Multi-Center Study: The Biochemical Efficacy, Safety and Tolerability of a New α₁-Proteinase Inhibitor, Zemaira

Abstract

Augmentation therapy with a plasma derived α₁-Proteinase Inhibitor (α₁-PI) has been demonstrated to be effective in restoring serum Alpha₁-antitrypsin (AAT)* levels in individuals with AAT Deficiency (note: α₁ PI and AAT are synonymous). The objective of this study was to demonstrate that the steady-state trough serum α₁-PI levels, achieved by a new plasma derived α₁-PI (Zemaira, study drug, ZLB Behring LLC, King of Prussia, Pennsylvania, USA), were bioequivalent to those achieved by the currently available α₁-PI therapy, Prolastin (control drug, Bayer Corporation, Berkeley, California, USA), and maintained weekly trough serum antigenic α₁-PI levels above the protective threshold of 11 μ M. This multi-center, controlled study randomized a total of 44 subjects to receive either study or control drug for a 10-week double-blind phase. The control group was then crossed over to receive the study drug for the remainder of the study (14 weeks). The difference in mean trough serum antigenic α₁-PI level between the treatment groups was 1.45 μ M (90% CI-2.77, −0.13), signifying bioequivalence. The mean trough serum antigenic α₁-PI level in the study drug group was greater than the therapeutic threshold of 11 μ M, achieving a level of 17.7 μ M during the steady-state period. Treatment-related adverse events (AEs) were seen in 7% and 21% of study and control drug treated subjects, respectively. No documented viral transmission occurred. These results demonstrate that the new plasma derived α₁-PI (Zemaira) is bioequivalent to the currently available product Prolastin, is well tolerated, and safe with respect to the risk of viral transmission.

Key words:

• α₁-antitrypsin deficiency
• Proteinase inhibitor
• Efficacy
• Safety.