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Medicine (Baltimore). 2017 Nov;96(45):e8492. doi: 10.1097/MD.0000000000008492.

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

Author information

1
aDepartment of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea bPaediatric GIT Unit, Mansoura University Children's Hospital cAbou El Reesh Children's Hospital, Cairo University, Egypt dMedical Genetics Center, Asan Medical Center eAsan Institute for Life Sciences, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea fBiochemical Genetics Department, National Research Centre, Cairo, Egypt gISU ABXIS Co., Ltd, Seongnam, Korea.

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.

PMID:
29137040
PMCID:
PMC5690733
DOI:
10.1097/MD.0000000000008492
[Indexed for MEDLINE]
Free PMC Article

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