SKYTROFA

SKYTROFA

®

®

(lonapegsomatropin) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of paediatric growth hormone deficiency.

(lonapegsomatropin) 已经由治疗用品管理局 (TGA) 注册，用于治疗儿童生长激素缺乏症。

[

[

1]

1]

SINGAPORE

新加坡

,

，

May 29, 2025

2025年5月29日

/PRNewswire/ --

/美通社/ --

Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce the Australian registration of SKYTROFA® (lonapegsomatropin)

独立生物制药公司Specialised Therapeutics（ST）欣然宣布SKYTROFA®（lonapegsomatropin）在澳大利亚注册。

as a new once-weekly injectable therapy for paediatric growth hormone deficiency (GHD).

作为一种新的每周一次的儿科生长激素缺乏症（GHD）注射疗法。

[2]

[2]

The Therapeutic Goods Administration (TGA) has approved SKYTROFA as a treatment for

治疗用品管理局 (TGA) 已批准 SKYTROFA 作为治疗

'g

'g

rowth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion'

由于内源性生长激素分泌不足，导致3岁至18岁儿童和青少年的生长发育迟缓。

.

。

[1,2]

[1,2]

GHD is a rare disease affecting around 2-3/10,000 Australians, with approximately 2,000 children thought to be living with the condition.

GHD 是一种罕见病，大约影响着两万到三万澳大利亚人中的 2-3 人，估计约有 2,000 名儿童患有此病。

[3]

[3]

It occurs when the pituitary gland, located at the base of the brain, does not produce an adequate level of growth hormone, which is essential for promoting healthy growth in children.

当位于大脑底部的垂体不产生足够水平的生长激素时，就会发生这种情况，而这种激素对促进儿童健康成长至关重要。

[3]

[3]

In children, GHD is typically characterised by slow height growth, resulting in short stature, and may also include delayed puberty, impaired hair growth and headaches.

在儿童中，生长激素缺乏症通常表现为身高增长缓慢，导致身材矮小，还可能包括青春期延迟、头发生长受损和头痛。

[3]

[3]

The goal of treatment for GHD is to restore normal levels of growth hormone in the body, reduce physical symptoms, enhance metabolic health and improve quality of life.

生长激素缺乏症治疗的目标是恢复体内正常的生长激素水平，减轻身体症状，改善代谢健康并提高生活质量。

[3]

[3]

ST Chief Executive Officer, Mr

意法半导体首席执行官，先生

Carlo Montagner

卡洛·蒙塔尼奥

, said the TGA approval of SKYTROFA demonstrated the company's ongoing mission to support patients with rare diseases in

，TGA批准SKYTROFA表明了公司持续支持罕见病患者的使命在

Australia

澳大利亚

and the

和

Asia-Pacific

亚太地区

region.

地区。

'We are delighted to have secured TGA registration of SKYTROFA for eligible Australian children with growth hormone deficiency. Beyond short stature, children and adolescents with the condition may experience considerable physical and psychosocial impacts on their daily life, such as poor concentration, decreased strength or muscle development, fatigue, and reduced quality of life..

“我们很高兴为符合条件的澳大利亚生长激素缺乏症儿童获得了SKYTROFA的TGA注册。除了身材矮小之外，患有该疾病的儿童和青少年可能会在日常生活中经历显著的身体和心理社会影响，例如注意力不集中、体力或肌肉发育下降、疲劳以及生活质量降低。”

[4]

[4]

'This announcement also represents a significant milestone for Specialised Therapeutics, marking the second endocrinology therapy we have successfully registered in

“这一公告也代表了特种治疗公司的一个重要里程碑，标志着我们成功注册的第二个内分泌治疗药物。

Australia

澳大利亚

, following the approval of YORVIPATH

，继YORVIPATH获批后

®

®

(palopegteriparatide) earlier this year,' said Mr Montagner.

（帕洛培格特立帕肽）在今年早些时候，蒙塔涅先生说。

SKYTROFA is being registered in

SKYTROFA 正在注册中

Australia

澳大利亚

by ST, under an exclusive distribution agreement with biopharmaceutical company Ascendis Pharma A/S that covers

由ST公司根据与生物制药公司Ascendis Pharma A/S签订的独家分销协议提供，该协议涵盖

Australia

澳大利亚

,

，

New Zealand

新西兰

,

，

Singapore

新加坡

,

，

Malaysia

马来西亚

,

，

Brunei

文莱

,

，

Thailand

泰国

, and

，以及

Vietnam

越南

. The Australian registration of SKYTROFA follows approvals issued to Ascendis Pharma by the United States Food and Drug Administration (US FDA)

澳大利亚对SKYTROFA的注册紧随美国食品和药物管理局（US FDA）对Ascendis Pharma的批准之后。

[5]

[5]

in

在

August 2021

2021年8月

and the European Medicines Agency (EMA)

和欧洲药品管理局 (EMA)

[6]

[6]

in

在

January 2022

2022年1月

.

。

Developed using Ascendis Pharma's proprietary TransCon

采用Ascendis Pharma专有的TransCon技术开发

TM

商标

platform, SKYTROFA is delivered as a subcutaneous injection, available in various strengths.

平台，SKYTROFA 以皮下注射的形式提供，有多种强度可供选择。

[2]

[2]

The starting dose depends on the patient's body weight and is then adjusted individually by the treating endocrinologist based on the patient's response to treatment.

起始剂量取决于患者的体重，然后由治疗的内分泌科医生根据患者对治疗的反应进行个体化调整。

[2]

[2]

Ascendis Pharma's innovative TransCon

Ascendis Pharma创新的TransCon

TM

商标

technology platform is designed to create new therapies with the potential to optimise therapeutic effect, including improving treatment efficacy, safety and dosing frequency.

技术平台旨在创造具有优化治疗效果潜力的新疗法，包括提高治疗效力、安全性和给药频率。

[7]

[7]

TransCon

TransCon

TM

商标

molecules have three components: an unmodified parent drug, an inert TransCon™ carrier that protects it, and a TransCon

分子有三个组成部分：未修饰的母体药物、保护它的惰性TransCon™载体，以及TransCon

TM

商标

linker that temporarily binds the two.

暂时连接两者的链接器。

[7]

[7]

SKYTROFA is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin.

SKYTROFA 是一种每周给药一次的生长激素前药，旨在提供持续释放活性、未修饰的生长激素。

[6]

[6]

This allows the medicine to slowly release unmodified growth hormone into the body over the course of one week, removing the need for daily injections.

这使得药物能够在一周的时间内缓慢释放未经修饰的生长激素到体内，消除了每天注射的需要。

SKYTROFA consists of three components: the parent drug somatropin, an inert methyloxypolyethlene glycol carrier (mPEG), and a proprietary TransCon

SKYTROFA由三个组成部分：母药生长激素、惰性的甲氧基聚乙二醇载体（mPEG）和特有的TransCon技术。

TM

商标

linker that transiently binds the other two elements.

链接器，暂时结合另外两个元素。

[8,9]

[8,9]

While bound, the carrier inactivates the somatropin and shields it from renal excretion and receptor-mediated clearance.

在结合状态下，载体使生长激素失活，并保护其免受肾排泄和受体介导的清除。

[10]

[10]

Following injection, autocleavage of the linker occurs under physiologic conditions, and SKYTROFA releases fully active, unmodified somatropin in a predictable manner.

注射后，连接子在生理条件下发生自切割，SKYTROFA以可预测的方式释放完全活性、未修饰的生长激素。

[10]

[10]

The TGA registration of SKYTROFA was based on the results of Ascendis Pharma's three pivotal Phase 3 clinical trials,

SKYTROFA 的 TGA 注册是基于 Ascendis Pharma 的三项关键 III 期临床试验结果，

heiGHt

高度

,

，

fliGHt

航班

and

和

enliGHten

启发

, which collectively treated more than 300 paediatric patients diagnosed with GHD, including from

，共计治疗了300多名确诊为GHD的儿科患者，其中包括来自

Australia

澳大利亚

.

。

[2,8,11,12]

[2,8,11,12]

SKYTROFA was generally well-tolerated across all three clinical trials. The most commonly reported adverse events include viral infections, fever, cough, nausea and vomiting, haemorrhage, diarrhoea, abdominal pain, arthralgia, arthritis, and increased blood phosphate levels.

SKYTROFA在所有三项临床试验中通常耐受性良好。最常见的不良事件包括病毒感染、发热、咳嗽、恶心和呕吐、出血、腹泻、腹痛、关节痛、关节炎以及血磷水平升高。

PBS Information:

PBS 信息：

SKYTROFA is not listed on the Pharmaceutical Benefits Scheme (PBS).

SKYTROFA 未列入药品福利计划 (PBS)。

About Specialised Therapeutics

关于专业治疗公司

Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in

成立于2007年，Specialised Therapeutics是该地区最大的独立专业制药公司，为患者提供新的治疗方法和技术。

Australia

澳大利亚

,

，

New Zealand

新西兰

and across

并且横跨

Southeast Asia

东南亚

. Headquartered in

总部位于

Singapore

新加坡

, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions.

，ST与全球制药、生物技术和诊断公司合作，为受各种疾病影响的患者带来新的医疗机会。ST在应对不同地区的复杂监管、报销和商业化环境方面，建立了良好的成功记录。

The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA)..

ST的使命是提供在未满足需求的领域中的专业治疗。公司广泛的治疗组合目前包括肿瘤学、血液学、中枢神经系统、神经病学、内分泌学、眼科学和支持性护理中的新药，尽管并不局限于这些领域。ST是世界孤儿药联盟（WODA）的成员。

Additional information can be found at

更多信息可以在

www.stbiopharma.com

www.stbiopharma.com

About Ascendis Pharma

关于Ascendis制药公司

Ascendis Pharma is a global biopharmaceutical company focused on applying its innovative TransCon

Ascendis Pharma是一家全球生物制药公司，专注于应用其创新的TransCon

TM

商标

technology platform to make a meaningful difference for patients. Guided by core values of Patients, Science, and Passion, and following its algorithm for product innovation, Ascendis Pharma applies TransCon

技术平台，为患者带来有意义的改变。Ascendis Pharma以患者、科学和热情为核心价值观，并遵循其产品创新算法，应用TransCon技术。

TM

商标

to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in

开发具有同类最佳潜力的新疗法，以满足未被满足的医疗需求。Ascendis总部位于

Copenhagen, Denmark

丹麦哥本哈根

and has additional facilities in

并且在以下地方有额外的设施

Europe

欧洲

and

和

the United States

美国

. Please visit:

请访问：

ascendispharma.com

ascendispharma.com

to learn more.

了解更多。

About Paediatric Growth Hormone Deficiency

关于小儿生长激素缺乏症

Paediatric growth hormone deficiency (GHD) is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Physiological levels of growth hormone are required for overall endocrine health and development of healthy bone, muscle, and adipose tissue. Children with GHD are characterised by short stature and may also experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life.

小儿生长激素缺乏症（GHD）是一种严重的罕见病，由脑垂体未能产生足够的生长激素引起。生理水平的生长激素对于整体内分泌健康以及健康骨骼、肌肉和脂肪组织的发育至关重要。患有GHD的儿童通常表现为身材矮小，还可能出现代谢异常、心理社会挑战以及整体生活质量较差。

For decades, the standard of care for GHD has been a daily subcutaneous injection of human growth hormone (hGH) to improve growth and overall endocrine health..

几十年来，生长激素缺乏症的标准治疗方案是每天皮下注射人生长激素（hGH），以促进生长和整体内分泌健康。

About SKYTROFA (lonapegsomatropin)

关于SKYTROFA（隆培促生长素）

Once-weekly SKYTROFA is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous growth hormone.

每周一次的SKYTROFA是生长激素的一种前药，旨在提供未修饰生长激素的持续释放。从隆培生长激素中释放的未修饰、未结合的生长激素具有与内源性生长激素相同的191个氨基酸序列和大小。

[8]

[8]

SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve children with growth hormone deficiency and those switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly..

SKYTROFA单剂量预填充药筒有九种剂量规格，提供了便捷的剂量灵活性。该药筒专为配合SKYTROFA自动注射器使用而设计，可在室温下存放长达六个月。对于未曾接受过治疗的生长激素缺乏症儿童以及从每日使用生长激素（somatropin）转为每周用药的患者，推荐剂量为0.24毫克/公斤体重，每周一次。

[2]

[2]

The dose may be adjusted based on the child's weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).

剂量可以根据儿童的体重和胰岛素样生长因子-1标准差评分（IGF-1 SDS）进行调整。

[2]

[2]

SKYTROFA was studied in over 300 children with GHD across the Phase 3 program, which consisted of the

SKYTROFA 在第三阶段项目中对 300 多名 GHD 儿童进行了研究，该项目包括

HeiGHt

高度

Trial

试用

[8]

[8]

(for treatment-naïve patients), the

（针对初治患者），

FliGHt

航班

Trial

试用版

[11]

[11]

(for treatment-experienced patients), and the

（针对有治疗经验的患者），以及

EnliGHten

启蒙

Trial

试用

[12]

[12]

(a long-term extension trial). Patients who completed the

（一项长期扩展试验）。完成的患者

HeiGHt

高度

or

或

FliGHt

航班

Trials were able to continue in

审判能够继续进行

EnliGHten

启蒙

, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing open-label portion of the global Phase 3

，其中一些患者接受lonapegsomatropin治疗已超过四年。Ascendis Pharma还正在进行全球3期临床试验的开放标签部分。

ForesiGHt

远见

Trial of SKYTROFA in adults with GHD.

成人GHD的SKYTROFA试验。

Ascendis

阿森迪斯

®

®

, TransCon

，TransCon

TM

商标

and SKYTROFA

和SKYTROFA

®

®

are trademarks owned by the Ascendis Pharma group (NASDAQ:

是 Ascendis Pharma 集团（纳斯达克：

ASND

ASND

).

）。

About TransCon

关于TransCon

TM

商标

Technologies

技术

TransCon refers to 'transient conjugation'. Ascendis Pharma's innovative technology platform to create new therapies designed to potentially optimise therapeutic effect, including improving efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert TransCon carrier that protects it, and a TransCon linker that temporarily binds the two.

TransCon指“暂时连接”。Ascendis Pharma的创新技术平台，用于创造新的疗法，旨在优化治疗效果，包括提高疗效、安全性和给药频率。TransCon分子有三个组成部分：未修饰的母体药物、保护它的惰性TransCon载体，以及临时连接两者的TransCon接头。

When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g. pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained.

结合后，载体失活并保护母体药物不被清除。当注入体内时，生理条件（如 pH 值和温度）会以可预测的方式启动未修饰的活性母体药物的释放。由于母体药物未经修饰，预计其原始作用机制将得以保持。

TransCon technology can be applied to a broad variety of therapeutics, such as antibodies, antibody fragments, proteins, peptides, or small molecules, and can be designed for systemic or local administration..

TransCon技术可应用于多种治疗药物，如抗体、抗体片段、蛋白质、肽或小分子，并且可以设计用于全身或局部给药。

References:

参考文献：

[1] Therapeutic Goods Administration. Australian Register of Therapeutic Goods (ARTG). Available at:

[1] 治疗用品管理局。澳大利亚治疗用品注册（ARTG）。可于以下网址获取：

https://www.tga.gov.au/resources/artg?keywords=skytrofa

https://www.tga.gov.au/resources/artg?keywords=skytrofa

. [Accessed May 2025].

。[访问时间：2025年5月]。

[2] SKYTROFA (lonapegsomatropin) Product Information, Australia.

[2] SKYTROFA（lonapegsomatropin）产品信息，澳大利亚。

[3] Australian Pituitary Foundation. Factsheet: A Guide To Growth Hormone Deficiency, 2023. Available at:

[3] 澳大利亚垂体基金会。事实说明书：生长激素缺乏症指南，2023年。可访问：

https://pituitary.asn.au/wp-content/uploads/2023/09/APF-Growth-Hormone-Deficiency-Fact-Sheet-2023_Digital-Final.pdf

https://pituitary.asn.au/wp-content/uploads/2023/09/APF-生长激素缺乏症-事实表-2023_数字版最终.pdf

[Accessed May 2025].

[访问于2025年5月]。

[4] Brod M et al.

[4] 布罗德 M 等。

Qual Life Res.

生活质量研究。

2017 Jul;26(7):1673-1686.

2017年7月；26（7）：1673-1686。

[5] US Food and Drug Administration (FDA). Drug Approval Package: SKYTROFA. Available at:

[5] 美国食品药品监督管理局 (FDA)。药物批准文件：SKYTROFA。可于以下网址获取：

https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761177Orig1s000TOC.cfm

https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761177Orig1s000TOC.cfm

[Accessed May 2025].

[访问于2025年5月]。

[6] European Medicines Agency (EMA). Skytrofa (previously Lonapegsomatropin Ascendis Pharma), January 2022. Available at:

[6] 欧洲药品管理局 (EMA)。Skytrofa（之前称为Lonapegsomatropin Ascendis Pharma），2022年1月。可访问：

https://www.ema.europa.eu/en/medicines/human/EPAR/skytrofa

https://www.ema.europa.eu/en/medicines/human/EPAR/skytrofa

[Accessed May 2025].

[访问时间：2025年5月]。

[7] Ascendis Pharma A/S. TransCon: A powerful technology platform central to our innovative approach. Available at:

[7] Ascendis制药公司。TransCon：我们创新方法核心的强大技术平台。可用地址：

https://ascendispharma.com/technology/

https://ascendispharma.com/technology/

[Accessed May 2025].

[访问于2025年5月]。

[8] Thornton PS, et al.

[8] 桑顿 PS，等。

J Clin Endocrinol Metab.

临床内分泌与代谢杂志。

2021 Oct;106(11):3184-3195.

2021年10月；106（11）：3184-3195。

[9] SKYTROFA

[9] SKYTROFA

®

®

[package insert]. Palo Alto, CA: Ascendis Pharma, Inc.

[包装说明书]。加利福尼亚州帕洛阿尔托：Ascendis制药公司。

[10] Sprogøe K, et al.

[10] Sprogøe K, 等。

Endocr Connect.

内分泌连接。

2017;6(8):R171-r181.

2017；6(8):R171-r181。

[11] Maniatis AK, et al.

[11] 马尼亚蒂斯 AK，等。

Horm Res Paediatr.

激素研究与儿科。

2022 Mar;95(3):233-243.

2022年3月；95（3）：233-243。

[12] Maniatis AK, et al.

[12] 马尼亚蒂斯 AK，等。

J Clin Endocrinol Metab.

临床内分泌与代谢杂志。

2022 Jun;107(7):e2680-e2689.

2022年6月；107（7）：e2680-e2689。

SOURCE Specialised Therapeutics

来源：专业治疗公司

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