amifampridine

Ligand id: 8032

Name: amifampridine

Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 3
Hydrogen bond donors 2
Rotatable bonds 0
Topological polar surface area 64.93
Molecular weight 109.06
XLogP -0.66
No. Lipinski's rules broken 0

Molecular properties generated using the CDK

No information available.
Summary of Clinical Use
Amifampridine is approved in the EU as a treatment for the orphan disease, Lambert-Eaton myasthenic syndrome (LEMS). It is not yet approved in the US, where it is being further evaluated in a Phase III clinical trial. It may be useful for treatment other congenital myasthenic syndromes.
Mechanism Of Action and Pharmacodynamic Effects
Lambert-Eaton myasthenic syndrome (LEMS) arises from the autogenic destruction of pre-synaptic proteins, in particular P/Q‐type voltage-gated calcium channels, at the neuromuscular junction. The damage results in reduced release of acetylcholine into the synaptic cleft, impairing muscle contraction which would be the normal response to activation of post-synpatic acetylcholine receptors. Amifampridine acts to block pre-synaptic potassium channels, thereby extending the duration of the action potential, and increasing acetylcholine release. The subsequent increase in acetylcholine receptor activation improves muscle tone.
LEMS often occurs as a paraneoplastic condition [1] in cancer patients or in patients with other autoimmune diseases [2,4].