PURPOSE: To evaluate the safety and tolerability of Zemaira® (ZLB Behring LLC) [Z(A1-PI)] in two multiple-dose studies of patients with alpha1-antitrypsin deficiency.
METHODS: The two investigations comprised: (A) a 26-week multicenter, open-label study of weekly intravenous infusions of Z(A1-PI) 60 mg/kg (n=9); and (B) a 24-week multicenter, controlled study in 43 patients randomized 2:1 to Z(A1-PI) or an active control preparation (Talecris Biotherapeutics) [P(A1 PI)] 60 mg/kg given by weekly intravenous infusions. Adverse events (AEs), rated as mild, moderate or severe, AE rates per infusion, and relationship of AE to study medication, were recorded.
RESULTS: Fifty-two patients (35 male, 17 female; mean age 51, SD 8 years), received a total of 1259 infusions of Z(A1-PI). In study B, 14 subjects received a total of 142 infusions of P(A1-PI). All patients presented with alpha1-antitrypsin deficiency (mean A1-PI level at screening: 6.0 μM, SD 1.5 μM) and emphysema. Fourteen (100%) of P(A1-PI)- and 50 (96%) of Z(A1-PI)-treated subjects reported at least 1 treatment-emergent AE. In 1259 infusions of Z(A1-PI), there were 254 AEs [0.202 AEs per infusion]; 73 AEs occurred in 142 infusions of P(A1-PI) [0.514 per infusion]. Only 3 AEs in each group were considered related to study drug [Z(A1-PI) 0.003 related AEs per infusion; P(A1-PI) 0.021 related AEs per infusion]. The majority of AEs were either mild or moderate in severity. No treatment-related serious AEs were reported in either study.
CONCLUSION: Z(A1-PI) is safe and well-tolerated. There was no significant difference in the number and severity of AEs between the infusions of the two preparations.
CLINICAL IMPLICATIONS: Z(A1-PI) is suitable for augmentation therapy in patients with alpha1-antitrypsin deficiency.
DISCLOSURE: Friedrich Kueppers, Grant monies (from industry related sources) FK has received funding from the Arlene Meth Fund, Baxter, and ZLB Behring; Other This research was funded by ZLB Behring LLC.