Efgartigimod alfa is currently in clinical development for the treatment of adults with generalised myasthenia gravis (gMG). gMG is a long-term autoimmune disorder that leads to muscle weakness and tiredness, and which can be seriously debilitating and life-threatening. Currently there are no medicinal products recommended by NICE specifically for the treatment of gMG, and medicines that are used are mostly unlicensed, can take a long time to work and result in side-effects.
Efgartigimod alfa is a modified human antibody (a protein produced by the immune system) fragment that is administered by intravenous (IV) infusion. In patients with myasthenia gravis, the body produces antibodies against the acetylcholine receptors. This medicine works by blocking a protein called FcRn, which attaches to these antibodies and protects them from degradation. Blocking FcRn leads to the degradation of the antibodies that damage the acetylcholine receptors; this is expected to restore the normal contraction of the muscles. If licensed, efgartigimod alfa will offer a treatment option for gMG and reduce the treatment burden for these patients.