Real-World Efgartigimod Data Can Guide gMG Care: Emily Chen, PharmD, BCPPS

Investigators at University of Virginia (UVA) Health are evaluating how efgartigimod performs for patients with generalized myasthenia gravis (gMG) in routine practice, where antibody profiles, treatment histories, and insurance hurdles vary widely. The team outlined its single-center study in a poster at the National Association of Specialty Pharmacy (NASP) 2026 Annual Meeting & Expo.¹

Why Real-World Efgartigimod Data Matter

Patients share the same hallmark symptom of fatigable weakness, but “the underlying pathophysiology can be so different,” Emily Chen, PharmD, BCPPS, of UVA Health and lead author of the poster, said in an interview with The American Journal of Managed Care^®. Antibodies, prior treatments, and comorbidities differ from person to person.

The most recent international consensus guidance, published in 2021, predates FcRn inhibitors and newer complement inhibitors. In May 2026, the FDA expanded efgartigimod’s indications to all adults with gMG, regardless of antibody serotype,² building on pivotal evidence from the phase 3 ADAPT trial.³ Chen’s team wants to understand how efgartigimod affects specific populations and whether it can help reduce corticosteroid doses. “It would be nice to know where it might fit into the treatment of MG in general, but also maybe specifically in certain cases,” Chen said.

The observational cohort study spans January 2024 through December 2026. It includes patients 16 years or older with gMG meeting the Myasthenia Gravis Foundation of America class II, III, or IV criteria who received at least 1 cycle or 4 doses. The primary outcome is average time on therapy from initiation to discontinuation. Secondary outcomes include change in the Myasthenia Gravis Activities of Daily Living score through week 12, discontinuation events, and the proportion of patients achieving minimal symptom manifestation.¹

Specialty Pharmacy Workflow and Prior Authorization

At UVA, the clinic provider enters the prescription and notifies the pharmacist, who speaks with the patient and identifies prior authorization needs. A medication access technician then completes the authorization and enrolls the patient in financial assistance when needed.

As FcRn inhibitors are expensive, the path to a first dose hinges on insurance coverage and whether a patient meets both label and payer criteria. Whether the formulation is administered by a health care provider or at home also affects the timeline, and many patients need copay assistance to stay on therapy.

“That whole PA [prior authorization] process can take 1 to 2 weeks,” Chen said.

Faster-Acting gMG Therapies Shift Expectations

Chen explained that traditional therapies other than intravenous immunoglobulin and plasma exchange can take 6 to 18 months to take effect. “With some of these newer therapies like complement inhibitors and [FcRn inhibitors], you can see the effect a lot quicker,” she said.

That speed may help keep patients out of the hospital during exacerbations and reduce missed workdays. “Patients are able to live their lives without as much interruption, and that’s really helpful,” Chen said.

Future Directions for Pharmacist-Led gMG Care

The investigators plan to stratify outcomes by antibody status and by early- vs late-onset disease. They also aim to standardize pharmacy involvement in therapy initiation, assessment, and monitoring. In addition, they will examine whether pharmacist-led coordination shortens time to first dose or speeds therapy transitions.

Chen added that newer options may let clinicians scale back older treatments, noting the potential “to tweak or modify therapies that traditionally have been used for treatment of MG that can also have long-term chronic side effects.”

References

1. Chen E, Holian A, Petitte M. Real-world clinical outcomes with efgartigimod and efgartigimod hyaluronidase in patients with generalized myasthenia gravis at an adult neuromuscular clinic: a single-center retrospective review. Poster presented at: National Association of Specialty Pharmacy 2026 Annual Meeting & Expo; September, 2026; National Harbor, Maryland.
2. Shaw M. FDA clears efgartigimod for all adult patients with myasthenia gravis. AJMC. May 11, 2026. Accessed October 27, 2026. https://www.ajmc.com/view/fda-clears-efgartigimod-for-all-adult-patients-with-myasthenia-gravis
3. Howard JF Jr, Bril V, Vu T, et al; ADAPT Investigator Study Group. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021;20(7):526-536. doi:10.1016/S1474-4422(21)00159-9