Efficacy of CIDP Treatments Immunoglobulin 10% and Efgartigimod Compared Through Matching-Adjusted Indirect Analysis

Author(s):

Key Takeaways

The MAIC compared IVIg and SC efgartigimod using patient-level data from ProCID and ADHERE trials, focusing on treatment outcomes and deterioration post-ECI.
Efgartigimod, an FcRn blocker, was approved for CIDP in 2024, following its initial FDA approval for myasthenia gravis in 2021.
MAICs offer valuable insights into treatment efficacy when head-to-head randomized controlled trials are unavailable, aiding in CIDP treatment decisions.

A recently presented matching-adjusted indirect comparison assessed outcomes of immune globulin subcutaneous (Human)-ifas, 10% solution versus subcutaneous efgartigimod in patients with CIDP.

David Cornblath, MD

(Credit: ResearchGate)

In a newly presented matching-adjusted indirect comparison (MAIC), researchers evaluated the efficacy of immunoglobulin (IVIg) subcutaneous (SC) 10% solution maintenance therapy (Panzyga; Pfizer) compared with SC efgartigimod (Vyvgart Hytrulo; Argenx) in the treatment of patients with chronic inflammatory demyelinating polyneuropathy (CIDP). For this analysis, investigators used individual patient data and reported outcomes from previously published clinical trials of the CIDP therapies.¹

The MAIC utilized patient-level data from 2 IVIg arms in the phase 3 ProCID trial (NCT02638207; 1.0 g/kg, n = 69; 2.0 g/kg, n = 36) and matched these to aggregate baseline characteristics of patients from Stage A of the phase 2 ADHERE trial (NCT04281472) of SC efgartigimod (n = 322). Matching was based on key clinical variables such as age and sex. Only patients in the ProCID study who had achieved confirmed Evidence of Clinical Improvement (ECI) were included in the comparison with SC efgartigimod-treated patients entering Stage B of ADHERE (n = 111).

The first MAIC focused on outcomes during the Stage A treatment period of the ADHERE trial and included measures such as confirmed ECI (treatment responders), change from baseline in adjusted Inflammatory Neuropathy Cause and Treatment score, Inflammatory Rasch-built Overall Disability Scale score, grip strength in both hands, and Medical Research Council sum score. Researchers also conducted a second MAIC to compare deterioration following confirmed ECI in both IVIg arms of the ProCID study with outcomes observed in the Stage B portion of the ADHERE study among SC efgartigimod-treated patients.

Results from this analysis were presented at the 2025 Peripheral Nerve Society (PNS) Annual Meeting, held May 17-20, in Edinburgh, Scotland, by lead author David Cornblath, MD, a professor in the Department of Neurology at Johns Hopkins University. Investigators noted that although head-to-head randomized controlled trials remains the standard for establishing comparative efficacy, the use of MAICs may offer meaningful insights when such trials are lacking. Overall, these analyses aimed to provide additional evidence to help guide treatment decisions for patients with CIDP.

Efgartigimod, a neonatal Fc receptor (FcRn) blocker, received its first FDA approval in 2021 as a medication for generalized myasthenia gravis.² Nearly 2 years later, the agency gave greenlight to a SC formulation, a more enhanced version coformulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE drug delivery technology, which further allows for SC delivery of biologics that are typically administered via infusion.³ Efgartigimod was then approved in June 2024 to treat patients with CIDP, becoming the first and only FcRn blocker indicated for this patient population supported by results from the ADHERE trial.^4,5

Click here for more PNS 2025 coverage.

REFERENCES
1. Cornblath D, Wilson J, Baquie M, Wissmann C, Clodi E. Intravenous Immunoglobulin Versus Subcutaneous Efgartigimod For CIDP: Matching-Adjusted Indirect Comparison. Presented at: 2025 Peripheral Nerve Society (PNS) Annual Meeting; May 17-20; Edinburgh, Scotland. P455.
2. FDA Approves New Treatment for Myasthenia Gravis. News release. FDA. December 17, 2021. Accessed May 19, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-myasthenia-gravis
3. argenx Announces U.S. Food and Drug Administration Approval of VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) Injection for Subcutaneous Use in Generalized Myasthenia Gravis. News release. Argenx. June 20, 2023. Accessed May 19, 2025. https://www.globenewswire.com/news-release/2023/06/20/2691658/0/en/argenx-Announces-U-S-Food-and-Drug-Administration-Approval-of-VYVGART-Hytrulo-efgartigimod-alfa-and-hyaluronidase-qvfc-Injection-for-Subcutaneous-Use-in-Generalized-Myasthenia-Grav.html
4. argenx Announces FDA Approval of VYVGART Hytrulo for Chronic Inflammatory Demyelinating Polyneuropathy. News release. Argenx. June 21, 2024. Accessed May 19, 2025. https://www.us.argenx.com/news/argenx-announces-fda-approval-vyvgart-hytrulo-chronic-inflammatory-demyelinating-polyneuropathy
9. Argenx reports positive topline data from ADHERE study of Vyvgart Hytrulo in patients with chronic inflammatory demyelinating polyneuropathy. News release. Argenx. July 17, 2023. Accessed May 19, 2025. https://www.globenewswire.com/news-release/2023/07/17/2705309/0/en/argenx-Reports-Positive-Topline-Data-from-ADHERE-Study-of-VYVGART-Hytrulo-in-Patients-with-Chronic-Inflammatory-Demyelinating-Polyneuropathy.html