FDA Approves Vyvgart Hytrulo for Rare Neuromuscular Disorder CIDP

The US Food and Drug Administration (FDA) has granted approval to argenx’s Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for adult patients suffering from chronic inflammatory demyelinating polyneuropathy (CIDP). This approval distinguishes Vyvgart Hytrulo as the inaugural neonatal Fc receptor blocker sanctioned for the treatment of this rare autoimmune disorder.

CIDP is an uncommon and often progressive immune-mediated neurological condition affecting the peripheral nervous system. It leads to muscle weakness, fatigue, and sensory loss in the limbs. In the United States, approximately 24,000 individuals are receiving treatment for CIDP, with 85% requiring continuous therapy and nearly 88% of those under treatment continuing to experience residual impairment and disability.

Vyvgart Hytrulo has been approved for administration as a subcutaneous injection, which takes between 30 and 90 seconds and is given once a week. Its mechanism of action involves reducing circulating levels of immunoglobulin G (IgG), which is implicated in the pathology of CIDP.

The FDA’s decision to approve Vyvgart Hytrulo was primarily based on the findings of the ADHERE study. This study involved 322 adult patients with CIDP who had not been actively treated in the previous six months, had recently been diagnosed, or were undergoing treatment with either IgG therapy or corticosteroids. The study results were compelling, showing that 69% of patients treated with Vyvgart Hytrulo exhibited clinical improvement, including enhancements in mobility, function, and strength. Furthermore, the study achieved its primary endpoint by demonstrating a 61% reduction in the risk of relapse compared to a placebo. The safety results were consistent with Vyvgart’s established safety profile from previous clinical trials.

Argenx has announced that this combination treatment will be made available to US patients immediately. Luc Truyen, the chief medical officer at argenx, emphasized the significance of the clinical evidence, stating, "We have generated strong clinical evidence that Vyvgart Hytrulo can meaningfully improve and stabilize disease symptoms with a favorable safety profile and a simple route of administration."

In addition to its recent approval for CIDP, Vyvgart Hytrulo is already authorized in the United States for the treatment of adult patients with generalized myasthenia gravis (gMG). This rare and chronic autoimmune disease affects patients who are positive for anti-acetylcholine receptor (AChR) antibodies. Moreover, argenx’s subcutaneous formulation of Vyvgart has also been approved by the Medicines and Healthcare products Regulatory Agency for use alongside standard therapy in adult gMG patients who are AChR antibody-positive.

The approval of Vyvgart Hytrulo represents a significant advancement in the treatment of CIDP, offering a new therapeutic option for patients grappling with this challenging and debilitating condition. The convenience of a subcutaneous injection combined with its demonstrated efficacy and favorable safety profile positions Vyvgart Hytrulo as a promising treatment for those affected by CIDP.