Chronic inflammatory demyelinating polyradiculoneuropathy - Emerging treatments

Efgartigimod alfa/hyaluronidase

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is associated with pathogenic IgG autoantibodies. The neonatal Fc receptor (FcRn) extends the half-life of endogenous IgG by binding and transporting it back to the cell surface to prevent it from being degraded by lysosomes. Efgartigimod alfa is a humanized IgG1-derived Fc fragment that competitively inhibits FcRn. Efgartigimod alfa is available as an intravenous formulation, or a subcutaneous formulation (coformulated with hyaluronidase). The subcutaneous formulation, efgartigimod alfa/hyaluronidase, is approved by the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of CIDP after positive results from the phase 2 ADHERE trial.[163] However, in June 2025, the FDA announced that it is evaluating the need for regulatory action after observing a potential safety signal of severe worsening of CIDP in some patients switched from traditional intravenous immune globulin (IVIG) to efgartigimod alfa/hyaluronidase.[164] One phase 4 trial is under way to assess the safety and tolerability of transition to efgartigimod alfa/hyaluronidase in patients with CIDP within 1 week of stopping IVIG.[165] The intravenous formulation of efgartigimod alfa is not approved for the treatment of CIDP.

Rozanolixizumab

Rozanolixizumab is a humanized IgG4 monoclonal antibody that binds to FcRn. It is approved by the FDA and the EMA for the treatment of generalized myasthenia gravis. It has been assessed in a phase 2 trial for CIDP, which did not show efficacy but was well tolerated, with an acceptable safety profile.[166]

Serum neurofilament light chain

Serum neurofilament light chain (sNfL) levels are correlated with axonal damage, and so have the potential to be used as a serum biomarker of disease activity and treatment response in patients with CIDP. Studies have demonstrated that higher sNfL levels are associated with an active disease state (nonresponders and patients who relapse after intravenous immune globulin withdrawal), and treatment-naive patients beginning induction therapy had higher levels of sNfL than those on maintenance therapy or in long-term remission without treatment. Larger studies will be needed to determine how to potentially utilize this to guide management of CIDP.[153][167][168][169]