Rush of Targeted Myasthenia Gravis Therapies Transforms Treatment Landscape

The pharmaceutical industry is witnessing a surge in the development and approval of targeted therapies for myasthenia gravis (MG), a rare autoimmune disorder affecting neuromuscular junctions. This rapid expansion of treatment options is reshaping the market and offering new hope for patients who have long struggled with limited therapeutic choices.

Market Growth and New Entrants

The global MG market is experiencing double-digit growth, with analysts projecting revenues to exceed $10 billion by 2033. This boom is driven by the introduction of several new MG-specific therapies over the past eight years, starting with AstraZeneca's Soliris in 2017. Since then, the market has seen the approval of five new treatments, with companies like argenx, UCB, Amgen, and Regeneron vying for market share.

Dr. Tom Ragole, a neuromuscular neurologist at the UCHealth Neurosciences Center in Colorado, notes, "Until recently, we hadn't seen a newly approved MG therapy in many decades." The influx of new treatments has been so significant that it has sometimes been challenging to recruit enough patients for clinical trials, as many are already benefiting from newly available options.

Targeted Approaches and Mechanism of Action

The new generation of MG drugs focuses on two key pathways in the disease's progression: the complement system and neonatal Fc receptor (FcRn) signaling. These targeted therapies represent a significant shift from traditional treatments like corticosteroids and immunosuppressants.

Notable approvals include:

• Soliris (AstraZeneca): A C5 complement inhibitor approved in 2017
• Vyvgart (argenx): An FcRn-targeted therapy approved in 2021
• Zilbrysq and Rystiggo (UCB): C5 complement inhibitor and FcRn therapy, respectively, both approved in 2023
• Imaavy (J&J): Approved in early 2025

Recent Clinical Developments

The momentum in MG research continues unabated. In August 2025, argenx reported positive Phase III trial results for Vyvgart in patients lacking the acetylcholine receptor (AChR) antibody, a population that accounts for approximately 20% of gMG patients and often experiences more severe symptoms. This development could potentially expand Vyvgart's reach to an additional 11,000 patients.

Regeneron also made headlines with Phase III results for its RNA-based candidate cemdisiran in AChR-positive gMG patients. The study met its primary and secondary endpoints while only inhibiting the complement system by 74%, suggesting a potential for improved quality of life without completely suppressing complement function.

Dr. Andres Sirulnik, head of Hematology Clinical Development at Regeneron, emphasized the unique features of cemdisiran, including its 12-week dosing schedule, which represents a significant improvement over existing therapies that require monthly or even daily administration.