Abstract
BACKGROUND: Vestibular migraine (VM) is the second most common cause of episodic vertigo worldwide, yet effective therapeutic options remain limited. This study aimed to assess the clinical effectiveness of rimegepant in patients with VM. METHODS: A total of 33 VM cases were enrolled in this multicenter, prospective self-controlled cohort study, screened from an initial pool of 106 patients. Participants were orally administered rimegepant at 75 mg every other day as a preventive therapy. Mean 4-week days of vestibular symptom or headache, along with Dizziness Handicap Inventory (DHI), Generalized Anxiety Disorder-7 (GAD-7), Patient Health Questionnaire-9 (PHQ-9), Headache Impact Test-6 (HIT-6), Vertigo Symptom Scale–Short Form (VSS-SF), Activities-specific Balance Confidence Scale (ABC) scores, and hearing sensitivities were compared between pre-dosing and postdosing of rimegepant over a 4-week follow-up. RESULTS: At baseline, participants exhibited a high symptom burden, with elevated DHI, VSS-SF, GAD-7, PHQ-9, and HIT-6 scores and low ABC scores. After rimegepant treatment, there were substantial reductions in monthly vestibular symptom days and headache days (differences: vestibular symptoms, -8.48; headache, -5.88). Totally 63.6% and 70.4% of the patients experienced a 50% reduction in vestibular symptom days and headache days, respectively. Notably, these improvements were already apparent at 2 weeks, indicating early onset of efficacy. Additionally, rimegepant treatment led to significant improvements in vestibular function (differences: DHI, -27.33; VSS-SF, -6.21), psychological status (differences: GAD-7, -3.33; PHQ-9, -3.33), and quality of life (differences: HIT-6, -11.27; ABC, 19.98). In the 22 participants who underwent baseline and 4-week hearing assessments, pure tone audiometry (PTA) results also indicated improvements in hearing. CONCLUSIONS: Rimegepant provides relief of vestibular symptoms and improves psychological well-being and hearing in patients with VM, with efficacy evident as early as 2 weeks. TRIAL REGISTRATION: NCT04939922. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s10194-025-02243-5.