Navigating the Shifting Landscape of Myasthenia Gravis Treatment: The Impact of Expensive Targeted Therapies

Myasthenia Gravis (MG) is a rare and chronic autoimmune disorder characterized by muscle weakness and fatigue. This condition occurs when the immune system attacks acetylcholine receptors (AChR) in the neuromuscular junctions, disrupting nerve-muscle communication and causing muscle weakness which can impact various parts of the body, including the eyes, face, throat, and limbs. In some cases, antibodies may also attack other proteins such as the muscle-specific kinase (MuSK) or low density lipoprotein receptor-related protein 4 (LRP4), worsening the condition. MG can affect people of all ages and genders, but it is more common in women under 40 years old and men over 60.

Over the years, the approach to treating MG has evolved significantly. Standard treatments traditionally included acetylcholinesterase (AChE) inhibitors, corticosteroids, and other immunosuppressive drugs, which broadly modify the immune system. AChE inhibitors work by blocking the enzyme AChE which breaks down acetylcholine, a neurotransmitter vital for muscle contraction. By inhibiting this enzyme, more acetylcholine remains available at the neuromuscular junction improving muscle strength. It is the most prescribed first line treatment for MG, which according to IQVIA's Forecast Link is projected to have high volume but flat sales during the forecast period. Corticosteroids can help by decreasing the production of antibodies that interfere with the transmission of nerve signals to muscles. Other immunosuppressive drugs with fewer side effects are used when patients do not respond sufficiently to AChE and corticosteroids or are contraindicated to corticosteroids.

The current market leader acts by inhibiting the complement system but is losing its US patent in 2027. It is expected to lose its position to a drug which acts by inhibiting the neonatal Fc receptor (FcRn) and offers better efficacy without any boxed warning. Before 2017, there was a significant unmet need for targeted therapies as only symptomatic treatments were available for patients suffering from MG. The landscape of MG treatment is set to change significantly, with new drugs targeting intricate nuances of the body's immune response.

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