Amifampridine to treat LEMSJuly 5, 2019
The US FDA granted approval for amifampridine (Ruzurgi) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age.
Amifampridine is the first FDA approved treatment for this rare neuromuscular disorder of children, said Jacobus Pharmaceutical Company, Inc. The only other treatment approved for LEMS is only approved for use in adults.
Use of amifampridine in patients 6 to less than 17 years of age is supported by evidence from a randomized, double-blind, placebo-controlled withdrawal study of 32 adult patients in which patients were taking the drug for at least three months prior to entering the study.
The study compared patients continuing on amifampridine to patients switched to placebo. Effectiveness was measured by the degree of change in a test that assessed the time it took the patient to rise from a chair, walk three meters, and return to the chair for three consecutive laps without pause.
The patients that continued on amifampridine experienced less impairment than those on placebo. Effectiveness was also measured with a self-assessment scale for LEMS-related weakness that evaluated the feeling of weakening or strengthening. The scores indicated a greater perceived weakening in the patients switched to placebo.