Rethinking myasthenia gravis: Experts debate when to go biologic and why diagnosis still comes too late

At a NeurologyLive Clinical Forum in Philadelphia, neuromuscular specialists led by Chafic Karam, MD, of Penn Medicine tackled the still-unsettled question of how best to treat generalized myasthenia gravis (MG). A recurring theme was how often diagnosis comes dangerously late — many new MG cases at Penn Medicine are first identified in the emergency department, after patients have already progressed to respiratory failure. Young women are particularly vulnerable, as their symptoms are frequently dismissed as generalized fatigue.

The panel reviewed the three FDA-approved FcRn inhibitors (efgartigimod, rozanolixizumab, nipocalimab) and three complement inhibitors (eculizumab, ravulizumab, zilucoplan), discussing their trial data and practical use cases. Experts also flagged limitations of the MG-ADL scale — the standard disability measure — noting it weights ocular symptoms equally to respiratory failure, potentially masking true disease burden.

Key Takeaways:

• Efgartigimod showed a 68% MG-ADL response rate vs. 30% on placebo
• Antibody titers often don't correlate with disease severity — antibody quality may matter more
• Newer biologics are increasingly used as a bridge while slower immunosuppressants take effect
• A pipeline of CAR-T, BTK inhibitors, and B-cell-targeted agents is on the horizon

Why it matters: With more approved therapies than ever, the field is shifting focus from what to treat MG with to when — and experts are increasingly arguing for earlier use of advanced biologics rather than cycling through older immunosuppressants first.