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.

Lysosomes as a therapeutic target

“…For example, in Pompe disease, the level of expression of mannose receptors on skeletal muscle cells is low, necessitating high doses of erT to achieve a therapeutic effect 202 . Numerous developments are being studied to address such limitations, with a focus on enzyme modifications that enable better access of enzymes to their receptors and on nanomaterials that enable safe and efficient delivery of enzymes via intra-cerebroventricular/intrathecal administration 10,46,200,203 .…”

The road to biosimilars in rare diseases ‐ ongoing lessons from Gaucher disease

“…Abcertin was initially approved in South Korea in 2012 via a national pathway that permitted the submission of phase 3 data after approval, and outside of approval pathways for biosimilars and other orphan drugs. It was approved based on the results of three clinical studies: a 5‐day, double‐blind, placebo‐controlled Phase 1 dose‐escalation study conducted in 24 healthy volunteers (NCT01881633); a 24‐week prospective, Phase 2, open‐label switch‐over study in one adult and four children/adolescents with confirmed GD type 1 previously treated with Cerezyme; and a post‐approval, 24‐week, Phase 3 trial in 7 treatment‐naïve children with GD type 1 . Studies providing a direct comparison to the RP, Cerezyme, and physicochemical, immunological, or structural data have not been presented.…”

“…Authors of the Phase 2 study initially referred to Abcertin as a “biosimilar” for Cerezyme . Although they have since corrected their claim in an erratum to their initial publication, confusion could persist regarding the precise nature of Abcertin .…”

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

“…In the article, “A multicenter, open-label, phase III study of Abcertin in Gaucher disease”, [ 1 ] which appears in Volume 96, Issue 45 of Medicine , Table 1 contained incorrect information and the correct table appears below.…”