Children aged 6-17 with the autoimmune disorder Lambert-Eaton myasthenic syndrome (LEMS) have the first treatment for their disease with the Food and Drug Administration-approved Ruzurgi.
LEMS causes a disruption in the connection between the nerves and muscle cells, resulting in muscle weakness and other symptoms. It often occurs in patients with cancers like small cell lung cancer.
“We continue to be committed to facilitating the development and approval of treatments for rare diseases, particularly those in children,” said Billy Dunn, director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research. “This approval will provide a much-needed treatment option for pediatric patients with LEMS who have significant weakness and fatigue that can often cause great difficulties with daily activities.”
A randomized, double-blind, placebo-controlled withdrawal study with adult patients taking Ruzurgi for three months or more before the study. Some of the patients in the study of 32 were switched to placebo once the study started. Patients were asked to do a variety of physical tests to determine the effectiveness of Ruzurgi. The patients on the drug experienced less problems with the tests than those on the placebo.
Side effects in pediatric and adult patients included burning or prickling sensation, abdominal pain, indigestion, dizziness, nausea and possible seizures.
Ruzurgi received Priority Review, Fast Track and Orphan Drug designations.
It is believed that LEMS only occurs in every three per million people worldwide.