FDA Approves Efgartigimod Alfa and Hyaluronidase-Qvfc for Chronic Inflammatory Demyelinating Polyneuropathy

The FDA has approved efgartigimod alfa and hyaluronidase-qvfc (Vyvgart Hytrulo; argenx SE) for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP), according to a news release from argenx.¹

Image credit: Vitalii Vodolazskyi | stock.adobe.com

The once weekly 30- to 90-second subcutaneous injection is the first and only neonatal Fc receptor (FcRn) blocker that is approved for the treatment of CIDP.¹ The drug binds to the neonatal FcRn, which results in the reduction of circulating IgG.²

CIDP is a rare and progressive immune-mediated neuromuscular disorder of the peripheral nervous system. The effects of the disorder can be debilitating and include disabling sensory and mobility issues, pain and fatigue, and frequent falling, with many patients becoming wheelchair bound.¹ Due to this, 85% of patients require ongoing treatment and 88% of patients who are treated continue to have residual disability and impairment.¹

“While CIDP patients face many daily concerns and challenges, fear of disease progression should not be one of them. CIDP can be debilitating and have significant impact on quality of life and many patients with CIDP require treatments that may be burdensome,” Lisa Butler, executive director of the GBS | CIDP Foundation, said in the news release.

“The approval of this promising new treatment option for CIDP may provide hope to patients that they can treat their disease beyond just managing symptoms. CIDP patients deserve treatment options and we look forward to a future of choices for optimal and individualized care.”¹

The FDA’s approval of Vyvgart Hytrulo is based on data from the ADHERE study, which is the largest clinical trial to date analyzing CIDP. In the open-label Stage A of the study, 67% of patients (214/322) demonstrated evidence of clinical improvement based on the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score, indicating that IgG autoantibodies have an essential role in the biology of CIDP.²

Of the Stage A responders, 221 entered Stage B which evaluated the primary endpoint of relative risk of relapse. Vyvgart Hytrulo significantly reduced relapse risk compared to placebo, demonstrating a 61% risk reduction (HR: 0.39; 95% CI, 0.25-0.61). At week 24 and week 48, Vyvgart patients had a lower relapse rate compared to placebo, with 26% versus 54% and 34% versus 60%, respectively.²

Patients treated with Vyvgart Hytrulo also had a clinical benefit across all patient subgroups, regardless of any prior therapy.² Importantly, Vyvgart Hytrulo was well-tolerated among the study participants, and had a safety profile that was consistent with prior clinical trials and the known safety profile of Vyvgart.²

“Today marks a groundbreaking day for the treatment of CIDP. Existing treatments have been limited to corticosteroids and plasma-derived therapies. These treatments, while effective for many patients, can be challenging for some patients to receive,” Jeffrey Allen, MD, professor at the Department of Neurology at the University of Minnesota and principal investigator in the ADHERE trial, said in the news release.¹

“Today’s approval of Vyvgart Hytrulo gives doctors and patients a new, safe and effective treatment option that may lessen the burden of treatment that some patients experience,” Allen continued.¹

References

1. Argenx. argenx Announces FDA Approval of VYVGART Hytrulo for Chronic Inflammatory Demyelinating Polyneuropathy. News release. Published June 21, 2024. Accessed July 1, 2024. https://www.us.argenx.com/news/argenx-announces-fda-approval-vyvgart-hytrulo-chronic-inflammatory-demyelinating-polyneuropathy

2. Argenx. argenx Reports Positive Topline Data from ADHERE Study of VYVGART Hytrulo in Patients with Chronic Inflammatory Demyelinating Polyneuropathy. News release. Published July 13, 2023. Accessed July 1, 2024. https://www.us.argenx.com/news/argenx-reports-positive-topline-data-adhere-study-vyvgart-hytrulo-patients-chronic