A 53-week, open-label phase IIIb study of velaglucerase alfa in Chinese patients with type 1 Gaucher disease: Safety, efficacy, and pharmacokinetics

IF 1.8 4区医学 Q3 GENETICS & HEREDITY

Molecular Genetics and Metabolism Reports Pub Date : 2026-06-01 Epub Date: 2026-06-02 DOI:10.1016/j.ymgmr.2026.101324

Lihui Zhang , Xue-Qun Luo , Yongjun Fang , Fengkui Zhang , Yan Meng , Xiaoping Luo , Liansheng Zhang , Zhengqing Qiu , Xiao Ge , Jaco Botha , Jenny Zhan , Tianyou Wang

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引用次数: 0

Abstract

Background

Velaglucerase alfa is approved in China for treating type 1 Gaucher disease (GD1), but data on its use in Chinese pediatric and adult patients are limited. This study evaluated the safety, efficacy, and pharmacokinetics of velaglucerase alfa in Chinese patients with GD1.

Methods

This was a phase IIIb, multicenter, open-label, single-arm, 53-week study (NCT05529992). Patients received intravenous velaglucerase alfa (60 U/kg) every 2 weeks. The primary endpoint was the incidence of serious treatment-emergent adverse events (TEAEs). Secondary endpoints evaluated safety (incidence of TEAEs), efficacy (changes in hemoglobin concentrations, platelet counts, liver and spleen volumes [as percent of body weight, % BW]), and quality of life (QoL).

Results

Twenty patients were enrolled (treatment-naïve, n = 16; previously treated, n = 4). Four patients experienced 4 serious TEAEs requiring hospitalization; none were treatment-related. Overall, 19 of 20 patients experienced 104 TEAEs, of which 9 were treatment-related (8.7%; all mild). No TEAEs or deaths led to discontinuation of treatment. Mean (SD) improvements in hemoglobin concentrations (baseline, 10.5 g/dL [2.2]; change, +2.3 g/dL [1.3]; +25.5%), platelet counts (baseline, 59.9 × 10⁹/L [23.4]; change, +42.1 × 10⁹/L [27.7]; +82.3%), normalized spleen (baseline, 5.2% BW [2.6]; change, −3.1% BW [1.6]; −58.4%), and liver volume (baseline, 4.6% BW [1.3]; change, −1.1% BW [0.9]; −21.5%) were observed through Week 53. Nineteen of 20 (95.0%) patients reported improved QoL.

Conclusions

Velaglucerase alfa was well-tolerated in Chinese patients with GD1, with no new safety signals, and resulted in improved hemoglobin and platelet levels, as well as liver and spleen volumes.

Abstract Image

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一项为期53周的开放标签iii期研究：velaglucerase alfa在中国1型戈谢病患者中的安全性、有效性和药代动力学

velaglucerase alfa在中国被批准用于治疗1型戈谢病（GD1），但其在中国儿童和成人患者中的使用数据有限。本研究评估了velaglucerase alfa在中国GD1患者中的安全性、有效性和药代动力学。方法：这是一项IIIb期、多中心、开放标签、单组、53周的研究（NCT05529992）。患者每2周静脉注射维拉脲酶（60 U/kg）。主要终点是严重治疗不良事件（teae）的发生率。次要终点评估了安全性（teae的发生率）、有效性（血红蛋白浓度、血小板计数、肝脏和脾脏体积[占体重的百分比，体重百分比]的变化）和生活质量（QoL）。结果共纳入20例患者（treatment-naïve, n = 16；既往治疗，n = 4）。4例患者发生4次严重teae，需要住院治疗；没有一个是治疗相关的。总体而言，20例患者中有19例经历了104例teae，其中9例与治疗相关（8.7%，均为轻度）。没有teae或死亡导致停止治疗。到第53周，血红蛋白浓度（基线，10.5 g/dL[2.2]；变化，+2.3 g/dL[1.3]; +25.5%）、血小板计数（基线，59.9 × 109/L[23.4]；变化，+42.1 × 109/L[27.7]; +82.3%）、脾脏正常化（基线，5.2% BW[2.6]；变化，- 3.1% BW[1.6]; - 58.4%）和肝脏体积（基线，4.6% BW[1.3]；变化，- 1.1% BW[0.9]; - 21.5%）均有改善。20例患者中有19例（95.0%）报告生活质量改善。结论velaglucerase alfa在中国GD1患者中耐受性良好，无新的安全性信号，可改善血红蛋白和血小板水平，改善肝脏和脾脏体积。

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来源期刊

Molecular Genetics and Metabolism Reports Biochemistry, Genetics and Molecular Biology-Endocrinology

CiteScore

4.00

自引率

5.30%

发文量

105

审稿时长

33 days

期刊介绍： Molecular Genetics and Metabolism Reports is an open access journal that publishes molecular and metabolic reports describing investigations that use the tools of biochemistry and molecular biology for studies of normal and diseased states. In addition to original research articles, sequence reports, brief communication reports and letters to the editor are considered.