ELK GROVE VILLAGE, Ill.

伊利诺伊州埃尔克格罗夫村

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，

March 9, 2026

2026年3月9日

/PRNewswire/ -- Orsini, a leader in rare disease pharmacy solutions, is pleased to announce it was selected by Immedica Pharma US Inc. as the exclusive specialty pharmacy and hub services partner for LOARGYS

/PRNewswire/ -- Orsini，罕见病药房解决方案的领导者，欣然宣布被Immedica Pharma美国公司选为LOARGYS的独家专业药房和中心服务合作伙伴。

®

®

(pegzilarginase-nbln), an arginine specific enzyme indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction.

(pegzilarginase-nbln)，一种精氨酸特异性酶，用于治疗2岁及以上患有精氨酸酶1缺乏症（ARG1-D）的成人和儿童患者的高精氨酸血症，配合饮食蛋白质限制使用。

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LOARGYS is the first and only therapy proven to lower arginine in patients 2 years of age and older living with ARG1-D.

LOARGYS 是首个也是唯一一个被证明可以降低 2 岁及以上 ARG1-D 患者精氨酸水平的疗法。

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See Important Safety Information below and full prescribing information for complete safety information.

请参阅下面的重要安全信息以及完整的处方信息以获取完整的安全信息。

ARG1-D is an ultra-rare, debilitating, and progressive metabolic disease resulting in persistent elevation of plasma arginine, also known as hyperargininemia.

ARG1-D 是一种超罕见、致残且进展性的代谢疾病，会导致血浆精氨酸持续升高，也称为高精氨酸血症。

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ARG1-D affects an estimated 250 people living in the U.S. and current standard of care relies primarily on symptom management, including dietary protein restriction, arginine-free amino acid supplementation and nitrogen scavenging agents if necessary.

ARG1-D影响着美国大约250人，目前的护理标准主要依赖于症状管理，包括限制饮食蛋白质、补充无精氨酸氨基酸，以及在必要时使用氮清除剂。

Through this partnership, Orsini will play a key role in helping patients, caregivers and healthcare providers navigate medication access, including insurance coverage and financial assistance. In addition to integrated specialty pharmacy and hub services, Orsini will provide 3PL services for Immedica, as .

通过该合作，奥尔西尼将帮助患者、护理人员和医疗服务提供者处理药物获取问题（包括保险覆盖和经济援助）方面发挥关键作用。除了综合的专业药房和中心服务外，奥尔西尼还将为Immedica提供第三方物流服务。

previously announced

之前已宣布

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。

'We're thrilled to support Immedica with comprehensive commercialization services for the launch of LOARGYS,' Brandon Tom, Orsini's CEO, said. 'By bringing together our 3PL capabilities, specialty pharmacy services and patient services hub, we're fully equipped to ensure patients with ARG1-D have streamlined access to this first-of-its-kind therapy.

“我们很高兴为Immedica提供全面的商业化服务，以支持LOARGYS的上市，”Orsini的首席执行官布兰登·汤姆表示。“通过整合我们的第三方物流能力、专业药房服务以及患者服务中心，我们完全有能力确保ARG1-D患者能够顺畅获取这种首创疗法。”

Our team is proud to stand alongside Immedica as we work to provide meaningful support for the rare disease community.'.

我们的团队很自豪能与Immedica并肩合作，为罕见病社区提供有意义的支持。

References

参考文献

LOARGYS (pegzilarginase-nbln)

洛阿尔吉斯（聚乙二醇化精氨酸酶-nbln）

US Prescribing Information

美国处方信息

. Immedica Pharma, 2026.

Immedica Pharma，2026年。

Russo RS, et al. Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled multi-centre trial.

Russo RS 等。Pegzilarginase 在精氨酸酶 1 缺乏症中的疗效与安全性（PEACE）：一项 III 期、随机、双盲、安慰剂对照的多中心试验。

EClinicalMedicine

EClinicalMedicine

. 2024;68:102405.

. 2024;68:102405.

Burlina A, et al. Arginase 1 deficiency: a treatable form of spastic paraplegia.

布尔利纳 A，等。精氨酸酶1缺乏症：一种可治疗的痉挛性截瘫形式。

Neurol Sci.

神经科学杂志。

2025;46:4219–4228.

2025；46：4219–4228。

IMPORTANT SAFETY INFORMATION

重要安全信息

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

警告：包括过敏反应在内的超敏反应

See full prescribing information for complete boxed warning

请参阅完整的处方信息以获取完整的加框警告。

Initiate LOARGYS in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue LOARGYS, and immediately initiate appropriate medical treatment, including use of epinephrine.

在医疗机构中启动LOARGYS，并提供适当的医疗监测和支持措施，包括获取心肺复苏设备。如果发生严重超敏反应（如过敏性休克），应停止使用LOARGYS，并立即开始适当的医疗救治，包括使用肾上腺素。

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WARNINGS AND PRECAUTIONS

警告和注意事项

Hypersensitivity Reactions Including Anaphylaxis

包括过敏反应在内的超敏反应

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：

Life-threatening hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including LOARGYS. Hypersensitivity reactions that were mild to moderate in severity occurred in 13% (6/48) of LOARGYS-treated subjects in clinical trials. Hypersensitivity reactions have included facial swelling, rash, flushing and dyspnea.

接受酶替代疗法（包括LOARGYS）治疗的患者中，已发生危及生命的超敏反应，包括过敏性反应。在临床试验中，13%（6/48）的使用LOARGYS的受试者发生了轻度至中度的超敏反应。超敏反应的症状包括面部肿胀、皮疹、潮红和呼吸困难。

The reactions generally occurred with the first few doses but may occur later in treatment..

通常在头几次服药时就会出现反应，但也可能在治疗后期出现。

Administration of LOARGYS should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis in a healthcare setting with appropriate medical monitoring and support measures. Premedication with an antihistamine and/or corticosteroid should be considered in patients who previously have developed a hypersensitivity reaction.

LOARGYS的使用应由在过敏反应（包括过敏性休克）管理方面有经验的医疗保健提供者监督，并在具备适当医疗监测和支持措施的医疗环境中进行。对于既往曾发生过敏反应的患者，应考虑使用抗组胺药和/或皮质类固醇进行预处理。

If a .

如果一个。

severe

严重的

hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue LOARGYS and immediately initiate appropriate medical treatment, including use of epinephrine. Consider the risks and benefits of re-administering LOARGYS in patients who have experienced a severe hypersensitivity reaction. Caution should be exercised upon rechallenge.

如果发生超敏反应（例如，过敏性休克），请停止使用LOARGYS并立即开始适当的医疗处理，包括使用肾上腺素。对于曾经发生过严重超敏反应的患者，重新给予LOARGYS时应权衡风险和益处。再次挑战时应谨慎。

Inform patients of the symptoms of life-threatening hypersensitivity reactions and to seek immediate medical attention should symptoms occur. If a mild or moderate reaction occurs, consider treatment with antihistamines and/or corticosteroids..

告知患者可能危及生命的超敏反应的症状，并在出现症状时立即寻求医疗帮助。如果发生轻度或中度反应，考虑使用抗组胺药和/或皮质类固醇进行治疗。

ADVERSE REACTIONS

不良反应

The most common adverse reactions are vomiting, pyrexia, infusion associated reactions and constipation.

最常见的不良反应是呕吐、发热、输液相关反应和便秘。

USE IN SPECIFIC POPULATIONS

在特定人群中使用

Pregnancy

怀孕

: There are no available data on LOARGYS use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes.

：目前尚无关于LOARGYS在孕妇中使用的可用数据，无法评估其与药物相关的重大出生缺陷、流产或其他不良母体或胎儿结局的风险。

Lactation

泌乳

: There is no data on the presence of LOARGYS in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for LOARGYS and any potential adverse effects on the breast-fed infant from LOARGYS or from the underlying maternal condition..

：目前尚无关于LOARGYS在人乳或动物乳中是否存在、对哺乳婴儿的影响或对乳汁分泌影响的数据。在考虑母亲对LOARGYS的临床需求时，应权衡母乳喂养对婴儿发育和健康的益处，以及LOARGYS或母体基础状况可能对哺乳婴儿造成的任何潜在不良影响。

Pediatric

儿科的

: The safety and effectiveness of LOARGYS have been established for the reduction of plasma arginine in pediatric patients 2 years and older with ARG-1 D, in conjunction with dietary protein restriction. The safety and effectiveness of LOARGYS have not been established for the reduction of plasma arginine in pediatric patients aged less than 2 years with ARG-1 D..

LOARGYS 对于降低 2 岁及以上患有 ARG-1 D 的儿科患者的血浆精氨酸水平的安全性和有效性已得到证实，需结合饮食蛋白质限制。对于年龄小于 2 岁的 ARG-1 D 儿科患者，LOARGYS 降低血浆精氨酸水平的安全性和有效性尚未得到证实。

Geriatric

老年医学的

: Clinical studies of LOARGYS did not include subjects 65 years of age and older to determine whether they respond differently from younger adult subjects.

：LOARGYS的临床研究未包括65岁及以上的受试者，以确定他们是否与年轻成年受试者有不同的反应。

INDICATION

适应症

LOARGYS is an arginine specific enzyme indicated for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency (ARG1-D), in conjunction with dietary protein restriction.

LOARGYS 是一种精氨酸特异性酶，适用于治疗 2 岁及以上患有精氨酸酶 1 缺乏症 (ARG1-D) 的成人和儿童患者的高精氨酸血症，同时配合饮食蛋白质限制。

This indication is approved under accelerated approval based on reduction of plasma arginine. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

该适应症是基于血浆精氨酸减少而获得加速批准的。此适应症的持续批准可能取决于在确证性试验中对临床益处的验证和描述。

About Orsini

关于奥尔西尼

Providing patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services hub, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies.

自1987年以来，奥尔西尼一直为患者提供全面且富有同情心的护理，是罕见病和基因治疗领域的领导者。奥尔西尼与生物制药创新者、医疗保健提供者和支付方合作，支持患者及其家人获取革命性的罕见病治疗方法。通过整合的罕见病药房解决方案，包括药品分销、患者服务中心、临床管理以及便捷的居家输液服务，奥尔西尼简化了患者获得先进治疗的方式。

Orsini's high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that .

奥西尼的高接触护理模式以经验丰富且训练有素的治疗护理团队为中心，提供个性化的患者护理以确保。

No Patient is Left Behind™

不落下任何患者™

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Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, visit

奥西尼持有医疗卫生认证委员会 (ACHC)、联合委员会、URAC 和 NABP 的认证。奥西尼荣获 URAC 的罕见病药房卓越中心称号以及 ACHC 的罕见病和孤儿药杰出认证。欲了解更多信息，请访问

www.orsini.com.

www.orsini.com.

SOURCE Orsini

源奥尔西尼

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