The United States Food and Drug Administration has approved Ruzurgi (amifampridine) in children of age from 6 years to 17 years for the treatment of Lambert-Eaton myasthenic syndrome (LEMS).
LEMS is an autoimmune disorder where the connection between the nerves and muscles is lost leading to weakness. In patients with LEMS, body immune systems attack the neuromuscular junction, leading to disruption in the nerve signals to the muscles.
LEMS is a rare disease with the prevalence in children was not established.
The approval is based on the double-blind, placebo-controlled withdrawal study in 32 adults. After taking Ruzurgi for 3 months, patients switched to placebo. The effectiveness is measured in terms of time the patient took to rise from the chair, walk three meters, get back to the chair for three consecutive times without a break. The scores were less in patients who switched to placebo.
Earlier the product received Priority review and Fast Track designation for the product. The product has also received the Orphan designation.