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Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is characterized by progressive or relapsing, and often debilitating, sensory-motor deficits. Pathogenic immunoglobulin G (IgG) autoantibodies causing demyelination and/or conduction block have been implicated in CIDP pathogenesis. Efgartigimod alfa is a neonatal Fc receptor (FcRn) inhibitor that reduces circulating IgG; it is approved to treat myasthenia gravis. Investigators now report an industry-funded, international, multistage trial of efgartigimod in adults with active CIDP who worsened before study entry or relapsed after discontinuing prior treatments during a run-in phase.
In stage A (open-label), 322 patients were treated weekly, for up to 12 weeks, with subcutaneous…