In a phase 2/3 study, nearly half of rimegepant-treated patients demonstrated ≥50% reduction in the number of moderate-to-severe migraine days per month
The oral calcitonin-gene-related peptide (CGRP) inhibitor rimegepant (NURTEC ODT; Biohaven) when administered as preventive treatment for migraine, resulted in a -4.3-day reduction in the mean number of migraine days per month.
Based on these findings, published in The Lancet, rimegepant is the only medication targeting CGRP to show efficacy for both acute and preventive treatment of migraine.
Rimegepant, an orally disintegrating tablet, was approved by the FDA for acute treatment of migraine in February 2020.
The US-based phase 2/3 double-blind, randomized, placebo-controlled trial included 747 adults with at least a 1-year history of migraine. After a 4-week lead in period, participants were randomized to receive either rimegepant 75 mg (n-373) or placebo (n=347) every other day for 12 weeks. The primary efficacy endpoint was change in mean number of migraine days per month from baseline during the last 4 weeks of the 12-week study period.
The change from the observation period in mean number of migraine days per month during weeks 9–12 was −4.3 days (95% CI –4.8 to –3.9) with rimegepant and −3.5 days (–4.0 to –3.0) with placebo (least squares mean difference −0.8 days, 95% CI −1.46 to −0.20; p=0.0099).
Rimegepant was also superior to placebo across several secondary endpoints including a 50% reduction from baseline in mean number of moderate-to-severe migraine days/month among 49% in the treatment group compared to 41% in the placebo group.
"These data demonstrate the continued innovation in migraine treatment aimed at CGRP mechanisms," said study coauthor Peter J. Goadsby, MD, PhD, DSc, Professor of Neurology at King's College, London and the University of California, Los Angeles. "This is the first time an oral CGRP migraine treatment has shown dual efficacy in both the acute and preventive treatment of migraine."
Goadsby called therapies with this type of flexibility "truly disruptive" and noted that they "change the treatment paradigm for people living with migraine."
The most common adverse events (AEs) were nausea (2% in participants receiving rimegepant and 0.5% in those receiving placebo) and hypersensitivity, including dyspnea and rash, occurring in less than 1% of patients treated with rimegepant.