Myasthenia gravis (MG) is an autoimmune and neuromuscular disease characterized by muscle weakness and fatigue. Currently, most of the first-line treatment options are off-label symptomatic therapies, mainly due to their affordability and accessibility, while disease modifying therapies (DMTs) are typically reserved for refractory patients diagnosed with class II-V MG. As a result, major unmet needs plague the classification and treatment of MG, says GlobalData, a leading data and analytics company.

In July 2023, GlobalData surveyed 82 high-prescribing neurologists from the 7MM* to identify current trends in the MG field, including unmet needs. Overall, the neurologists agreed that the most significant unmet needs are for better physician and public awareness of the disease.

Barbora Salcman, Neurology Analyst at GlobalData, comments: “Better awareness of the disease is particularly important with early-stage ocular MG, as it can sometimes manifest itself as non-specific fatigue or can be nearly asymptomatic, leading to an easy misdiagnosis. Furthermore, the currently available diagnostic techniques and tools were highly regarded by the surveyed neurologists; however, they did highlight a major need for prognostic biomarkers to help identify the best treatment strategies for patients diagnosed with different MG types. MG is an extremely heterogenous disease, and it can be challenging to select appropriate treatments for a given patient.”

One of the unmet needs that neurologists and key opinion leaders (KOLs) interviewed by GlobalData agreed on is the necessity for standardized treatment guidelines and more specifically, an update to the current MGFA** guidelines. The biggest criticism among KOLs was the lack of a “class 0” classification for patients, which would account for patients in a remission who are symptom free.

While the opinion of the surveyed neurologists was split regarding access problems with high-cost DMTs, KOLs stressed this was a major issue.

Salcman continues: “For example, in the US, Alexion’s Soliris is priced at nearly $700,000 per year, while Alexion’s Ultomiris and Argenx’s Vyvgart are priced at around $500,000 and $220,000 per year, respectively. Most insurance companies will cover the cost of these DMTs only if the patient has failed to respond to between two and four previous off-label treatments. This requires patients to spend months or years working through ineffective drugs before gaining access to DMTs.”

Although currently available DMTs have almost an immediate effect on treating symptoms and can slow progression, none of them are curative and need to be administered indefinitely unless the patient enters remission. There are seven new pipeline drugs for MG in Phase III clinical trials, but most exhibit similar mechanisms of action to currently available DMTs, and none are likely to be curative.

Salcman concludes: “KOLs do not expect any of the upcoming pipeline agents to have a significant influence on changing the treatment paradigm or improving access to DMTs for non-refractory patients. GlobalData therefore expects most of the unmet needs in MG to not be fully addressed over the next decade.”

*7MM: US, France, Germany, Italy, Spain, UK, and Japan

** Myasthenia Gravis Foundation of America