Atogepant Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Atogepant comprises 42 peer-reviewed publications across 15 journals, 14 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Migraine Disorders. Most recent publication: Gepants for headache prevention in children and adolescents: A multicenter chart review study., Headache, 2026.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. Atogepant Is Not Associated With Clinically Meaningful Alanine Aminotransferase Elevations in Healthy Adults.
    Min KC, Kraft WK, Bondiskey P, et al. · Clin Transl Sci · 2021
  2. Safety, tolerability, and efficacy of orally administered atogepant for the prevention of episodic migraine in adults: a double-blind, randomised phase 2b/3 trial.
    Goadsby PJ, Dodick DW, Ailani J, et al. · Lancet Neurol · 2020
  3. Atogepant for the Preventive Treatment of Migraine.
    Ailani J, Lipton RB, Goadsby PJ, et al. · N Engl J Med · 2021
  4. Safety and efficacy of atogepant for the preventive treatment of episodic migraine in adults for whom conventional oral preventive treatments have failed (ELEVATE): a randomised, placebo-controlled, phase 3b trial.
    Tassorelli C, Nagy K, Pozo-Rosich P, et al. · Lancet Neurol · 2024
  5. Gepants for headache prevention in children and adolescents: A multicenter chart review study.
    Shin L, Mentz O, Gelfand AA, et al. · Headache · 2026
  6. Population Pharmacokinetics of Atogepant for the Prevention of Migraine.
    Schlachter L, Stodtmann S, Voelkner A, et al. · Clin Pharmacokinet · 2026
  7. Atogepant for the preventive treatment of chronic migraine (PROGRESS): a randomised, double-blind, placebo-controlled, phase 3 trial.
    Pozo-Rosich P, Ailani J, Ashina M, et al. · Lancet · 2023
  8. Rates of Response to Atogepant for Migraine Prophylaxis Among Adults: A Secondary Analysis of a Randomized Clinical Trial.
    Lipton RB, Pozo-Rosich P, Blumenfeld AM, et al. · JAMA Netw Open · 2022
    PubMed: PMID 35675076 · NCT03777059 · Migraine Disorders

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT02848326Migraine DisordersChange From Baseline in Mean Monthly Migraine Days (Migraine/Probable Migraine Headache Days) Across the 12-Week Treatment Period
Baseline (First 28 Days of Screening/Baseline Period) to Week 12
Atogepant 10 mg QD-4.00 migraine days per month (±0.32 Standard Error)
Atogepant 30 mg BID-4.23 migraine days per month (±0.35 Standard Error)
Atogepant 30 mg QD-3.76 migraine days per month (±0.23 Standard Error)
Atogepant 60 mg BID-4.14 migraine days per month (±0.33 Standard Error)
Atogepant 60 mg QD-3.55 migraine days per month (±0.23 Standard Error)
Placebo-2.85 migraine days per month (±0.23 Standard Error)
NCT03700320Percentage of Participants With at Least 1 Treatment Emergent Adverse Event (TEAE)
From first dose up to the end of study (median treatment of 52 weeks) + 4 weeks follow-up
Atogepant 60 mg67.0 percentage of participants
Oral SOC Migraine Preventive Medication78.6 percentage of participants
NCT03777059Migraine DisordersChange From Baseline in Mean Monthly Migraine Days Across the 12-Week Treatment Period
Baseline (Day -28 to Day -1) to Week 12
Atogepant 10 mg-3.69 migraine days per month (±0.210 Standard Error)
Atogepant 30 mg-3.86 migraine days per month (±0.206 Standard Error)
Atogepant 60 mg-4.20 migraine days per month (±0.206 Standard Error)
Placebo-2.48 migraine days per month (±0.210 Standard Error)
NCT03855137Change From Baseline in Mean Monthly Migraine Days Across 12-Week Treatment Period in mITT Population
Baseline to Week 12
Atogepant 30 mg BID-7.13 migraine days per month
Atogepant 60 mg QD-7.27 migraine days per month
Placebo-4.63 migraine days per month
NCT03855137Change From Baseline in Mean Monthly Migraine Days Across 12-Week Treatment Period in Off-Treatment Hypothetical Estimand Population
Baseline to Week 12
Atogepant 30 mg BID-7.00 migraine days per month
Atogepant 60 mg QD-7.19 migraine days per month
Placebo-4.65 migraine days per month
NCT03939312Migraine DisordersPercentage of Participants With at Least 1 Treatment-Emergent Adverse Event and Treatment-Emergent Serious Adverse Event (TEAEs/TESAEs)
From dose of study drug until 30 days following last dose of study drug (up to approximately Week 44)
Atogepant 60 mg62.5 percentage of participants
Atogepant 60 mg3.4 percentage of participants
NCT04437433
JPN ATO OLE
Percentage of Participants With at Least 1 Treatment-Emergent Adverse Event and Treatment-Emergent Serious Adverse Event (TEAEs/TESAEs)
From first dose of study drug until 4 weeks following the last dose of study drug (up to 56 weeks)
Atogepant 60 mg Chronic Migraine88.4 percentage of participants
Atogepant 60 mg Chronic Migraine4.5 percentage of participants
Atogepant 60 mg Episodic Migraine0.0 percentage of participants
Atogepant 60 mg Episodic Migraine90.3 percentage of participants
NCT04740827
ELEVATE
Change From Baseline in Mean Monthly Migraine Days Across 12-Week Treatment Period in mITT Population
Baseline to Week 12
Atogepant 60 mg-4.29 migraine days per month (±0.397 Standard Error)
Placebo-1.86 migraine days per month (±0.389 Standard Error)
NCT04740827
ELEVATE
Change From Baseline in Mean Monthly Migraine Days Across 12-Week Treatment Period in OTHE Population
Baseline to Week 12
Atogepant 60 mg-4.20 migraine days per month (±0.393 Standard Error)
Placebo-1.85 migraine days per month (±0.388 Standard Error)
NCT04829747Migraine DisordersNumber of Participants With a Change in Columbia-Suicide Severity Rating Scale (C-SSRS)
Baseline of the study 3101-303-002 to 16 weeks
Atogepant0 Participants
NCT04829747Migraine DisordersNumber of Participants With Adverse Events
Baseline of the study 3101-303-002 to 16 weeks
Atogepant1 Participants
NCT04829747Migraine DisordersNumber of Participants With Significant Change in Clinical Laboratory Determinations
Baseline of the study 3101-303-002 to 16 weeks
Atogepant0 Participants
NCT04829747Migraine DisordersNumber of Participants With Significant Change in Electrocardiogram (ECG) Parameters
Baseline of the study 3101-303-002 to 12 weeks
Atogepant0 Participants
NCT04829747Migraine DisordersNumber of Participants With Significant Change in Vital Sign Measurements
Baseline of the study 3101-303-002 to 16 weeks
Atogepant0 Participants

Publications by year

20142026: 42 publications.

2014
2
2017
2
2018
1
2020
2
2021
6
2022
4
2023
3
2024
8
2025
8
2026
6

Publications by indication

Migraine Disorders (25)

  • Safety, tolerability, and efficacy of atogepant added to onabotulinumtoxinA for the preventive treatment of chronic migraine: A phase 3, multicenter, 24-week, open-label study.
    Cephalalgia · 2026 · PMID 41944477 · NCT05216263
  • The chronopharmacology of atogepant in migraine prevention: A real-world evaluation of influence of timing of administration on effectiveness and tolerability.
    Cephalalgia · 2026 · PMID 41878762 · NCT06414044
  • Atogepant for migraine in real-world clinical practice: Insights from a large multicentre study in a treatment-resistant population (GEMA project).
    Cephalalgia · 2026 · PMID 41920093 · NCT06241313
  • Effectiveness and tolerability of atogepant in the prevention of migraine: A real life, prospective, multicentric study (the STAR study).
    Cephalalgia · 2025 · PMID 40267275 · NCT06414044
  • Atogepant for the preventive treatment of episodic migraine in Japanese participants: A phase 2/3, randomized, double-blind, placebo-controlled trial with an active treatment extension (RELEASE).
    Cephalalgia · 2025 · PMID 41002192 · NCT05861427

Publications by journal

Trial-results highlights

In studies evaluating the change from baseline in mean monthly migraine days across a 12-week treatment period, various doses of Atogepant were assessed. In NCT02848326, participants treated with Atogepant 10 mg QD experienced a reduction of -4.00 migraine days per month, Atogepant 30 mg BID showed a reduction of -4.23 migraine days per month, Atogepant 30 mg QD showed -3.76 migraine days per month, Atogepant 60 mg BID showed -4.14 migraine days per month, and Atogepant 60 mg QD showed -3.55 migraine days per month, compared to -2.85 migraine days per month for placebo. Another study, NCT03777059, reported reductions of -3.69 migraine days per month for Atogepant 10 mg, -3.86 migraine days per month for Atogepant 30 mg, and -4.20 migraine days per month for Atogepant 60 mg, versus -2.48 migraine days per month for placebo. In NCT03855137 (mITT population), Atogepant 30 mg BID resulted in a reduction of -7.13 migraine days per month, and Atogepant 60 mg QD resulted in -7.27 migraine days per month, compared to -4.63 migraine days per month for placebo. The off-treatment hypothetical estimand population in NCT03855137 showed reductions of -7.00 migraine days per month for Atogepant 30 mg BID and -7.19 migraine days per month for Atogepant 60 mg QD, versus -4.65 migraine days per month for placebo. Furthermore, in NCT04740827, Atogepant 60 mg led to a reduction of -4.29 migraine days per month, compared to -1.86 migraine days per month for placebo.

Regarding safety, the percentage of participants experiencing at least one treatment-emergent adverse event (TEAE) was reported in several studies. In NCT03700320, 67.0 percentage of participants in the Atogepant 60 mg arm experienced at least one TEAE, while 78.6 percentage of participants on oral SOC migraine preventive medication experienced at least one TEAE. NCT03939312 reported that 62.5 percentage of participants receiving Atogepant 60 mg had at least one TEAE, and 3.4 percentage had at least one treatment-emergent serious adverse event (TESAE). In NCT04437433, for Atogepant 60 mg in chronic migraine, 88.4 percentage of participants experienced at least one TEAE and 4.5 percentage experienced at least one TESAE. For Atogepant 60 mg in episodic migraine in the same study, 90.3 percentage of participants experienced at least one TEAE, and 0.0 percentage experienced at least one TESAE. All values are sourced from primary registry reporting; individual papers should be consulted for clinical decisions.

All Atogepant publications (42)

2026 (6 papers)

  1. Gepants for headache prevention in children and adolescents: A multicenter chart review study.
    Shin L, Mentz O, Gelfand AA, et al. · Headache · 2026 · Derived
  2. Population Pharmacokinetics of Atogepant for the Prevention of Migraine.
    Schlachter L, Stodtmann S, Voelkner A, et al. · Clin Pharmacokinet · 2026 · Derived
  3. Safety, tolerability, and efficacy of atogepant added to onabotulinumtoxinA for the preventive treatment of chronic migraine: A phase 3, multicenter, 24-week, open-label study.
    Rothrock J, Najib U, Ailani J, et al. · Cephalalgia · 2026 · Derived
  4. Atogepant for the Preventive Treatment of Migraine in Japanese Participants: A Phase 3, Open-Label, 52-Week Extension Study.
    Matsumori Y, Nagy K, Takahashi A, et al. · Neurol Ther · 2026 · Derived
  5. The chronopharmacology of atogepant in migraine prevention: A real-world evaluation of influence of timing of administration on effectiveness and tolerability.
    Iannone LF, Sebastianelli G, Santis F, et al. · Cephalalgia · 2026 · Derived
    PubMed: PMID 41878762 · NCT06414044 (STAR) · Migraine Disorders
  6. Atogepant for migraine in real-world clinical practice: Insights from a large multicentre study in a treatment-resistant population (GEMA project).
    Gago-Veiga AB, Lopez-Rodriguez AB, Sanchez Jimenez M, et al. · Cephalalgia · 2026 · Derived

2025 (8 papers)

  1. Effectiveness and tolerability of atogepant in the prevention of migraine: A real life, prospective, multicentric study (the STAR study).
    Vernieri F, Iannone LF, Lo Castro F, et al. · Cephalalgia · 2025 · Derived
    PubMed: PMID 40267275 · NCT06414044 (STAR) · Migraine Disorders
  2. UpToDate®.
    Nurse Pract · 2025 · Derived
  3. Atogepant for the preventive treatment of episodic migraine in Japanese participants: A phase 2/3, randomized, double-blind, placebo-controlled trial with an active treatment extension (RELEASE).
    Matsumori Y, Yamada H, Nagaseki Y, et al. · Cephalalgia · 2025 · Derived
  4. Efficacy of ubrogepant and atogepant in males and females with migraine: A secondary analysis of randomized clinical trials.
    Goadsby PJ, Jürgens TP, Brand-Schieber E, et al. · Cephalalgia · 2025 · Derived
  5. Pharmacokinetics of Atogepant in Healthy Lactating Female Participants: Results from a Phase 1 Lactation Study.
    Boinpally RR, Smith JH, De Abreu Ferreira RL, et al. · Neurol Ther · 2025 · Derived
  6. Milk and plasma pharmacokinetics of single-dose ubrogepant in healthy lactating women.
    Boinpally RR, Smith JH, Cohen E, et al. · Headache · 2025 · Derived
  7. Long-term safety, efficacy and functional outcomes of atogepant for the preventive treatment of migraine.
    Ashina S, Ashina M, Holle-Lee D, et al. · Cephalalgia · 2025 · Derived
  8. Safety and tolerability of ubrogepant for the acute treatment of migraine in participants taking atogepant for the preventive treatment of episodic migraine: Results from the TANDEM study.
    Ailani J, Lipton RB, Blumenfeld AM, et al. · Headache · 2025 · Derived

2024 (8 papers)

  1. Safety and efficacy of atogepant for the preventive treatment of episodic migraine in adults for whom conventional oral preventive treatments have failed (ELEVATE): a randomised, placebo-controlled, phase 3b trial.
    Tassorelli C, Nagy K, Pozo-Rosich P, et al. · Lancet Neurol · 2024 · Derived
  2. Safety and tolerability of atogepant for the preventive treatment of migraine: a post hoc analysis of pooled data from four clinical trials.
    Rizzoli P, Marmura MJ, Robblee J, et al. · J Headache Pain · 2024 · Derived
    PubMed: PMID 38462625 · NCT03939312 · Migraine Disorders
  3. Weight loss with atogepant during the preventive treatment of migraine: A pooled analysis.
    Peterlin BL, Bond DS, Ailani J, et al. · Cephalalgia · 2024 · Derived
  4. Sustained response to atogepant in episodic migraine: post hoc analyses of a 12-week randomized trial and a 52-week long-term safety trial.
    Lipton RB, Nahas SJ, Pozo-Rosich P, et al. · J Headache Pain · 2024 · Derived
    PubMed: PMID 38773375 · NCT03777059 · Migraine Disorders
  5. Early Improvements With Atogepant for the Preventive Treatment of Migraine: Results From 3 Randomized Phase 3 Trials.
    Lipton RB, Gandhi P, Tassorelli C, et al. · Neurology · 2024 · Derived
  6. Effect of preventive treatment with atogepant on quality of life, daily functioning, and headache impact across the spectrum of migraine: Findings from three double-blind, randomized, phase 3 trials.
    Gottschalk C, Gandhi P, Pozo-Rosich P, et al. · Cephalalgia · 2024 · Derived
  7. Efficacy of Atogepant in Chronic Migraine With and Without Acute Medication Overuse in the Randomized, Double-Blind, Phase 3 PROGRESS Trial.
    Goadsby PJ, Friedman DI, Holle-Lee D, et al. · Neurology · 2024 · Derived
  8. Gepants for Acute and Preventive Migraine Treatment: A Narrative Review.
    Rissardo JP, Caprara ALF · Brain Sci · 2024 · Background
    PubMed: PMID 36552072 · NCT06882122 (ATOM) · Migraine Disorders

2023 (3 papers)

  1. Atogepant for the preventive treatment of chronic migraine (PROGRESS): a randomised, double-blind, placebo-controlled, phase 3 trial.
    Pozo-Rosich P, Ailani J, Ashina M, et al. · Lancet · 2023 · Derived
  2. Effect of Atogepant for Preventive Migraine Treatment on Patient-Reported Outcomes in the Randomized, Double-blind, Phase 3 ADVANCE Trial.
    Lipton RB, Pozo-Rosich P, Blumenfeld AM, et al. · Neurology · 2023 · Derived
    PubMed: PMID 36396451 · NCT03777059 · Migraine Disorders
  3. Patient-reported migraine-specific quality of life, activity impairment and headache impact with once-daily atogepant for preventive treatment of migraine in a randomized, 52-week trial.
    Lipton RB, Halker Singh RB, Mechtler L, et al. · Cephalalgia · 2023 · Derived

2022 (4 papers)

  1. Time course of efficacy of atogepant for the preventive treatment of migraine: Results from the randomized, double-blind ADVANCE trial.
    Schwedt TJ, Lipton RB, Ailani J, et al. · Cephalalgia · 2022 · Derived
    PubMed: PMID 34521260 · NCT03777059 · Migraine Disorders
  2. Rates of Response to Atogepant for Migraine Prophylaxis Among Adults: A Secondary Analysis of a Randomized Clinical Trial.
    Lipton RB, Pozo-Rosich P, Blumenfeld AM, et al. · JAMA Netw Open · 2022 · Derived
    PubMed: PMID 35675076 · NCT03777059 · Migraine Disorders
  3. A Single Supratherapeutic Dose of Atogepant Does Not Affect Cardiac Repolarization in Healthy Adults: Results From a Randomized, Single-Dose, Phase 1 Crossover Trial.
    Boinpally R, McNamee B, Yao L, et al. · Clin Pharmacol Drug Dev · 2022 · Derived
  4. The endocannabinoid system and related lipids as potential targets for the treatment of migraine-related pain.
    Greco R, Demartini C, Zanaboni AM, et al. · Headache · 2022 · Background
    PubMed: PMID 35179780 · NCT06882122 (ATOM) · Migraine Disorders

2021 (6 papers)

  1. Atogepant Is Not Associated With Clinically Meaningful Alanine Aminotransferase Elevations in Healthy Adults.
    Min KC, Kraft WK, Bondiskey P, et al. · Clin Transl Sci · 2021 · Trial result
  2. Atogepant for the Preventive Treatment of Migraine.
    Ailani J, Lipton RB, Goadsby PJ, et al. · N Engl J Med · 2021 · Trial result
  3. Peripheral changes of endocannabinoid system components in episodic and chronic migraine patients: A pilot study.
    Greco R, Demartini C, Zanaboni AM, et al. · Cephalalgia · 2021 · Background
    PubMed: PMID 32967434 · NCT06882122 (ATOM) · Migraine Disorders
  4. Spinal nociceptive sensitization and plasma palmitoylethanolamide levels during experimentally induced migraine attacks.
    De Icco R, Greco R, Demartini C, et al. · Pain · 2021 · Background
    PubMed: PMID 33587406 · NCT06882122 (ATOM) · Migraine Disorders
  5. Neurophysiological and biomolecular effects of erenumab in chronic migraine: An open label study.
    De Icco R, Fiamingo G, Greco R, et al. · Cephalalgia · 2021 · Background
    PubMed: PMID 32715736 · NCT06882122 (ATOM) · Migraine Disorders
  6. Barriers to care in episodic and chronic migraine: Results from the Chronic Migraine Epidemiology and Outcomes Study.
    Buse DC, Armand CE, Charleston L, et al. · Headache · 2021 · Background
    PubMed: PMID 33797078 · NCT06882122 (ATOM) · Migraine Disorders

2020 (2 papers)

  1. Safety, tolerability, and efficacy of orally administered atogepant for the prevention of episodic migraine in adults: a double-blind, randomised phase 2b/3 trial.
    Goadsby PJ, Dodick DW, Ailani J, et al. · Lancet Neurol · 2020 · Trial result
  2. Plasma levels of CGRP and expression of specific microRNAs in blood cells of episodic and chronic migraine subjects: towards the identification of a panel of peripheral biomarkers of migraine?
    Greco R, De Icco R, Demartini C, et al. · J Headache Pain · 2020 · Background
    PubMed: PMID 33066724 · NCT06882122 (ATOM) · Migraine Disorders

2018 (1 paper)

  1. Frequency-Dependent Habituation Deficit of the Nociceptive Blink Reflex in Aura With Migraine Headache. Can Migraine Aura Modulate Trigeminal Excitability?
    Perrotta A, Anastasio MG, De Icco R, et al. · Headache · 2018 · Background
    PubMed: PMID 28488755 · NCT06882122 (ATOM) · Migraine Disorders

2017 (2 papers)

  1. Serum MicroRNA Signatures in Migraineurs During Attacks and in Pain-Free Periods.
    Andersen HH, Duroux M, Gazerani P, et al. · Mol Neurobiol · 2017 · Background
    PubMed: PMID 25636687 · NCT06882122 (ATOM) · Migraine Disorders
  2. Visual and auditory cortical evoked potentials in interictal episodic migraine: An audit on 624 patients from three centres. Response to the letter by Omland et al.
    Ambrosini A, Kisialiou A, Schoenen J, et al. · Cephalalgia · 2017 · Background
    PubMed: PMID 27872339 · NCT06882122 (ATOM) · Migraine Disorders

2014 (2 papers)

  1. Altered processing of sensory stimuli in patients with migraine.
    de Tommaso M, Ambrosini A, Brighina F, et al. · Nat Rev Neurol · 2014 · Background
    PubMed: PMID 24535465 · NCT06882122 (ATOM) · Migraine Disorders
  2. Habituation and sensitization in primary headaches.
    Coppola G, Di Lorenzo C, Schoenen J, et al. · J Headache Pain · 2014 · Background
    PubMed: PMID 23899115 · NCT06882122 (ATOM) · Migraine Disorders

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).