The US Food and Drug Administration (FDA) has approved Jacobus Pharmaceutical’s Ruzurgi (amifampridine) as a treatment for Lambert-Eaton myasthenic syndrome (LEMS).

Ruzurgi is one of the first LEMS drugs to be approved in the US for children aged between six and 17 years. The drug holds priority review, fast track and orphan drug designations.

LEMS is a rare autoimmune condition that causes the body’s immune system to attack the neuromuscular junction, preventing nerve cells from sending signals to the muscles.

Director of the FDA’s division of neurology products Billy Dunn said: "This approval will provide a much-needed treatment option for paediatric patients with LEMS who have significant weakness and fatigue that can often cause great difficulties with daily activities."

"This approval will provide a much-needed treatment option for paediatric patients with LEMS who have significant weakness and fatigue."

The drug has been investigated in multiple studies involving adult patients. Use in children is supported by data from these studies, as well as pharmacokinetic (PK) data from adults.

PK modelling and simulation data were used to determine the dosing regimen in paediatric patients.

A randomised, double-blind, placebo-controlled withdrawal study assessed Ruzurgi in a total of 32 adult patients. It enrolled subjects that were on Ruzurgi for at least three months before the study.

Less impairment was observed in patients continuing with Ruzurgi compared to those that changed to placebo.

A test to evaluate the effectiveness of the drug examined the time taken by the patient to rise from a chair, walk 3m and return to the chair for three consecutive laps without pause.

Measurements were made using a self-assessment scale for LEMS-related weakness. Scores on this scale are said to suggest a greater perceived weakening in the participants that switched to placebo.

The most common Ruzurgi-related side effects were burning or prickling sensations, abdominal pain, indigestion, dizziness and nausea. The effects were similar in paediatric and adult patients.

The drug also caused seizures in patients that did not have a prior history of seizures.

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