per month, the burden can be substantial, disrupting academic performance and social engagement.1,2

Preventive treatment options for pediatric migraine have historically been limited, with most therapies used off-label and lacking robust pediatric-specific data. Although topiramate is FDA-approved for adolescent migraine, its efficacy has been inconsistent in trials. The approval of fremanezumab introduces a targeted preventive treatment designed around a mechanistic understanding of migraine pathophysiology.1-3

Fremanezumab: mechanism of action

Fremanezumab is a fully humanized monoclonal antibody that targets CGRP, a neuropeptide involved in the transmission of migraine pain and vasodilation during migraine attacks. By binding to CGRP and preventing its interaction with receptors, fremanezumab interrupts a key pathway in migraine development.4 It is administered as a monthly 225 mg subcutaneous injection by a healthcare professional or at home by the patient or caregiver.¹

Pivotal data: the Phase III SPACE trial

The FDA approval was supported by results from the Phase III SPACE trial (NCT04458857), a randomized, double-blind, placebo-controlled study enrolling 237 children and adolescents aged six to 17 years, each weighing at least 45 kg and experiencing episodic migraine.5

Participants were randomized to receive either fremanezumab or placebo over a 12-week treatment period. Among the 234 efficacy-evaluable participants, the key findings included:

  • A statistically significant reduction in monthly migraine days (MMD) from baseline: –2.5 days with fremanezumab versus –1.4 days with placebo (p = 0.0210).5
  • Reduction in monthly headache days (MHD): –2.6 versus –1.5 (p = 0.0172).5
  • A ≥50% responder rate (patients achieving at least 50% reduction in MMD) of 47.2% in the treatment group versus 27.0% with placebo (p = 0.0016).5
  • Consistent efficacy across subgroups by age (6–11 and 12–17 years) and sex.5

Adverse events (AEs) occurred in 55% of patients receiving fremanezumab, compared with 49% in the placebo group. Most were mild to moderate; serious AEs were rare (≤3%), and discontinuation due to AEs occurred in <1% of participants.5 Importantly, the safety profile in this younger cohort was consistent with that observed in adult populations, and no new safety signals emerged.6

Clinical implications

The approval of fremanezumab offers a new, evidence-based preventive option for children and adolescents experiencing episodic migraine, representing a significant step forward in a group historically underserved by approved preventive therapies. Unlike many oral therapies used off-label in pediatric settings, fremanezumab has a well-characterized mechanism, consistent monthly dosing, and a proven safety and efficacy profile from a large, controlled trial in the target population.¹

This development is particularly meaningful given the limited number of FDA-approved preventive treatments for pediatric migraine, which often leads to delays in adequate care and reliance on less targeted interventions. With fremanezumab now available for pediatric use, clinicians may be better equipped to tailor preventive strategies based on a patient’s age, weight, and clinical profile, potentially improving both adherence and long-term outcomes.1-3

Written by Hannah Elkheir

Reviewed by Anya Dragojlovic Kerkache and Henry Shippey

References

  1. Teva Pharmaceuticals. FDA approves expanded indication for AJOVY® (fremanezumab-vfrm), the first anti-CGRP preventive treatment for pediatric episodic migraine. Available here. (Last accessed: 08/07/2025).
  2. Greene K, Irwin SL, Gelfand AA. Pediatric migraine: an update. Neurologic Clinics. 2019 Nov;37(4):815–33.
  3. Kohandel Gargari O, Aghajanian S, Togha M, et al. Preventive medications in pediatric migraine: a network meta-analysis. JAMA Network Open. 2024 Oct;7(10):e2438666.
  4. Urits I, Clark G, An D, et al. An evidence-based review of fremanezumab for the treatment of migraine. Pain Ther. 2020 Mar 28;9(1):195–215.
  5. Hershey AD, Szperka CL, Barbanti P, et al. Efficacy and safety of fremanezumab for the preventive treatment of episodic migraine in children and adolescents: a phase 3, randomized, double-blind, placebo-controlled study (PL5.001). Neurology. 2025 Apr;104(7_Supplement_1).
  6. Teva Pharmaceuticals. Teva Presents Positive Efficacy and Safety Data of AJOVY® (fremanezumab) for the Prevention of Episodic Migraine in Children and Adolescents from Phase 3 SPACE Trial. Available here. (Last accessed: 08/07/2025).