Octagam^® 5%, an Intravenous IgG Product, Is Efficacious and Well Tolerated in Subjects with Primary Immunodeficiency Diseases

Abstract

Octagam^® is an intravenous immunoglobulin preparation registered in Europe for treating primary immunodeficiency diseases (PID). The present clinical trial was designed to demonstrate that Octagam^® meets the minimal efficacy requirement of the U.S. Food and Drug Administration—that treatment should result in ≤1 serious infection/subject/year. The objectives of this clinical trial were to show that Octagam^® meets this requirement, and to confirm the safety of Octagam^®. Forty-six subjects with well-defined PID received Octagam^® (either 400–600 mg/kg every 28 days or 300–450 mg/kg every 21 days) for 12 months. The primary efficacy variable was the number of serious infections/subject/year. The estimated infection rate was 0.1 serious infections/subject/year. The half-life (T ^1/2) of total IgG was 41 days. Adverse events potentially related to Octagam^® occurred in 5% of infusions, either during or within 30 min of the procedure. Octagam^® meets the Food and Drug Administration minimal requirement for efficacy. In addition, Octagam^® had a T ^1/2 (IgG) comparable with published data, and was well tolerated. Octagam^® treatment is safe and resulted in 0.1 serious infections/subject/year in primary immunodeficient subjects.