预约演示
更新于：2026-07-28
Pegzilarginase
更新于：2026-07-28
概要
基本信息
非在研机构- |
最高研发阶段批准上市 |
首次获批日期 欧盟 (2023-12-15), |
最高研发阶段(中国)- |
特殊审评突破性疗法 (美国)、快速通道 (美国)、加速批准 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、孤儿药 (澳大利亚) |
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结构/序列
Sequence Code 523960959
来源: *****
关联
10
项与 Pegzilarginase 相关的临床试验NL-OMON58219
Analysis of patients with arginase 1 deficiency treated with Loargys in standard clinical care enrolled in a European, non-interventional, multicentre registry - Analysis of standard clinical care treatment of ARG1 deficient patients enrolled in European multicentre registry
CTIS2024-510797-25-00
A Phase 3 Open-Label Study of Safety, Pharmacokinetics and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 months old with Arginase 1 Deficiency
NCT06582524
A Phase 3 Open-Label Study of Safety, Pharmacokinetics, and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency
100 项与 Pegzilarginase 相关的临床结果
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100 项与 Pegzilarginase 相关的转化医学
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100 项与 Pegzilarginase 相关的专利（医药）
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11
项与 Pegzilarginase 相关的文献（医药）2026-04-28Children-Basel
Pegzilarginase in Arginase 1 Deficiency: Clinical and Biochemical Effects of Treatment Initiation, Discontinuation and Re-Initiation.
Article
作者: Gigante, Silvia ; Balduzzi, Adriana ; Gasperini, Serena ; Marchetti, Vera ; Bolgè, Maria Valvassori ; Pretese, Roberta ; Crescitelli, Viola ; Faraguna, Martha Caterina ; Bonfanti, Cristina ; Sgroi, Giusi ; Sala, Stefania
BACKGROUND:
Arginase 1 deficiency (ARG1-D) is an ultra-rare urea cycle disorder characterized by hyperargininemia and progressive neurological impairment, including spasticity, loss of motor function, and reduced quality of life. Conventional management based on dietary protein restriction and ammonia scavengers rarely achieves adequate metabolic control or prevents neurological deterioration. Pegzilarginase, a recombinant human arginase 1 enzyme, is the first disease-modifying therapy for ARG1-D.
METHODS:
We report the first Italian real-world experience with pegzilarginase in three pediatric patients with genetically confirmed ARG1-D enrolled in the phase 3 PEACE trial. Clinical, biochemical, functional, nutritional and quality-of-life data were retrospectively collected over a long-term follow-up (2003-2025). Outcomes were evaluated across three phases: treatment initiation (Start), a 13-month treatment interruption due to trial closure (Stop), and therapy re-initiation through an early access program (Restart).
RESULTS:
Pegzilarginase rapidly normalized plasma arginine levels and was associated with improvements in motor function, spasticity, walking endurance, dietary protein tolerance, bone mineral density, and quality of life. During treatment interruption, all patients experienced biochemical worsening and clinical deterioration, including increased spasticity, reduced mobility, and emotional distress. Re-initiation of pegzilarginase restored metabolic control and led to progressive neurological and functional recovery, including partial reversal of long-standing motor deficits.
CONCLUSIONS:
This real-world experience supports pegzilarginase as a disease-modifying therapy for ARG1-D. Sustained normalization of plasma arginine, rather than subthreshold biochemical control, correlates with functional and neurological improvement and may partially reverse non-lesional metabolic brain injury. Early initiation of pegzilarginase, including in newborn-screened patients, may further modify the natural history of ARG1-D.
2026-03-31ANALYTICAL CHEMISTRY
Rapid Detection of
l
-Arginine Using a Fluorescent
Bacillus caldovelox
Arginase for Monitoring Arginase Deficiency
Article
作者: Leung, Yun-Chung ; Tam, Suet-Ying ; Chung, Sai-Fung ; Lee, Leo Man-Yuen ; Wong, Kwok-Yin ; So, Pui-Kin ; So, Paull Yik-Hing ; Wong, Wai-Ting
Patients with arginase deficiency require lifelong treatment with pegzilarginase and regular monitoring of blood l-arginine (l-Arg) levels. To address this need, we developed a site-directed, fluorescently labeled Bacillus caldovelox arginase, BCA(T16C)f, designed to directly and specifically detect l-Arg. BCA(T16C)f functions as a "turn-on" fluorescent biosensor, producing an increased fluorescence signal upon reacting with l-Arg. This is due to the labeling site being strategically positioned near the enzyme's active site, allowing it to be displaced into the external aqueous environment during interaction with l-Arg. The biosensor effectively quantified l-Arg levels ranging from 100 to 1000 μM by measuring the percentage change in fluorescence intensity within the first minute. When applied to detect 200 or 400 μM l-Arg in fetal bovine serum to mimic the condition of arginase deficiency, the results closely matched those obtained using mass spectrometry. This study presents a rapid, simple, and specific fluorescent biosensor, offering a practical solution for patients with arginase deficiency to monitor their l-Arg levels routinely.
2026-03-01JOURNAL OF INHERITED METABOLIC DISEASE
Clinical Characteristics of Arginase 1 Deficiency: Natural History Insights From International Clinical Trials
Article
作者: Enns, Gregory M. ; Karall, Daniela ; Sharma, Reena ; Ghosh, Arunabha ; Rudebeck, Mattias ; Shelkowitz, Emily ; Gorce, Magali ; Braverman, Nancy ; Zori, Roberto ; McNutt, Markey ; Chang, Richard
ABSTRACT:
Arginase 1 deficiency (ARG1‐D) is an ultra‐rare inherited metabolic disorder of the urea cycle, caused by partial or complete loss of arginase 1 function, characterised by hyperargininaemia and a distinct, progressive neurological phenotype. The clinical development programme of pegzilarginase, a recombinant human ARG1 enzyme therapy, provides an opportunity to study the largest ARG1‐D cohort to date. The analysis included 48 paediatric and adult subjects (≥ 2 years) enrolled in the pegzilarginase trials. Baseline data collected before treatment included demographics, genotypes, red blood cell arginase activity, biochemical measures, age of symptom onset, neuromotor and neurological characteristics, growth indicators, quality of life, and use of treatments and assistive devices. The mean (SD) age of onset was 2.2 (3.6) years, which preceded diagnosis at 3.7 (5.0) years. Clinical features included motor impairment (48/48, 100%), spasticity (33/48, 69%), cognitive deficits (31/48, 65%), intellectual disability (23/36, 64%), speech and language deficits (26/48, 54%), and seizures (18/48, 38%), with symptom‐onset data consistent with a progressive phenotype. Median GMFCS Level II indicated moderate mobility limitation; two‐thirds scored < 69 on FSIQ, and mean PedsQL total proxy scores were around 20% lower than typically developing peers. All subjects followed a protein‐restricted diet, and 90% used ammonia scavengers. ARG1‐D presents with a heterogeneous array of progressive and debilitating neurologic symptoms. These findings reflect the progressive impact of the disease and offer insights into its burden and natural history based on a large cohort, assessed using standardised neuromotor, cognitive, and quality‐of‐life instruments across international sites.
100 项与 Pegzilarginase 相关的药物交易
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研发状态
批准上市
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 消化系统疾病 | 加拿大 | 2026-05-29 | |
| 代谢性疾病 | 加拿大 | 2026-05-29 | |
| 高精氨酸血症 | 欧盟 | 2023-12-15 | |
| 高精氨酸血症 | 冰岛 | 2023-12-15 | |
| 高精氨酸血症 | 列支敦士登 | 2023-12-15 | |
| 高精氨酸血症 | 挪威 | 2023-12-15 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 广泛期小细胞肺癌 | 临床2期 | 美国 | 2017-12-21 | |
| 难治性急性髓细胞白血病 | 临床2期 | 美国 | 2016-08-01 | |
| 难治性急性髓细胞白血病 | 临床2期 | 加拿大 | 2016-08-01 | |
| 难治性骨髓增生异常综合征 | 临床2期 | 美国 | 2016-08-01 | |
| 难治性骨髓增生异常综合征 | 临床2期 | 加拿大 | 2016-08-01 | |
| 复发性急性髓细胞白血病 | 临床2期 | 美国 | 2016-08-01 | |
| 复发性急性髓细胞白血病 | 临床2期 | 加拿大 | 2016-08-01 | |
| 晚期恶性实体瘤 | 临床2期 | 美国 | 2015-10-01 | |
| 葡萄膜黑色素瘤 | 临床2期 | 美国 | 2015-10-01 |
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临床结果
临床结果
适应症
分期
评价
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临床3期 | 3 | 淵窪鹹鏇築繭觸鑰願衊(網顧選鏇製鑰夢簾鑰窪) = 糧簾艱遞鹹窪鬱醖網夢 鑰夢衊鑰顧積襯齋糧顧 (願壓顧積壓選築鹽繭簾, 81.26) 更多 | - | 2026-04-30 | |||
临床3期 | 3 | 廠範觸簾夢夢壓衊餘構(膚網艱廠糧廠遞築齋襯) = clinically meaningful reduction 構鬱積餘範積襯廠鏇網 (選膚鏇顧選蓋築繭廠遞 ) 更多 | 积极 | 2026-02-02 | |||
临床3期 | 32 | (Pegzilarginase) | 積醖鹹廠製網膚網鹹範(遞繭範糧選襯繭艱顧餘) = 蓋鑰鹽壓鹽鬱製簾醖淵 蓋選蓋醖鹹網繭襯淵觸 (鬱廠襯淵膚顧繭廠遞鬱, 1.635) 更多 | - | 2024-11-19 | ||
Placebo (Placebo) | 積醖鹹廠製網膚網鹹範(遞繭範糧選襯繭艱顧餘) = 觸窪鑰艱簾遞醖網憲膚 蓋選蓋醖鹹網繭襯淵觸 (鬱廠襯淵膚顧繭廠遞鬱, 1.371) 更多 | ||||||
临床3期 | 高精氨酸血症 plasma arginine (pArg) | 32 | 網壓餘遞蓋簾鹽簾遞糧(鬱顧鹽餘糧窪窪壓襯鬱) = 鹹鹹遞艱構鹽獵淵夢築 選鹽築鹽廠餘鹹範築鏇 (艱憲鹽襯衊餘淵鬱鬱窪 ) | 积极 | 2022-08-30 | ||
临床1/2期 | 16 | 顧餘夢齋築夢憲鑰糧憲(襯簾網廠願築壓淵範廠) = five patients were all observed in 101A 憲窪衊願觸顧淵憲糧築 (範糧窪糧艱淵繭繭願鑰 ) 更多 | 积极 | 2021-07-01 | |||
临床1/2期 | 高精氨酸血症 plasma arginine | 16 | 觸繭繭構夢網壓襯艱夢(觸齋憲襯醖範衊遞餘選) = 鏇壓鬱鹽蓋鑰築築網築 遞鬱築顧窪憲範選夢齋 (齋齋構獵鏇膚構繭糧願 ) | - | 2019-08-25 | ||
临床1期 | - | 醖憲艱糧繭憲壓憲積範(繭齋繭襯齋廠窪網醖簾) = increased levels of granzyme A, IFN-g and TNF-a 餘膚膚糧衊繭糧遞獵鹹 (窪遞蓋築觸願繭膚築構 ) 更多 | 积极 | 2018-11-06 | |||
aOX40 mAb | |||||||
临床1期 | 40 | 齋窪淵構構觸獵遞醖衊(齋餘獵廠觸網齋鏇願膚) = 0.33 mg/kg once weekly 繭選觸遞製膚蓋窪衊餘 (繭淵艱淵衊齋鹽鹹範觸 ) 更多 | 积极 | 2018-07-01 | |||
临床1期 | 21 | 鹽憲網壓壓憲夢醖鑰廠(鹽蓋憲糧蓋鏇鹹顧網觸) = 憲糧鏇製糧艱淵廠衊齋 鬱廠積鹹積蓋蓋觸襯鬱 (蓋窪繭艱齋蓋糧鏇襯齋 ) | 积极 | 2018-06-04 |
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