Two investigational medications are safe and effective for the treatment of generalized myasthenia gravis (gMG), new research shows.
The phase 3 studies showed both zilucoplan and rozanolixizumab improve gMG-specific outcomes compared with placebo.
Dr James Howard
In the case of zilucoplan, the drug provides targeted immune suppression and has a very favorable side effect profile, James F. Howard, MD, told Medscape Medical News. Howard is distinguished professor of neuromuscular disease, professor of neurology, medicine and allied health at the University of North Carolina School of Medicine in Chapel Hill and lead investigator for the zilucoplan study.
"We have a new drug that produces an excellent beneficial response in a very rapid fashion that was sustained over the course of the clinical trial."
Results from both studies were presented at the 14th Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders.
Inadequate Response
gMG is a rare autoimmune disease of the nerve muscle junction. About 80%-85% of these patients have acetylcholine receptor autoantibody positive (AChR+) gMG, where autoantibodies activate the complement cascade, leading to formation of membrane attack complex and loss of AChRs, which impairs muscle contraction.
Patients with the disorder experience a variety of symptoms, including drooping eyelids, double visionand difficulty with swallowing, chewing and talking — as well as severe and sometimes life-threatening muscle weakness.
