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

    loading  Checking for direct PDF access through Ovid

Abstract

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. Muscle Nerve53: 717–725, 2016

Related Topics

    loading  Loading Related Articles