Unmet Needs in Myasthenia Gravis: What KOLs Say Must Change | REACH MarketVue®

Neurologists surveyed as part of the MarketVue® primary market research effort described a treatment landscape that has improved substantially - but remains incomplete. Even with the FDA approval of multiple FcRn-targeting and complement-inhibiting biologics in the last five years, the fundamental clinical challenges that define gMG have not been fully addressed. The FDA-approved biologic menu for gMG now spans three mechanisms - FcRn inhibitors efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and nipocalimab (Imaavy); complement inhibitors eculizumab (Soliris), ravulizumab (Ultomiris), and zilucoplan (Zilbrysq); and CD19-directed B-cell depleter inebilizumab (Uplizna) - yet incomplete and variable responses, the absence of pharmacological remission, limited options for heterogeneous patient subtypes, and persistent access barriers remain the defining unmet needs of this rare autoimmune disease.

Five Unmet Needs Shaping the gMG Market

1. Longer Remission Times and Sustained Response

Durable, sustained response was the priority neurologists raised most consistently. Today’s FcRn inhibitors and complement agents reduce symptoms, but the benefit is not sustained in a meaningful subset of patients, and true pharmacological remission - defined as being fully asymptomatic and off all medication for at least one year - remains aspirational. The only near-example is rituximab in MuSK-antibody-positive patients, where durable responses have been observed, particularly when treatment begins early. For the larger AChR-positive population, no comparable benchmark has been met.

Incomplete responders continue to cycle through treatment adjustments, and even patients who score well on validated scales like the MG-ADL report persistent fatigue and functional limitations the instruments miss. The mismatch between clinical measurement and lived patient experience points to a gap no currently approved therapy has closed.

2. Prevention of Exacerbations and Myasthenic Crisis

Beyond durability, neurologists stressed the ongoing risk of exacerbations and the acute danger of myasthenic crisis - respiratory failure requiring mechanical ventilation - which current biologics are not designed or approved to manage acutely. Patients with partial or worsening responses remain vulnerable to decompensation triggered by infection, medication changes, or physiological stress, and the standard rescue strategies (plasma exchange and IVIg) work acutely but are not durable solutions. The recurring cycle of crisis, rescue, and attempted stabilization is itself an unmet need, especially for patients without access to specialist neuromuscular centers.

3. Improvement of Symptoms Across a Heterogeneous Patient Population

gMG is not one disease. Neurologists describe it as heterogeneous across antibody subtype, severity, age of onset, comorbidity burden, and the muscle groups involved - and the approved biologic landscape was, until recently, built largely around AChR-antibody-positive patients. MuSK-positive patients respond differently and have had fewer targeted options; seronegative patients face the narrowest set of choices, with insurers often unwilling to approve biologics absent a confirmed subtype. The recent approvals of nipocalimab (Imaavy) and inebilizumab (Uplizna) broaden coverage to AChR- and MuSK-positive populations, but response remains unpredictable and seronegative patients remain underserved.

Neurologists also noted that validated scales such as the MG-ADL do not capture the full burden patients experience. Generalized fatigue - distinct from neuromuscular weakness - commonly persists even when functional scores improve substantially, pointing to a need for next-generation outcome measures alongside therapies designed to address the full clinical picture.

4. Access, Cost, and the Gap Between Approval and Adoption

For many patients, neurologists were emphatic that access - not efficacy - is the the binding constraint. Step-therapy requirements, high biologic costs, and prior-authorization burden all delay agents that physicians increasingly want to use earlier in the treatment algorithm. The tension is acute: clinical judgment increasingly favors earlier biologic use, yet the insurance and formulary architecture typically requires documented failure on multiple prior therapies first, including prolonged steroid exposure with its attendant risks.

Competitive pricing pressure across a more crowded biologic landscape may eventually ease some of these constraints - a dynamic neurologists cited as a potential pathway to broader and earlier access. For now, however, the administrative and financial barriers to biologic use remain among the most clinically significant unmet needs in gMG management.

5. Treatment Guidelines and Head-to-Head Evidence

The pace of new approvals has outrun the guidance clinicians need to choose among options. With multiple biologics now spanning FcRn inhibition and complement blockade - and no head-to-head data - treatment decisions rest on practical factors such as dosing convenience, route of administration, patient preference, and prior therapies tried, rather than on comparative efficacy or durability. Updated international consensus guidelines incorporating the new approvals and real-world experience since 2022 are needed, especially for neurologists outside academic neuromuscular centers who face this complexity without specialist infrastructure.

Treatment Evolution

Today, seven biologics are now approved across three mechanisms - FcRn inhibitors, complement inhibitors, and B-cell depleters. Despite expanded options, pharmacological remission remains rare, access barriers persist, and seronegative patients remain underserved. Near term, increased competition is expected to apply pricing pressure and ease formulary restrictions, allowing earlier biologic use, and updated international consensus guidelines are anticipated to help community neurologists navigate a more complex treatment algorithm. Longer term, KOLs anticipate therapies targeting novel mechanisms - CAR-T/CAAR-T, memory B-cell inhibitors, oral agents - and potential combination strategies. A therapy inducing true treatment-free remission would be game-changing for the field.

The Bottom Line

The MG treatment landscape has changed substantially in recent years, with four additional biologics approved since 2022. But more options do not automatically mean more answers. The unmet needs neurologists describe - sustained remission, crisis prevention, coverage for heterogeneous patient subtypes, equitable access, and an evidence base that enables informed choice - define the commercial and clinical opportunity ahead. The precision benchmarks, threshold data, and market modeling that translate these needs into actionable strategy are available in the full MarketVue® report.