Wrap-Up: Vyvgart Targets Four Populations at AAN 2026

A Summary of the 2026 AAN Annual Meeting for Rare Diseases from a Managed Care Perspective – Myasthenia Gravis and CIDP.

In addition to the seronegative data from the ADAPT SERON trial for seronegative gMG, results from the ADAPT OCULUS trial for purely ocular gMG will bring new hope to patients with these diseases. Moreover, the data from the ADAPT trial program for rare diseases such as gMG and CIDP, will signal a shift to more targeted biologic options for the treatment of these conditions. Results and data from pediatric efficacy and treatment-naive CIDP will require updates in the specialty pharmacy frameworks and step-therapy protocols for many of the rare diseases managed today. As for managed care organizations, this meeting will require them to set up coverage criteria for what is expected to be a wave of products that will receive FDA approval in the near future. Below is a summary of highlights from the meeting along with links to detailed articles for those interested in more information.

Target 1: Seronegative Myasthenia Gravis

The results of the ADAPT SERON trial for seronegative gMG have been released. The results will be used to support a supplemental Biologics License Application (sBLA) for the treatment of adults with acetylcholine receptor antibody-seronegative (AChR − ) generalized myasthenia gravis (gMG). The sBLA has been granted priority review by the FDA with a target action date of May 10, 2026 for Vyvgart (efgartigimod alfa) as reported by Argent.

ADAPT SERON

The study was a Phase 3, double-masked, placebo-controlled trial. The primary endpoint for the study was MG-ADL total score at Week 4. Patients treated with efgartigimod showed a significant improvement in MG-ADL total score from baseline to 4 weeks compared to placebo. The mean reduction in MG-ADL score for patients treated with efgartigimod was 3.35 compared to a mean reduction of 1.90 for patients treated with placebo. The open-label extension study demonstrated that the improvement in MG-ADL score for patients treated with efgartigimod in the double-masked study was enhanced in subsequent treatment cycles in all three groups of antibody-negative patients. These groups of patients were those who were MuSK+ (muscle-specific kinase), LRP4+ (low-density lipoprotein receptor-related protein 4), or triple seronegative (all of the above antibodies were less than 0.6).

Implications for Managed Care

Results of the trial will allow for a supplemental Biologics License Application (sBLA) for seronegative gMG to be given priority review with a target action date of May 10, 2016. For managed care organizations treating seronegative patients with gMG, the findings will allow for a narrow window of time for the organization to create a plan for the management of patients taking Vyvgart for gMG, if approved by the FDA in the spring of a few months. The population of seronegative gMG patients make up about 10% to 15% of all gMG patients and currently do not have any targeted biologic therapy options for their disease.

Seronegative gMG will likely be reviewed through current specialty pharmacy policy parameters rather than as a new molecular entity for which current policy would typically apply. It is currently approved and on formulary for AChR+ gMG patients and plans will need to update the clinical criteria to address the unique antibody profile of seronegative patients.

Unique Targeted Mechanism

The way in which Vyvgart works is via FcRn receptor blockade to reduce the circulating levels of all Immunoglobulin G (IgG) in the blood, regardless of the subclass of IgG involved in the disease. Therefore, this mechanism of action is unique compared with other candidates in the pipeline for gMG. The ADAPT SERON study results are therefore not unexpected and confirm the results seen in the AChR positive patients in the adaptive phase 3 clinical trials.

READ MORE: Vyvgart Crosses the Antibody Line in Seronegative Myasthenia Gravis

Target 2: Ocular Myasthenia Gravis

Efgartigimod Alfa for Ocular Myasthenia Gravis (oMG): a Biologic for a Disease Long Without a Pharmacological Option. Results of the ADAPT OCULUS trial with efgartigimod alfa (Vyvgart) have been released by argenx at the 2026 AAN Annual Meeting. ADAPT OCULUS is a Phase 3, double-blind, placebo-controlled study that is the first to investigate a targeted pharmacological treatment in patients with oMG. In the trial, the FcRn antagonist Vyvgart resulted in improvements in patient reported and clinically measured end points in patients with oMG. These findings will support the upcoming supplemental Biologics License Application (sBLA) to the FDA for Vyvgart in oMG.

ADAPT OCULUS

ADAPT OCULUS is a 24-week, double-blind, placebo-controlled Phase 3 clinical trial to evaluate Vyvgart in oMG patients in a globally diverse patient population. The trial demonstrated statistically significant improvements from baseline to Week 4 in MGII Patient-Reported Outcome (PRO) ocular subscore for Vyvgart-treated patients (P=0.012) compared to placebo-treated patients. Also, in a combined assessment of patients’ self-reported impairment and physicians’ examination findings, a statistically significant improvement was observed for Vyvgart-treated patients (P=0.018). This endpoint assesses symptoms that are typical for oMG, including diplopia (double vision) and ptosis (drooping of the upper eyelid).

Implications for Managed Care

Vyvgart is now a targeted biologic therapy option for oMG patients. Most oMG patients are currently treated with a combination of corticosteroids and cholinesterase inhibitors. As a new class of medication, Vyvgart will require managed care organizations to establish clinical criteria for patient selection and other coverage determinations that are different from those used for AChR-positive gMG patients. Such clinical criteria will need to address the disease severity and specific clinical manifestations found in oMG patients as well as the treating specialists for these patients.

The step therapy model that is already in place for the treatment of gMG with Vyvgart would likely be implemented for the oMG indication as well. Many patients with oMG are on long term corticosteroids and can experience severe toxicity as they age. A steroid-sparing indication for Vyvgart would be considered in a cost-effectiveness analysis and would likely be subject to the same step therapy requirements as the gMG indication.

READ MORE: Ocular Myasthenia Gravis Gets Its First Targeted Biologic Candidate.

Target 3: Pediatric Track Record in Myasthenia Gravis

Adolescent gMG patients were studied in the ADAPT Jr trial, which is the focus for Managed care organizations treating rare autoimmune neuromuscular conditions affecting the adolescent population. Argenx, the company that markets efgartigimod alfa (Vyvgart), a FcRn inhibitor for the treatment of generalized myasthenia gravis, recently presented data on adolescent gMG patients from the ADAPT Jr trial at the 2026 AAN Annual Meeting. As expected, the results for the adolescent gMG patients were consistent with those adult gMG patients studied in the ADAPT trials.

ADAPT Jr

The percentage of patients who achieved MSE in Cycle 1 was 72.7%, and this was increased to 80% in Cycle 2. Importantly, the adolescents with Vyvgart observed in the study in terms of improvement in MG-ADL scores were consistent and repeated between the two cycles of the study. These data will support the use of Vyvgart in adolescent patients with gMG and Argent are currently enrolling a younger pediatric cohort below the age of 12 in this trial.

New Pharmacy Category for Pediatric Rare Neuroimmune Disease

Implications for an adolescent population with MG, which has historically been managed with the use of immunosuppressive agents, many of which have significant toxicity with long-term use, an FDA approval for the use of Vyvgart in adolescents with MG will open up a new specialty pharmacy category for the management of pediatric rare neuroimmune disease for which most payers have not yet established formulary and utilization management criteria to manage such drugs. Plans managing pediatric specialty drug spend will have to consider clinical criteria for the use of Vyvgart in adolescents with MG that address the following: 1) diagnosis of MG; 2) treating specialist; and 3) measure of disease severity for which rating scales used in adults may not be appropriate for use in adolescents.

Payer strategies to manage pediatric specialty drug spend for such a disease will likely encounter similar criteria to that for treatment of children with rare diseases as a whole. Such criteria would include confirmation of precise and specific diagnosis by a specialist; definition of degree of severity on an appropriate scale for the patient’s age.

READ MORE: Efgartigimod Builds a Pediatric Track Record in Myasthenia Gravis

Target 4: Step Therapy Model in Treatment-Naive CIDP

We outline new evidence to challenge step therapy for the first to be targeted biologic candidate for CIDP, presented by argenx at the 2026 AAN Annual Meeting. Results from a post hoc analysis of the ADHERE study will be provided for 87.5% of treatment naive CIDP patients who received the subcutaneously administered formulation of efgartigimod alfa (Vyvgart Hytrulo, efgartigimod alfa and hyaluronidase-qvfc) who achieved confirmed clinical response. Early benefits were noted for this treatment in this population of patients with CIDP.

ADHERE Post Hoc Data

Chronic inflammatory demyelinating polyneuropathy (CIDP) is an autoimmune condition in which the body’s immune system attacks the peripheral nerves causing progressive weakness and impaired sensation in affected areas. CIDP affects between 40,000 and 100,000 people in the United States and is typically treated with intravenous immunoglobulin (IVIG) in newly diagnosed patients. However, argenx’ efgartigimod alfa (Vyvgart), a first-in-class monoclonal antibody that targets and reverses Pathogenic Antibodies (PAb), has been shown to be very effective in treating patients with CIDP who have failed on or are intolerable to IVIG. Therefore, Vyvgart is considered to be a steroid-sparing agent and is typically used as a treatment option following failure or intolerance of IVIG. Results from a post hoc analysis of the ADHERE study presented at the 2026 AAN Annual Meeting support earlier positioning of Vyvgart in the treatment of CIDP. The post hoc analysis found that 87.5% (n=16/18) of treatment naive CIDP patients treated with Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), the subcutaneous formulation of efgartigimod alfa, achieved a confirmed clinical response with treatment related response observed as early as cycle 1.

Implications for Healthsystems

IVIG are considered to be a “generic” treatment, but cost significantly. While many of these agents are administered intravenously (IV) to a patient in a clinic setting (e.g. a hospital or infusion suite), others are administered by subcutaneous injection (SC) and can be taken by a patient at home. The subcutaneous agent, Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), is more expensive on a per unit basis than IVIG, but can lead to a reduced total cost of care for the treatment of patients with CIDP, given the cost of the infusions, the nursing, etc. required for IVIG-treated patients.

Patients with CIDP who are in need of treatment will have their physicians attempt to get the IVIG step therapy requirements removed or relaxed in order to provide the best treatment possible for their condition as well as to try to keep them on a less expensive medication for as long as possible. Many patients with CIDP have contraindications to IVIG, do not have access to IVIG infusion centers, or other reasons that would allow for suboptimal treatment of their neuropathy over time with less expensive IVIG instead of Vyvgart Hytrulo.

READ MORE: Vyvgart Hytrulo Challenges the Step Therapy Model in Treatment-Naive CIDP

References

1. argenx SE. argenx brings neuromuscular leadership to AAN 2026 with new data supporting broader Vyvgart use across MG and CIDP. GlobeNewswire. April 18, 2026. https://www.globenewswire.com/news-release/2026/04/18/3276554/0/en/argenx-Brings-Neuromuscular-Leadership-to-AAN-2026-with-New-Data-Supporting-Broader-VYVGART-Use-Across-MG-and-CIDP.html
2. argenx SE. argenx brings neuromuscular leadership to AAN 2026 with new data supporting broader Vyvgart use across MG and CIDP. GlobeNewswire. April 18, 2026. https://www.globenewswire.com/news-release/2026/04/18/3276554/0/en/argenx-Brings-Neuromuscular-Leadership-to-AAN-2026-with-New-Data-Supporting-Broader-VYVGART-Use-Across-MG-and-CIDP.html
3. argenx SE. argenx brings neuromuscular leadership to AAN 2026 with new data supporting broader Vyvgart use across MG and CIDP. GlobeNewswire. April 18, 2026. https://www.globenewswire.com/news-release/2026/04/18/3276554/0/en/argenx-Brings-Neuromuscular-Leadership-to-AAN-2026-with-New-Data-Supporting-Broader-VYVGART-Use-Across-MG-and-CIDP.html
4. argenx SE. argenx brings neuromuscular leadership to AAN 2026 with new data supporting broader Vyvgart use across MG and CIDP. GlobeNewswire. April 18, 2026. https://www.globenewswire.com/news-release/2026/04/18/3276554/0/en/argenx-Brings-Neuromuscular-Leadership-to-AAN-2026-with-New-Data-Supporting-Broader-VYVGART-Use-Across-MG-and-CIDP.html