Long-Term Safety of Rimegepant 75 mg for the Acute Treatment of Migraine (Study 201) (4829)

Objective: Evaluate the long-term safety of rimegepant 75 mg dosed up to once daily for up to 52 weeks. Background: Rimegepant is a small molecule CGRP receptor antagonist with demonstrated efficacy in the acute treatment of migraine. Final results from this long-term safety study have not previously been reported. Design/Methods: This was a multicenter, long-term, open-label safety study (Study 201) of rimegepant in adults with a history of 2–14 monthly migraine attacks of moderate to severe intensity. Subjects used rimegepant 75 mg orally up to once daily PRN to treat attacks of any pain intensity for up to 52 weeks. A subgroup with 4–14 moderate to severe monthly attacks was assigned to rimegepant 75 mg QOD scheduled dosing for 12 weeks supplemented by PRN dosing (QOD+PRN) on nonscheduled dosing days. Results: In total, 1,800 subjects were treated (PRN [n=1514], QOD+PRN [n=286]). Most subjects (89.4%) were female; median age was 43 years and 3.7% were ≥65 years. Overall, 112,014 rimegepant doses were administered. On-treatment adverse events (AEs) were reported in 60.4% of subjects; the most common (≥5%) were upper respiratory tract infection (8.8%), nasopharyngitis (6.8%), and sinusitis (5.1%). Overall, 2.7% of subjects discontinued due to an AE. Serious AEs were reported in 47 (2.6%) subjects; none were considered by the investigator to be related. Overall, 1.0% of subjects experienced ALT or AST levels >3x ULN during treatment; no cases were assessed as probably related to rimegepant by an external panel of liver experts; there were no Hy’s law cases. In the 12-week QOD+PRN cohort (n=286), no rimegepant-treated subjects experienced ALT or AST levels >3x ULN or bilirubin levels >2x ULN during treatment (n=279). Conclusions: This open-label safety study in adults with migraine shows that long-term dosing of rimegepant 75 mg up to once daily is well-tolerated with a favorable safety profile and no signs of hepatotoxicity. Disclosure: Dr. Croop has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biohaven Pharamceuticals, Inc.. Dr. Croop has received compensation for serving on the Board of Directors of Biohaven Pharamceuticals, Inc.. Dr. Croop holds stock and/or stock options in Biohaven Pharamceuticals, Inc. which sponsored research in which Dr. Croop was involved as an investigator. Dr. Croop holds stock and/or stock options in Biohaven Pharamceuticals, Inc.. Dr. Berman has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Amgen and Alder.Dr. Kudrow has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Eli Lilly, Amgen, Novartis, Alder, Biohaven, and Teva. Dr. Kudrow has received research support from Amgen, Lilly, Teva, Alder, Allergan, Biohaven, Axome, and Electrocore.Dr. Mullin has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Amgen, Biohaven, electroCore, Eli Lilly. Dr. Stock has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biohaven Pharamceuticals. Dr. Stock holds stock and/or stock options in Biohaven Pharmaceuticals. Dr. Thiry has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biohaven Pharmaceuticals, Inc.. Dr. Thiry has received compensation for serving on the Board of Directors of Biohaven Pharmaceuticals, Inc.. Dr. Thiry holds stock and/or stock options in Biohaven Pharmaceuticals, Inc. which sponsored research in which Dr. Thiry was involved as an investigator. Dr. Thiry holds stock and/or stock options in Biohaven Pharmaceuticals, Inc.. Dr. Conway has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biohaven. Dr. Conway holds stock and/or stock options in Biohaven. Dr. Jensen has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biohaven Pharmaceuticals. Dr. Jensen holds stock and/or stock options in Biohaven Pharmaceuticals. Dr. Lovegren has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biohaven Pharmaceuticals. Dr. Lovegren holds stock and/or stock options in Biohaven Pharmaceuticals. Dr. Coric has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biohaven Pharmaceuticals, Inc.. Dr. Coric has received compensation for serving on the Board of Directors of Biohaven Pharmaceuticals, Inc.. Dr. Coric holds stock and/or stock options in Biohaven Pharmaceuticals, Inc. which sponsored research in which Dr. Coric was involved as an investigator. Dr. Coric holds stock and/or stock options in Biohaven Pharmaceuticals, Inc.. Dr. Lipton has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Teva Pharmaceuticals. Dr. Lipton has received compensation for serving on the Board of Directors of eNeura and Biohaven. Dr. Lipton holds stock and/or stock options in Biohaven which sponsored research in which Dr. Lipton was involved as an investigator. Dr. Lipton holds stock and/or stock options in Biohaven. Dr. Lipton has received research support from Migraine Research Foundation the National Headache Foundation and Amgen.