European Commission Approves argenx's Vyvgart (Efgartigimod Alfa) in [INDICATION]

What the Approval Covers

argenx SE, a global immunology company focused on treating severe autoimmune diseases, has announced that the European Commission (EC) has approved VYVGART® (efgartigimod alfa) 1000mg for subcutaneous injection as a standalone treatment for adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP). This approval applies to patients who have previously been treated with corticosteroids or immunoglobulins. The therapy can be self-administered or given by a caregiver or healthcare professional and is available in both vial and prefilled syringe forms. Treatment begins with a weekly injection and can be adjusted to every other week based on a doctor’s evaluation.

CIDP is a rare and often progressive autoimmune disease that affects the peripheral nervous system. It causes symptoms such as difficulty standing, pain, fatigue, and frequent falls. In more advanced cases, patients may become wheelchair-bound and unable to work. Most patients require long-term treatment, and many continue to experience lasting disability even with therapy.

What Doctors and Patient Groups Are Saying

Jean-Philippe Plançon, President of EPODIN, stated, "The EC’s decision has been met with hope and enthusiasm by the European Patient Organisation for Dysimmune and Inflammatory Neuropathies (EPODIN). We see the introduction of a new targeted therapy for CIDP as a major step forward for the patient community.”

Dr. Luis Querol, M.D., Ph.D., ADHERE Investigator, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain, said, “CIDP can severely affect quality of life by causing weakness, loss of balance and mobility, numbness and pain in a patient’s arms and legs. For far too long, physicians have had limited options for helping to improve patient outcomes. The approval of VYVGART SC for the treatment of CIDP marks a turning point in clinical practice, as physicians now have access to a new, effective treatment option that, for the first time, precisely targets a key mechanism of disease and provides meaningful functional improvements to patients.”

Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx, commented, “VYVGART SC is the first therapy with a novel mechanism of action to be approved for this community in more than 30 years. With VYVGART SC, CIDP patients and physicians across Europe will soon have access to an effective novel therapy with a favorable safety profile that has a precise mechanism of action and a convenient self-injection option. This approval further affirms the potential of efgartigimod in IgG-mediated autoimmune diseases.”

Clinical Data Behind the Approval

The approval of VYVGART SC by the EC follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) and is supported by results from the ADHERE clinical trial, which is the largest study conducted in CIDP patients to date. In the trial, 66.5% of patients treated with VYVGART SC showed clinical improvement in areas such as mobility, physical function, and strength. The benefits were observed across all patient subtypes, regardless of their previous treatments. The study met its main objective, showing a 61% reduction in the risk of disease relapse compared to placebo. Nearly all participants (99%) chose to continue into the open-label extension phase. The safety profile remained consistent with previous studies of VYVGART SC.

This approval is valid across all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway. argenx is coordinating with local health authorities to make VYVGART SC available to eligible patients across these regions. This marks the second approval of VYVGART SC in Europe, following its earlier authorization as an add-on treatment for adults with generalized myasthenia gravis who are anti-AChR antibody positive.This isn't the only recent CIDP approval news, VYVDURA, a related efgartigimod formulation, recently secured approval for CIDP treatment in Japan too, signaling wider global momentum for FcRn-blocker therapies in this space.

Source: The CHMP opinion and European Commission decision dates for this indication are published in EMA's post-authorisation record for Vyvgart.