CHMP Recommends argenx Treatment for Chronic Inflammatory Demyelinating Polyneuropathy

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Netherlands-based argenx has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended its efgartigimod alfa treatment, brand name Vyvgart, in 1000-mg subcutaneous injection, as a monotherapy for adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) who have been previously treated with corticosteroids or immunoglobulins (1).

According to a press release from argenx, efgartigimod is an antibody fragment that reduces pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor, thereby blocking the process by which IgG is recycled (1). The company’s efgartigimod alfa treatment is the first and only targeted IgG Fc-antibody treatment for CIDP, argenx said, and if approved, it would be the first novel mechanism of action for treatment of CIDP in more than 30 years.

CIDP is a serious, though rare, autoimmune disease of the peripheral nervous system (1). People diagnosed with CIDP report symptoms of fatigue, muscle weakness, and a loss of feeling in their arms and legs that may come and go or may worsen over time. In those cases, the symptoms can impair a person’s ability to function in everyday life; more than one-third of those diagnosed with CIDP will need a wheelchair if they are not treated. Estimates say there are more than 31,000 people living with CIDP in the European Union (1).

In the United States, FDA approved a form of the argenx treatment, efgartigimod alfa and hyaluronidase-qvfc (under the brand name Vyvgart Hytrulo), in June 2024, for treatment of adult patients with CIDP in a once-weekly, 30- to 90-second subcutaneous injection (2).

“Our mission is to develop innovative, targeted treatments for patients with rare and severe autoimmune diseases, who continue to face significant unmet needs,” Luc Truyen, MD, PhD, and chief medical officer of argenx, said in the April 2025 release (1). “The positive CHMP opinion for Vyvgart in CIDP brings us one step closer to providing patients across Europe with a transformational new treatment option that provides meaningful functional improvement.”

As with the FDA approval, CHMP’s recommendation was based on results from a clinical trial (ADHERE) that involved the largest number of CIDP patients to date; updated data from this trial show that 66.5% of patients (214 out of 322) treated with efgartigimod alfa demonstrated evidence of clinical improvement—particularly improvements in mobility, function, and strength—regardless of prior treatment (1).

"For the patient population represented by the European Patient Organisation for Dysimmune and Inflammatory Neuropathies (EPODIN) and for those affected by CIDP, this is excellent news," said Jean-Philippe Plançon, president of EPODIN (1). "There are still considerable unmet medical needs in the management of CIDP, and CHMP’s recommendation brings renewed hope for improved treatment options and quality of life."

The European Commission is expected to make a decision on the marketing authorization application for Vyvgart within approximately two months (1).

References

1. argenx. argenx Announces Positive CHMP Opinion for Vyvgart (efgartigimod alfa) Subcutaneous Injection for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Press Release. April 28, 2025.
2. argenx. argenx Announces FDA Approval of Vyvgart Hytrulo for Chronic Inflammatory Demyelinating Polyneuropathy. Press Release. June 21, 2024.