Long-term safety and efficacy of eculizumab in generalized myasthenia gravis

Research output: Contribution to journalArticlepeer-review


  • REGAIN Study Group

Colleges, School and Institutes

External organisations

  • Stanford University
  • Hanamaki General Hospital
  • University of Miami
  • International University of Health and Welfare
  • University of Kansas
  • Autonomous University of Barcelona
  • University of Copenhagen
  • University of Virginia
  • Ohio State University
  • Aarhus University Hospital
  • Fundación Pública Galega de Medicina Xenómica (FPGMX), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Genomics Medicine Group, Hospital Clínico, 15706 Santiago de Compostela, University of Santiago de Compostela, Galicia, Spain.
  • Ghent University
  • University of South Florida Tampa
  • Foundation of the Carlo Besta Neurological Institute
  • Alexion Pharmaceuticals
  • North Carolina State University
  • Yale University


INTRODUCTION: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy.

METHODS: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.

RESULTS: The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001).

DISCUSSION: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.

Bibliographic note

© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.


Original languageEnglish
Pages (from-to)14-24
Number of pages11
JournalMuscle & Nerve
Issue number1
Publication statusPublished - Jul 2019


  • Activities of Daily Living, Adult, Angioedema/chemically induced, Antibodies, Monoclonal, Humanized/therapeutic use, Aspergillosis/epidemiology, Complement Inactivating Agents/therapeutic use, Disease Progression, Female, Heart Diseases/chemically induced, Humans, Injection Site Reaction/epidemiology, Longitudinal Studies, Male, Meningococcal Infections/epidemiology, Meningococcal Vaccines/therapeutic use, Middle Aged, Muscle Strength, Myasthenia Gravis/drug therapy, Quality of Life, Treatment Outcome

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