CSL Behring, a global leader in the plasma protein biotherapeutics industry, has received approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for its Hizentra (Immune Globulin Subcutaneous [Human]) in treatment of primary immunodeficiency (PID) and for treatment of secondary immunodeficiency (SID). Hizentra is now the first and only 20 per cent subcutaneous immunoglobulin (SCIg) therapy in the world for the treatment of these conditions, both of which belong to group of rare and serious diseases of the immune system.

The Japanese MHLW approval marks the first time any SCIg therapy has been approved for use in Japan.

The approval was based on a phase III study of 25 Japanese patients that found when patients converted from intravenous immunoglobulin (IVIg) treatment to dose-equivalent Hizentra therapy, serum IgG (immunoglobulin), trough concentrations increased to levels higher than those seen with their previous IVIG therapy. Results showed that Hizentra provided effective passive immunity in adults and children, which controlled most recurrent infections and improved patients' overall quality of life.

"We are extremely pleased to have secured this important regulatory approval for Hizentra in Japan," said Christopher Church, vice president and general manager, CSL Behring Asia Pacific Ltd. "For some years, Hizentra has been available in several areas of the world, making a positive difference for patients and healthcare professionals who seek innovative options for treating PID and SID. Today, it is gratifying to bring such an option to Japan, where Hizentra can address a clear, unmet medical need."

In a prospective multi-centre, open-label, single-arm phase III study of Hizentra, 25 Japanese patients with PID, who required IgG replacement therapy, were evaluated following an IVIg treatment period. Patients received three infusions of IVIG therapy, followed by a 12-week wash-in/wash-out period where treatment was converted to Hizentra and a 12-week efficacy period. The primary endpoint was achievement of the total serum IgG trough levels with SCIg therapy as compared to the preceding IVIg treatment period. Secondary efficacy and safety endpoints included all infections and local reactions to treatment and adverse events (AEs).

Findings demonstrated that Hizentra produced serum IgG trough concentrations higher than those seen during previous IVIg therapy. Mean serum IgG levels increased from 6.51 (1.32) g/L in the IVIG period to 7.28 (1.47) g/L in the SCIg efficacy period. During the efficacy period, no serious bacterial infections were reported; 11 patients experienced a non-serious infection. Ninety-six percent of the patients enrolled experienced at least one AE; most common AEs were local reactions of mild intensity. No related serious AEs were reported.

Immunodeficiencies constitute a group of more than 150 diseases that affect the cells, tissues and proteins of the immune system. In people with PID or SID, the immune system is either absent or functioning inadequately, leaving them more susceptible to infection. In children, especially, infections may not improve with treatment as expected and may keep returning. As a result, patients may face repeated rounds of antibiotics and hospitalization for treatment. Repeated infections can lead to organ damage, which, over time, can become life threatening.

CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services.