Posted by Michael Wonder on 07 May 2019
FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome, a rare auto-immune disorder
6 May 2019 - The U.S. FDA today approved Ruzurgi (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome in patients 6 to less than 17 years of age.
This is the first FDA approval of a treatment specifically for paediatric patients with Lambert-Eaton myasthenic syndrome (LEMS). The only other treatment approved for LEMS is only approved for use in adults.
The effectiveness of Ruzurgi for the treatment of LEMS was established by a randomised, double-blind, placebo-controlled withdrawal study of 32 adult patients in which patients were taking Ruzurgi for at least three months prior to entering the study.
