Clinical Efficacy, Safety and Tolerability of a New Subcutaneous Immunoglobulin 16.5% (Octanorm [Cutaquig®]) in the Treatment of Patients With Primary Immunodeficiencies

Abstract

Introduction: Subcutaneously administered immunoglobulin (SCIG) is increasingly used to treat patients with primary immunodeficiencies (PIDs). Octanorm (marketed as cutaquig® in USA and Canada) is a new 16.5% solution of human SCIG, manufactured by a process based on that of the intravenous preparation (IVIG) octagam®.Objectives: To investigate the efficacy, safety and tolerability of octanorm in a prospective, open-label, single-arm phase 3 study involving adult and pediatric patients with PIDs (NCT01888484; clinicaltrials.gov/ct2/show/NCT01888484).Methods: Patients who were previously treated with IVIG received a total of 64 weekly SCIG infusions, including 12 weekly infusions during the wash-in/wash-out period, followed by 52 weekly infusions during the evaluation period.Results: A total of 61 patients aged 2–73 years received 3,497 infusions of octanorm. The mean dose per patient was 0.175 g/kg/infusion. The mean calculated dose conversion factor from the patients' previous IVIG dose for octanorm was 1.37. No serious bacterial infections developed during the study. The rate of other infections per person-year during the primary observation period was 3.43 (upper 95% CI 4.57). All but one non-bacterial infection were mild or moderate in intensity. IgG trough levels were constant during the course of the study. Eleven patients (18.0%) experienced 14 mild or moderate systemic adverse events (AEs) related to octanorm. The rate of related AEs per infusion was 0.004. In 76.7% of infusions, no infusion site reactions were observed and only two (0.3%) reactions were deemed severe. The incidence of site reactions decreased with successive infusions.Conclusion: The new 16.5% SCIG octanorm was shown to be efficacious in preventing infections in PIDs, and was well tolerated.

Treatment of Children with Primary Immunodeficiencies with a Subcutaneous Immunoglobulin 16.5% (Cutaquig ® [Octanorm])

“…In this analysis of 38 children and adolescents with PIDs treated with the 16.5% SCIG cutaquig, children and adolescents on a stable dose of IVIG who transitioned to subcutaneously administered cutaquig did not suffer from an increased incidence of severe bacterial infections and had stable IgG plasma levels, while benefiting from the increased convenience of home-based infusions. These findings are consistent with the previously reported results from the main study, which included a mixed population of adults and a small number of children [19].…”

“…A well-documented characteristic of SCIG is consistently stable IgG levels over time [5,19,39,40]. However, there are very limited data on the pharmacokinetics of SCIG in children and adolescents with PADs.…”

“…Eligibility criteria and study design have been published previously (Figure 1) [19]. In brief, patients with PADs, stable on IVIG treatment for at least six infusions (with IgG trough levels ≥5.0 g/l), underwent a 12-week washin/-out period, followed by a 52-week efficacy period (primary observation period).…”

“…Octanorm (marketed as cutaquig R in the USA, Canada and Europe; Octapharma AG, Lachen, Switzerland) is a 16.5% solution of human immunoglobulin for subcutaneous administration, manufactured utilizing the same well-established process used in the manufacture of the IVIG octagam R [18]. A Phase III, open-label clinical study examined the efficacy, safety, tolerability and pharmacokinetics of cutaquig in patients with PADs [19]. The study showed that cutaquig was effective and well tolerated in patients with PADs.…”

Reconstitution of IgG Subclasses Following Immunoglobulin Administration in Adult Patients with Common Variable Immune Deficiency

“…Immunoglobulin replacement is an established standard therapy for antibody deficiency disorders. Immunoglobulin is administered via intravenous (IVIG), subcutaneous (SCIG), and enzyme-fascilitated subcutaneous (fSCIG) routes (9)(10)(11)(12). Regardless of route of administration, immunoglobulin therapy has been effective in reducing the frequency and severity of infections [9][10][11][12][13].…”