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

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

Author information

1
Department of Neurology, University of Alabama at Birmingham, Birmingham, Alabama, USA.
2
Research Center of Neurology, Moscow, Russia.
3
Department of Neurology, Medical University of Warsaw, Poland.
4
University of Kansas Medical Center, Kansas City, Kansas, USA.
5
Gregorio Maranon Hospital, Madrid, Spain.
6
Columbia University Medical Center, New York, New York, USA.
7
Clinical Center of Serbia, Clinic of Neurology, Belgrade, Serbia.
8
Department of Neurology, University of California, Los Angeles, California, USA.
9
Hospital of Lyon, ENMG Service and Neuromuscular Pathology Hospital, Lyon, France.
10
Charite Universitatsmedizin Berlin-NeuroCure Clinical Research Center, Berlin, Germany.
11
University of Pécs, Department of Neurology, Pécs, Hungary.
12
Ludwig-Maximilians-University Munich Friedrich-Baur-Institute, Munich, Germany.
13
Neuromuscular Research Center, Phoenix, Arizona, USA. ksiva@nrcaz.com.
14
Stanford University, Stanford, California, USA.

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.

KEYWORDS:

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

PMID:
26852139
DOI:
10.1002/mus.25070
[Indexed for MEDLINE]

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