Amifampridine phosphate (Firdapse(®) ) is effective and safe in a phase 3 clinical trial in LEMS. Muscle & nerve Oh, S. J., Shcherbakova, N., Kostera-Pruszczyk, A., Alsharabati, M., Dimachkie, M., Blanco, J. M., Brannagan, T., Lavrnic, D., Shieh, P. B., Vial, C., Meisel, A., Komoly, S., Schoser, B., Sivakumar, K., So, Y. 2016; 53 (5): 717-725


We evaluated the efficacy and safety of amifampridine phosphate (Firdapse(®)) for symptomatic treatment in Lambert-Eaton myasthenic syndrome (LEMS).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.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.This study provides Class I evidence of efficacy of amifampridine phosphate as a symptomatic treatment for LEMS.

View details for DOI 10.1002/mus.25070

View details for PubMedID 26852139