(Reuters)—Jacobus Pharmaceutical Co Inc on Monday won U.S. approval for the first drug to treat children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder.
The drug, Ruzurgi (amifampridine), was approved for use in patients ages 6–17, the according to the U.S. Food and Drug Administration (FDA).
Lambert-Eaton myasthenic syndrome (LEMS), which affects about three people per million worldwide, affects the connection between nerves and muscles, disrupting the ability of nerve cells to send signals to muscle cells.
The treatment currently available for LEMS has been approved only for adults.
“We continue to be committed to facilitating the development and approval of treatments for rare diseases, particularly those in children,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, says in a statement. “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.”1
Reference
- U.S. Food and Drug Administration. News release: FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder. 2019 May 6.