Amifampridine phosphate (Firdapse®) is effective and safe in a phase 3 clinical trial in LEMS

Shin J. Oh, Natalya Shcherbakova, Anna Kostera-Pruszczyk, Mohammad Alsharabati, Mazen Dimachkie, Jose Munoz Blanco, Thomas Brannagan, Dragana Lavrnić, Perry B. Shieh, Christophe Vial, Andreas Meisel, S. Komoly, Benedikt Schoser, Kumaraswamy Sivakumar, Yuen So

Research output: Article

26 Citations (Scopus)


Objective: We evaluated the efficacy and safety of amifampridine phosphate (Firdapse®) for symptomatic treatment in Lambert-Eaton myasthenic syndrome (LEMS). Methods: Phase 3, randomized, double-blind, study. Patients were treated initially with amifampridine phosphate for 7-91 days, followed by randomization to continue amifampridine phosphate for 14 days or placebo (7-day taper, 7-day placebo). The primary efficacy endpoints were changes from baseline at day 14 in Quantitative Myasthenia Gravis and Subject Global Impression scores. Results: The coprimary efficacy end points and 1 of the secondary efficacy end points were met, showing a significant benefit of aminfampridine phosphate over placebo at Day 14. All 5 primary, secondary, and tertiary endpoints achieved statistical significance at Day 8. Amifampridine phosphate was well tolerated; the most common adverse events were oral and digital paresthesias, nausea, and headache. Conclusions: This study provides Class I evidence of efficacy of amifampridine phosphate as a symptomatic treatment for LEMS.

Original languageEnglish
JournalMuscle and Nerve
Publication statusAccepted/In press - 2016

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)
  • Physiology

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    Oh, S. J., Shcherbakova, N., Kostera-Pruszczyk, A., Alsharabati, M., Dimachkie, M., Blanco, J. M., Brannagan, T., Lavrnić, D., Shieh, P. B., Vial, C., Meisel, A., Komoly, S., Schoser, B., Sivakumar, K., & So, Y. (Accepted/In press). Amifampridine phosphate (Firdapse®) is effective and safe in a phase 3 clinical trial in LEMS. Muscle and Nerve.