Improvements in Patient-Reported Outcome Measures Through 64 Weeks' Rimegepant Treatment for Migraine: Analysis of a Randomized Controlled Trial with Open-Label Extension.

Purpose: The current exploratory analysis of a Phase 2/3 randomized controlled trial focusses on patient-reported outcome measures from a 64-week study evaluating rimegepant for the preventive treatment of migraine in the USA. Methods: Participants with migraine were randomly assigned to rimegepant 75 mg or placebo every other day during a 12-week double-blind treatment (DBT) phase. During a 52-week open-label extension (OLE) phase all participants took rimegepant 75 mg every other day and additionally on nonscheduled dosing days if needed. Migraine-Specific Quality of Life Questionnaire (MSQ) v.2.1 (including Role Function Restrictive [RFR] domain) and Migraine Disability Assessment Scale (MIDAS) were assessed throughout. Results: 741 participants (mean [standard deviation] age 41.2 [13.06] years, 82.7% female) were treated with ≥1 dose of study drug in the DBT phase, and 603 participants were treated with rimegepant in the OLE phase. At Week 12 (end of DBT phase), for the rimegepant and placebo groups, respectively, mean (95% CI) improvements from baseline in MSQ RFR score were 18.6 (16.24, 20.94) and 15.0 (12.60, 17.37), and improvements in MIDAS Total score were -13.0 (-16.57, -9.52) and -12.0 (-15.52, -8.54). At Week 64 (end of OLE phase, all participants taking open-label rimegepant), for participants originally assigned DBT rimegepant and DBT placebo, respectively, mean (95% CI) improvements from baseline in MSQ RFR score were 32.6 (29.68, 35.42) and 33.2 (30.03, 36.29), and improvements from baseline in MIDAS Total Score were -25.4 (-30.23, -20.48) and -24.4 (-28.29, -20.59). Conclusion: Notable numerical benefit for rimegepant versus placebo was observed at the end of the DBT phase (Week 12) for MSQ RFR domain score. During open-label treatment with rimegepant, improvements from baseline in MSQ domain scores and MIDAS Total Score were clinically meaningful at Week 64. Participants who received placebo in the DBT phase achieved similar benefit when they moved into the OLE phase as those who took rimegepant from the start of the study. Trial Registration: Clinicaltrials.gov NCT03732638.