The US FDA has recently given priority review and fast track designations to amifampridine for the treatment of pediatric patients suffering from Lambert- Eaton Myasthenic Syndrome (LEMS). This is the first approval specifically for pediatric patients aged 6 to 17 years. The drug was previously approved for treating LEMS in adult patients.
The drug was approved for LEMS, a rare autoimmune disorder which prudently affects the nerves and muscles leaving the muscles debilitated and hindering the daily activities. In LEMS, body’s own immune cells attack the connection between the nerves and muscles thereby taking out the functionality of nerve cells to send signals to muscle cells, leading to depressed tendon reflexes, and autonomic dysfunction. The patients with LEMS tend to develop serum antibodies against presynaptic P/Q-type voltage-gated calcium channels which in turn decreases…
