A multicenter, open-label, phase III study of Abcertin in Gaucher disease

Abstract

Background: Gaucher disease (GD) is caused by a deficiency in the lysosomal enzyme glucocerebrosidase. Enzyme replacement therapy (ERT) is recommended for clinical improvement.

Methods: The efficacy and safety of a new imiglucerase, Abcertin, were assessed in 7 Egyptian patients with treatment-naïve type 1 GD. Each patient was administered a biweekly 60 U/kg dose of Abcertin for 6 months. The primary endpoint was the change in hemoglobin concentration. The secondary endpoints were changes from baseline in platelet counts, spleen and liver volumes, biomarker levels, skeletal parameters, and bone mineral density.

Results: The hemoglobin concentration increased by a mean of 1.96 ± 0.91 g/dL (range 1.11-2.80 g/dL) or 20.6% (P = .001). Statistically significant increases in the platelet count and decreases in the spleen volume and biomarker levels were also observed. There were no severe drug-related adverse events. One patient developed anti-imiglucerase antibodies without neutralizing activity.

Conclusion: Our study results demonstrate the efficacy and safety of Abcertin in patients with type 1 GD. This suggests that Abcertin can be an alternative ERT option for type 1 GD.

Figure 1

Efficacy of Abcertin for Gaucher disease in the per protocol population. Mean changes in hemoglobin concentrations (A), platelet counts (B), angiotensin-converting enzyme (ACE) levels (C), acid phosphatase (ACP) levels (D), chitotriosidase levels (E), chemokine ligand 18 (CCL-18) levels (F), and L-spine bone densitometry (BMD) (G). Baseline refers to data obtained during the screening visit. Data are expressed as means ± standard deviations.

Figure 2

Mean plasma glucocerebrosidase concentration–time profile in the per protocol population (n = 7) after a single intravenous infusion of Abcertin 60 U/kg (top: linear, bottom: semi-log). Error bars represent standard deviations.