Currently, Firdapse (amifampridine phosphate or 3,4-DAP phosphate) is in Phase III development and it will be provided at no cost until sometime following approval.
Patient enrollment in the Phase III development is expected to start in the next few months, pending FDA review and approvals.
Catalyst Pharmaceutical Partners CEO Patrick McEnany said, "As part of our ongoing commitment to the LEMS community, we are making Firdapse available to those who would like access to this investigational drug, but are unable to participate in clinical studies or travel to sites where it is available."
University of Alabama at Birmingham professor Emeritus of Neurology Shin Oh said, "In previously published clinical trials, amifampridine has been shown to reduce neuromuscular weakness and symptoms of autonomic dysfunction in LEMS patients while being well tolerated."
In patients with LEMS, the release of acetylcholine is decreased at the neuromuscular junction, resulting in muscle weakness.