Biotest’s Investigational 10% Liquid IVIG Assessed in Patients with PIDD

An investigational 10% liquid intravenous immunoglobulin (IVIG) product currently under U.S. regulatory review for marketing approval appears to be safe and effective for prophylactic treatment of patients with primary immunodeficiency disorders (PIDDs), according to a study involving 63 PIDD patients conducted at 15 U.S. clinical study sites. Developed by Biotest Pharmaceuticals, this product is manufactured from source plasma by Cohn-Oncley fractionation followed by ion exchange chromatography.

Patients ranging in age from 6 years to 75 years received a total of 746 infusions of Biotest-IVIG every three or four weeks. The mean dose per infusion was 500 mg/kg, with a range of 254 to 1,029 mg/kg. Fifty-two patients were treated with the product for at least 12 months. Two serious bacterial infections (SBIs) were observed, translating into a rate of 0.035 SBIs per person per year, which is well below the ≤1.0 SBI per patient-year target recommended by the U.S. Food and Drug Administration (FDA). Two patients were hospitalized for infection, producing a rate of 0.21 hospitalization days/patient/year. Just 2.28 work or school days/patient/year were missed due to infection; nearly two-thirds of patients did not miss a day of work or school.

The proportion of infusions with one or more temporally associated adverse events (TAAEs) was 27.7% (upper one-sided 95% confidence limit, ≤30.6%), significantly below the 40% limit recommended by the FDA. The IgG half-life for all patients was about 30 days, with significant variation among individuals. Throughout the study, however, all patients maintained an average serum IgG level above 500 mg/dL.