Atogepant for the Preventive Treatment of Migraine in Japanese Participants: A Phase 3, Open-Label, 52-Week Extension Study.

INTRODUCTION

Atogepant is an oral calcitonin gene-related peptide receptor antagonist approved in the USA, EU, and Japan for the preventive treatment of migraine in adults. We evaluated the long-term safety, efficacy, and functional outcomes of atogepant for the preventive treatment of migraine in Japanese participants.

METHODS

This open-label, 52-week, long-term safety extension study evaluated atogepant 60 mg once daily for the preventive treatment of migraine in Japanese participants. The study enrolled participants with chronic migraine (CM) who completed the phase 3 PROGRESS trial and de novo participants with episodic migraine (EM). The primary objective was the safety and tolerability of atogepant. Efficacy and functional outcomes were exploratory endpoints.

RESULTS

Of 204 Japanese participants screened, 186 were enrolled and included in the safety population (CM, n = 155; EM, n = 31) and 180 in the modified intent-to-treat population (CM, n = 150; EM, n = 30). Treatment-emergent adverse events (TEAEs) occurred in 88.7% of participants. TEAEs occurring ≥ 10% were pyrexia (29.0%), nasopharyngitis (16.1%), and constipation (11.3%). Concurrent COVID-19 vaccination attributed to several TEAEs, including pyrexia. Serious TEAEs occurred in 3.8% of participants and none were considered related to atogepant by the investigator. TEAEs leading to discontinuation occurred in 4.8% of participants. Alanine aminotransferase and/or aspartate aminotransferase ≥ 3 × the upper limit of normal occurred in 4.8% of participants; none met criteria for Hy's law. The least squares mean change from baseline in monthly migraine days was - 6.4 days in PROGRESS CM completers and - 3.4 days in de novo EM participants at weeks 1-4 and was consistent over 52 weeks. Similar improvements were observed for the other efficacy and functional outcomes.

CONCLUSION

The safety profile of atogepant in Japanese participants was consistent with the known safety profile in the global population. No new safety signals were identified. Improvements in efficacy and functional outcomes persisted over 52 weeks.

TRIAL REGISTRATION: NCT04437433; https://clinicaltrials.gov/study/NCT04437433.