title: Amifampridine phosphate (Firdapse(®)) is effective and safe in a phase 3 clinical trial in LEMS.
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.
authors: Department of Neurology, University of Alabama at Birmingham, Birmingham, Alabama, USA.
authors: Shin J
authors: SJ
authors: Oh
authors: Research Center of Neurology, Moscow, Russia.
authors: Natalya
authors: N
authors: Shcherbakova
authors: Department of Neurology, Medical University of Warsaw, Poland.
authors: Anna
authors: A
authors: Kostera-Pruszczyk
authors: Department of Neurology, University of Alabama at Birmingham, Birmingham, Alabama, USA.
authors: Mohammad
authors: M
authors: Alsharabati
authors: University of Kansas Medical Center, Kansas City, Kansas, USA.
authors: Mazen
authors: M
authors: Dimachkie
authors: Gregorio Maranon Hospital, Madrid, Spain.
authors: Jose Munoz
authors: JM
authors: Blanco
authors: Columbia University Medical Center, New York, New York, USA.
authors: Thomas
authors: T
authors: Brannagan
authors: Clinical Center of Serbia, Clinic of Neurology, Belgrade, Serbia.
authors: Dragana
authors: D
authors: Lavrnić
authors: Department of Neurology, University of California, Los Angeles, California, USA.
authors: Perry B
authors: PB
authors: Shieh
authors: Hospital of Lyon, ENMG Service and Neuromuscular Pathology Hospital, Lyon, France.
authors: Christophe
authors: C
authors: Vial
authors: Charite Universitatsmedizin Berlin-NeuroCure Clinical Research Center, Berlin, Germany.
authors: Andreas
authors: A
authors: Meisel
authors: University of Pécs, Department of Neurology, Pécs, Hungary.
authors: Samuel
authors: S
authors: Komoly
authors: Ludwig-Maximilians-University Munich Friedrich-Baur-Institute, Munich, Germany.
authors: Benedikt
authors: B
authors: Schoser
authors: Neuromuscular Research Center, Phoenix, Arizona, USA. ksiva@nrcaz.com.
authors: Kumaraswamy
authors: K
authors: Sivakumar
authors: Stanford University, Stanford, California, USA.
authors: Yuen
authors: Y
authors: So
mesh_terms: D015761:4-Aminopyridine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000077770:Amifampridine; D015220:Calcium Channels / Q000276:immunology; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D015624:Lambert-Eaton Myasthenic Syndrome / Q000188:drug therapy* / Q000276:immunology; D008297:Male; D008875:Middle Aged; D053580:Muscle Strength; D010710:Phosphates / Q000627:therapeutic use*; D026902:Potassium Channel Blockers / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D015220:Calcium Channels; D010710:Phosphates; D026902:Potassium Channel Blockers; D015761:4-Aminopyridine; D000077770:Amifampridine
keywords: 3,4-DAP; 3,4-diaminopyridine; Amifampridine phosphate; LEMS; Lambert-Eaton myasthenic syndrome; Potassium channel blockers; Small cell lung cancer (SCLC)