People with myasthenia gravis (MG) and the doctors who treat them have many reasons to be optimistic. In June 2023, the U.S. Food and Drug Administration (FDA) approved rozanolixizumab-noli (Rystiggo) and an injectable form of efgartigimod (Vyvgart Hytrulo). Four months later, the FDA approved zilucoplan (Zilbrysq). These new drugs are proving to be more effective with fewer side effects than traditional medications like corticosteroids (prednisone). They are also much more expensive, with some costing more than $500,000 a year. And high prices don't just affect patients—the cumulative cost of newly approved drugs for MG and other rare diseases significantly affects our health care system and our country. Read our Finance story to learn how to pay for these new medications and advocate for fair drug prices.
Costs aside, researchers continue to test other potential treatments, many of which address new ways to reduce symptoms and flare-ups. We examine one such therapy—CAR T-cell—which is being studied in a phase 2b trial.
While we wait to see how these clinical trials pan out, we neurologists continue to focus on improving the lives of our patients. One way we can do that is to advise those considering starting a family on how the disease can affect pregnancy and the care of newborns. In Ask Your Neurologist, we assure readers that patients with MG are just as likely to have healthy babies as those without it. We also offer practical information on how patients can work with their neurologists and obstetricians to manage their disease before, during, and after pregnancy.
Another way we keep patients safe is to alert them to how various drugs prescribed for other reasons can sometimes make MG worse or even trigger the disease in someone undiagnosed. Whenever I see new patients, I review a list of medications they should avoid or use with caution, including certain antibiotics, beta-blockers, and even corticosteroids at times. Our feature story explains how to work with doctors and pharmacists to find alternatives, reduce the risk of MG worsening if exposed to one of these medications, and what to monitor when it's necessary to use a medication that's contraindicated.
Caring for people with MG requires a partnership between neurologists and their care teams, the patients and their families, and their communities. I hope this issue will help every member of that team provide the highest quality care. All of us have a role to play in ensuring every patient has access to new and affordable treatments.
Gordon Smith, MD, FAAN, Issue Editor
