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

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Citation

Oh SJ, Shcherbakova N, Kostera-Pruszczyk A, Alsharabati M, Dimachkie M, Blanco JM, Brannagan T, Lavrnic D, Shieh PB, Vial C, Meisel A, Komoly S, Schoser B, Sivakumar K, So Y

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

Muscle Nerve. 2016 May;53(5):717-25. doi: 10.1002/mus.25070. Epub 2016 Mar 3.

PubMed ID
26852139 [ View in PubMed
]
Abstract

OBJECTIVE: We evaluated the efficacy and safety of amifampridine phosphate (Firdapse((R))) 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.

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