Pegcetacoplan Subcutaneous Infusion: Early Access and Patient Training

Background: Alternative complement cascade dysregulation is the main pathogenesis for C3 Glomerulopathy (C3G) and idiopathic immune complex membranoproliferative glomerulonephritis (IC-MPGN). Both diseases are progressive, with a 30-50% risk of kidney failure within 10 years and high risk for recurrence after kidney transplant. Often treated with corticosteroid and mycophenolate with variable success, oral complement inhibitor, iptacopan, has been approved for C3G therapy. Pegcetacoplan showed positive results for both C3G and IC-MPGN in pre- and post-transplant patients. It binds to complement protein C3 and C3b, thereby regulating the cleavage of C3 and the generation of downstream effectors of complement. Pegcetacoplan is a self-administered subcutaneous infusion (SCI), eliminating the need for infusion center visits. Methods: Pegcetacoplan SCI 1080 mg twice weekly was given to 8 patients with C3G and IC-MPGN via the Freedom Edge syringe infusion system with an infusion time of 30-60 minutes. Patients or caregivers were trained by a nurse either inperson or remotely. Patient demographics are detailed in the table. The cohort was equally split between male and female, IC-MPGN and C3G, pre- and post-transplant. Results: All participants received 1 in-person training session except one who received 2 in-person sessions, one patient had one remote session. Our cohort accumulated a total of 67 months with twice weekly SCI of pegcetacoplan. Only one patient discontinued the therapy after two months due to a lack of benefit. There were no reported difficulties or adverse events related to the SCI administration or adverse effects with pegcetacoplan Conclusion: Patients can safely self-administered SCI infusions of pegcetacoplan with minimal training. One in-person training session was sufficient to make patients competent in self-administration and remote training was also successfully used. - Patient Age Sex Race Education Diagnosis Post-Transplant Training Duration of Therapy 1 26 M White College IC-MPGN Yes One sessionInperson 10 months 2 45 M Hispanic High School IC-MPGN No One sessionInperson 9 months 3 35 M White College C3G Yes One sessionInperson 24 months 4 65 F White College C3G Yes One sessionInperson 7 months 5 56 F White College IC-MPGN No One sessionInperson 7 months 6 21 M White College C3G No One sessionRemote 5 months 7 62 F IC-MPGN College IC-MPGN No Two sessionsInperson 2 months 8 21 F White High School C3G Yes One sessionInperson 3 months