ARO-CFB

ARO-CFB is an investigational small interfering RNA (siRNA) therapeutic developed by Arrowhead Pharmaceuticals. It is designed to reduce hepatic expression of complement factor B (CFB), a key protein in the activation and amplification of the alternative complement pathway. By silencing CFB production in the liver, ARO-CFB aims to inhibit alternative pathway activity, which plays a central role in the pathogenesis of complement-mediated kidney diseases such as immunoglobulin A nephropathy (IgAN). Interim clinical data have shown that ARO-CFB can achieve deep and durable reductions—up to 90%—in circulating CFB protein levels and near-complete inhibition of alternative pathway activity for over three months after dosing. The drug is administered subcutaneously and has demonstrated a favorable safety profile in early-phase studies[1][4][6][8].