Sabrina Sacconi, MD, PhD, Head of Neuromuscular Disease at Nice University Hospital, discusses recent guidelines for myasthenia gravis (MG).
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against the acetylcholine receptor in the neuromuscular junction. The exact reason this occurs is not known. Weakness tends to increase during periods of activity and improve after periods of rest. Common symptoms include weakness of the muscles that control:
- Eye and eyelid
- Facial expressions
- Chewing
- Talking
- Swallowing
As Dr. Sacconi notes, the therapeutic landscape for MG is rapidly changing with the advent of new therapies such as anti-FcRn and anti-complement receptors. With guidelines being updated across the world, recommendations now include the use of these novel therapies in addition to traditional first line treatments. (e.g., https://pmc.ncbi.nlm.nih.gov/articles/PMC11266451/ and https://pmc.ncbi.nlm.nih.gov/articles/PMC11236053/) These guidelines hope to produce minimal symptom expression in patients.
Clinical Trial Analysis
Dr. Sacconi also discusses a recent pooled analysis looking at the use of rozanolixizumab, an anti-FcRn receptor that was recently approved for MG. The analysis included data from the phase 3 MycarinG study as well as the MG0004 and MG0007 open-label extensions.
In the initial study, participants were treated with one cycle consisting of six once-weekly infusions of rozanolixizumab. It was observed that this treatment significantly improved MG outcomes versus placebo. Response to treatment was also sustained over the cycle. For patients who didn’t respond in the first cycle, greater than 50% responded in the second cycle and response was sustained.
Patient reported outcome measures were all positive. These measures included MG-Activities of Daily Living (MG-ADL) and Quantitative MG (QMG) response rates. Scores in the MG-ADL were improved by 2 or greater points and the QMG scores were improved by 3 or greater points.
Overall, patients receiving rozanolixizumab demonstrated a high response rate over multiple cycles.
To learn more about MG and other rare musculoskeletal disorders, visit https://checkrare.com/diseases/musculoskeletal-diseases/