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

作者：Oh Shin J^*; Shcherbakova Natalya; Kostera Pruszczyk Anna; Alsharabati Mohammad; Dimachkie Mazen; Munoz Blanco Jose; Brannagan Thomas; Lavrnic Dragana; Shieh Perry B; Vial Christophe; Meisel Andreas; Komoly Samuel; Schoser Benedikt; Sivakumar Kumaraswamy; So Yuen

来源：Muscle & Nerve, 2016, 53(5): 717-725.

DOI：10.1002/mus.25070

• Amifampridine phosphate
• 3,4-diaminopyridine
• 3,4-DAP
• Lambert-Eaton myasthenic syndrome
• LEMS
• Potassium channel blockers
• Small cell lung cancer (SCLC)

ObjectiveWe evaluated the efficacy and safety of amifampridine phosphate (Firdapse((R))) for symptomatic treatment in Lambert-Eaton myasthenic syndrome (LEMS).
MethodsPhase 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.
ResultsThe 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.
ConclusionsThis study provides Class I evidence of efficacy of amifampridine phosphate as a symptomatic treatment for LEMS. Muscle Nerve53: 717-725, 2016

• 出版日期2016-5