A Non-interventional, Observational, Registry-based Study to Investigate Long-term Safety and Clinical Parameters of Somapacitan Treatment in Paediatric Patients With Growth Hormone Deficiency During Routine Clinical Practice

The aim of this study is to look at the long-term safety and clinical parameters of somapacitan in paediatric participants with growth hormone deficiency under routine clinical practice conditions. The study population will include 400 paediatric growth hormone deficient participants from the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (GloBE-Reg) treated with once-weekly somapacitan and fulfilling the eligibility criteria of the study. The total duration of the study is planned to 10 years consisting of a 5-year recruitment period in the GLoBE-Reg followed by a 5-year follow-up period.

开始日期2024-12-13

申办/合作机构

Novo Nordisk A/S

NCT06109935

/ Enrolling by invitationN/A

A Multi-centre, Prospective, Open Label, Single-arm, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Clinical Parameters of Sogroya® Treatment in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed Under Normal Clinical Practice Conditions in Japan Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency (GHD) Where Epiphysial Discs Are Not Closed

The purpose of the study is to investigate the safety and effectiveness of Sogroya® in children with short stature due to growth hormone deficiency where epiphysial discs are not closed under real-world clinical practice in Japan. The study will last for about 1 year (at shortest) to 3 years (at longest) depending on when the participant takes part in the study. The participant will be asked to answer questionnaire(s) about how they feel about the growth hormone (GH) product treatment once during the study (at about 3 months after starting the Sogroya® treatment) and about 3 months after starting the Sogroya® treatment.

开始日期2023-10-04

申办/合作机构

Novo Nordisk A/S

NCT05718570

/ Enrolling by invitationN/A

A Multi-national, Multi-centre, Prospective, Single-arm, Observational, Non-interventional Post-authorisation Safety Study to Investigate Long-term Safety of Sogroya® (Somapacitan) in Adults With Growth Hormone Deficiency (AGHD) Under Routine Clinical Practice

In this study, the general long-term safety and effectiveness of Sogroya (somapacitan) in adults with growth hormone deficiency (AGHD) being treated per normal clinical practice is looked into. In the study, information on side effects and how well Sogroya (somapacitan) works during long term treatment in people with Adult Growth Hormone Deficiency (AGHD) will be collected and analysed. Participants will be treated with Sogroya (somapacitan) as prescribed by the study doctor, in accordance with normal clinical practice. The study will last for 5-10 years, depending on when the participant join the study. The participant will be asked to complete two short questionnaires during every visit to the clinic. The questionnaires will collect information on the participant's well-being, work ability and ability to perform daily activities.

开始日期2023-02-03

申办/合作机构

Novo Nordisk A/S

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项与帕西生长素相关的文献（医药）

2025-05-04·Expert Review of Endocrinology & Metabolism

Long-acting growth hormone in the treatment of children with growth hormone deficiency

Review

作者: Iughetti, Lorenzo ; Predieri, Barbara ; Lucaccioni, Laura ; Insalaco, Anna

INTRODUCTION:

The introduction of long-acting formulations in recent years is changing the landscape of growth hormone (GH) therapy. Daily recombinant human GH (rhGH) has been the treatment of choice for children and adults with GH deficiency (GHD), since its approval in 1985. However, decreasing adherence to treatment over time has been identified as a cause of the decline in rhGH efficacy, leading to significant efforts to develop long-acting rhGH (LAGH) formulations.

AREAS COVERED:

A comprehensive analysis of the literature was conducted to evaluate their mechanism of action, pharmacokinetics, pharmacodynamics, efficacy, safety profile, and administration route. The review focuses on the LAGH approved from both the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of pediatric growth hormone deficiency (PGHD): Lonapegsomatropin, Somatrogon and Somapacitan. We aim to facilitate evidence-based clinical decisions by analyzing the available data on the three LAGH formulations.

EXPERT OPINION:

Even if current evidence suggests a non-inferiority of all the three LAGH formulations when compared to daily rhGH, long-term concerns persist regarding the non-physiological GH profile associated with LAGH, characterized by weekly instead of daily peaks. Further research and real-life studies are required to better define the long-term efficacy of these formulations.

2025-04-30·EUROPEAN JOURNAL OF ENDOCRINOLOGY

Efficacy, safety, and insulin-like growth factor I of weekly somapacitan in children with growth hormone deficiency: 3-year results from REAL4

Article

作者: Kim, Ho-Seong ; Rasmussen, Michael Højby ; Lemminger, Anders Krogh ; Mori, Jun ; Polak, Michel ; Miller, Bradley S ; Blair, Joanne C ; Horikawa, Reiko ; Frystyk, Jan ; Maniatis, Aristides ; Böttcher, Volker

Abstract:

Objective:

Somapacitan is a long-acting GH approved for once-weekly treatment of GH deficiency (GHD). This study aims to evaluate the efficacy and tolerability of somapacitan after 3 years of treatment and 2 years after switch from daily GH in children with GHD.

Design:

Randomized, multi-national, open-labelled, active-controlled parallel-group phase 3 trial, with a 52-week main phase and 3-year safety extension (NCT03811535).

Methods:

Treatment-naïve children with GHD were randomized (2:1) to continuous somapacitan (0.16 mg/kg/week; “soma/soma” group) or daily GH (Norditropin®; 0.034 mg/kg/day) followed by somapacitan (0.16 mg/kg/week; “switch” group).

Results:

Of 200 participants, 188 completed 3 years of treatment. Sustained growth was observed in both groups. At week 156, mean (SD) height velocity (HV) between weeks 104 and 156 was 7.4 (1.5) cm/year in the soma/soma group and 7.8 (1.4) cm/year in the switch group. At week 156, the soma/soma and switch groups had reached a mean (SD) height SD score (HSDS) of −0.95 (0.98) and −1.08 (0.93), respectively, and were approaching the mean mid-parental HSDS of −0.74 (for both groups). Mean total insulin-like growth factor I (IGF-I) SDS during year 3 was similar between groups and within normal range (−2.0 to +2.0). Bioactive IGF-I and bioactive IGF-I to IGF-I ratio were similar between groups. Somapacitan was well tolerated, with low proportions reporting injection-site reactions.

Conclusions:

Sustained efficacy and tolerability were observed for continuous somapacitan treatment for 3 years, and for 2 years after the switching from daily GH treatment. HSDS in both groups was approaching mean mid-parental HSDS.

Clinical trial registration:

NCT03811535

2025-04-30·Annals of Pediatric Endocrinology & Metabolism

Long-acting growth hormones: innovations in treatment and guidance on patient selection in pediatric growth hormone deficiency

Article

作者: Faleschini, Elena ; Tornese, Gianluca ; Rodaro, Chiara ; Tamaro, Gianluca

Long-acting growth hormones (LAGHs) represent a significant advancement in the treatment of pediatric GH deficiency, offering an alternative to daily recombinant human GH (rhGH) therapy. Traditional rhGH treatments, although effective, require daily injections, often leading to poor adherence due to the frequency of dosing, injection pain, and difficulties with storage and travel. In contrast, LAGHs, such as somatrogon, somapacitan, and lonapegsomatropin, are designed for once-weekly administration, improving patient compliance and quality of life. LAGHs have demonstrated non-inferiority to daily rhGHs in phase 3 clinical trials, showing similar efficacy in terms of growth velocity and safety profiles. Despite these advantages, concerns remain regarding the altered pharmacodynamics of LAGHs such as the lack of pulsatile secretion and potential for antibody formation. Although the overall safety of LAGHs has been confirmed, side effects, such as lipoatrophy at the injection site, may occur, especially with PEGylated formulations. Guidelines for prescribing LAGHs are currently evolving. LAGHs are not yet approved for other conditions traditionally treated with rhGHs, such as Turner or Noonan syndrome. Pediatric endocrinologists should carefully consider which patient groups would benefit most from this therapy, particularly individuals at risk of poor adherence to daily injections such as patients undergoing multidrug therapy, patients with needle phobia or behavioral disorders, very young children, adolescents, patients with separated parents, families that travel frequently, or children involved in activities such as scouting. LAGHs present an opportunity to enhance therapeutic outcomes and adherence, but careful patient selection remains critical to maximize their potential benefits.

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2025-05-17

·CPHI制药在线

3个1类创新药国内上市；最高超10亿美元！石药集团与Cipla就伊立替康脂质体注射液达成授权合作 | 制药在线一周药闻复盘

点击蓝字关注我们本周，热点很多。首先是审评审批方面，最值得关注的就是3个1类创新药国内上市，分别是北海康成的注射用维拉苷酶β、海思科的安瑞克芬注射液以及轩竹生物的吡洛西利片；其次是研发方面，多个药取得重要进展，其中，百济神州BTK降解剂又启动一项头对头礼来匹妥布替尼的Ⅲ期临床研究；最后是交易及投融资方面，交易很多，值得一提的是，石药集团就伊立替康脂质体注射液达成授权合作，总交易额超10亿美元。本期盘点包括审评审批、研发以及交易及投融资三大板块，统计时间为2025.5.12-5.16，包含28条信息。视频点个审评审批NMPA上市批准1、5月14日，NMPA官网显示，诺华的盐酸阿思尼布片获批上市，用于治疗新诊断的费城染色体阳性的慢性髓细胞白血病（Ph+CML）慢性期（CP）成人患者。阿思尼布是一款基于ABL肉豆蔻酰口袋（STAMP）创新机制的靶向药物，可实现深度缓解率翻倍和不良事件相关停药风险减半，延缓疾病进展，为CML患者提供了全新治疗选择。2、5月14日，NMPA官网显示，诺华的CDK4/6抑制剂瑞波西利片（商品名：凯丽隆）获批新适应症，适用于与芳香化酶抑制剂联合使用，作为激素受体（HR）阳性、人类表皮生长因子受体2（HER2）阴性高复发风险的早期乳腺癌患者的辅助治疗。3、5月15日，NMPA官网显示，北海康成1类新药注射用维拉苷酶β（商品名：戈芮宁）已通过优先审评审批程序批准上市，适用于12岁及以上青少年和成人Ⅰ型和Ⅲ型戈谢病患者的长期酶替代治疗。维拉苷酶β是一款针对戈谢病开发的酶替代疗法（ERT）。4、5月15日，NMPA官网显示，轩竹生物1类新药吡洛西利片（商品名：轩悦宁）获批上市，适用于激素受体（HR）阳性、人表皮生长因子受体2（HER2）阴性晚期或转移性乳腺癌成人患者：与氟维司群联合用于既往接受内分泌治疗后出现疾病进展的患者；单药用于既往转移性阶段接受过两种及以上内分泌治疗和一种化疗后出现疾病进展的患者。5、5月15日，NMPA官网显示，海思科1类新药安瑞克芬注射液（HSK21542）获批上市，用于治疗腹部手术后的轻、中度疼痛。安瑞克芬（HSK21542）是一款外周kappa阿片受体选择性激动剂。除了注射液剂型，海思科也在开发HSK21542的口服制剂，目前正在开展临床研究。申请6、5月12日，CDE官网显示，百济神州1类新药索托克拉片新适应症申报上市，拟用于治疗既往接受过抗CD20治疗和BTKi治疗的套细胞淋巴瘤（MCL）成人患者。索托克拉片的该项上市申请已经被CDE纳入优先审评。7、5月14日，CDE官网显示，勃林格殷格翰的口服选择性磷酸二酯酶4B（PDE4B）抑制剂那米司特片（nerandomilast）第二个适应症申报上市，用于治疗成人进展性肺纤维化（PPF）。2025年2月，首个适应症用于治疗成人特发性肺纤维化（IPF）的上市申请已递交。临床批准8、5月14日，CDE官网显示，信念医药的BBM-P002注射液获批临床。BBM-P002注射液利用AAV载体将多巴胺合成所需的表达基因递送到患者大脑壳核中持续表达，有望实现“一次给药、长期有效”的帕金森病治疗。9、5月14日，CDE官网显示，百济神州1类新药BGB-16673片获批两项临床，拟开发治疗慢性自发性荨麻疹。BGB-16673是一种口服靶向布鲁顿氏酪氨酸激酶（BTK）的嵌合式降解激活化合物（CDAC），是百济神州CDAC平台首款在研药物。10、5月14日，CDE官网显示，基石药业的注射用CS2009获批临床，用于晚期实体瘤。这是该药首次在国内获批临床。CS2009是一款同时靶向PD-1/VEGFA/CTLA-4三特异性抗体，在实体瘤治疗领域具有广阔的临床应用前景。优先审评11、5月14日，CDE官网显示，德国默克申报的盐酸匹米替尼胶囊（ABSK021胶囊）拟纳入优先审评，用于需要系统性治疗的腱鞘巨细胞瘤（TGCT）成人患者。匹米替尼是和誉医药研发的一种新型、口服、高选择性且高效的小分子CSF-1R抑制剂。德国默克拥有该产品的全球商业化权利。12、5月16日，CDE官网显示，科伦博泰申报的注射用芦康沙妥珠单抗拟纳入优先审评，用于既往接受过内分泌治疗且在晚期或转移性阶段接受过其他系统治疗的不可切除的局部晚期或转移性激素受体阳性（HR+）且人表皮生长因子受体2阴性（HER2-）乳腺癌成人患者。芦康沙妥珠单抗是一款新型TROP2靶向抗体偶联药物（ADC），此前已经在中国获批两项适应症。突破性疗法13、5月16日，CDE官网显示，和誉医药1类新药ABSK-011胶囊拟纳入突破性治疗，适应症为：经免疫检查点抑制剂（ICI）和多靶点酪氨酸激酶抑制剂（mTKI）治疗的成纤维细胞生长因子19（FGF19）过表达的晚期肝细胞癌（HCC）。ABSK-011是一款具有高选择性的FGFR4小分子抑制剂。FDA上市批准14、5月14日，FDA官网显示，艾伯维的c-Met靶向ADC药物telisotuzumab vedotin（Teliso-V，ABBV-399）获批上市，用于治疗既往接受过治疗的c-Met蛋白过表达、表皮生长因子受体（EGFR）野生型晚期/转移性非鳞状非小细胞肺癌（NSCLC）成人患者。该药物是c-Met赛道首款获批上市的ADC产品，也是艾伯维首款自研ADC产品。15、5月16日，FDA官网显示，中国生物制药与清普生物合作开发的非阿片类镇痛新药美洛昔康注射液（II）（商品名：QAMZOVA）获批上市。普坦宁不仅成为了中国首款一日一次24h长效的非甾体抗炎药（NSAID）注射液，也是中国首款获得FDA批准的镇痛新药和全球唯一在售的长效NSAID注射液。研发临床状态16、5月13日，正大天晴公布了1类新药盐酸安罗替尼胶囊联合派安普利单抗注射液一线治疗晚期肝细胞癌（HCC）的关键注册Ⅲ期临床（APOLLO研究）结果，一线治疗晚期HCC取得无进展生存期（PFS）和总生存期（OS）双终点阳性结果。安罗替尼联合派安普利单抗一线治疗晚期肝细胞癌已于2024年11月递交上市申请。17、5月13日，GSK和iTeos宣布，由于中期临床数据未能达到预期效果，双方决定终止合作开发TIGIT抗体belrestotug。该药物是GSK于2021年6月以6.25亿美元的预付款，最高可达14.5亿美元总里程碑付款从iTeos获得。双方将合作开发和推广TIGIT抗体Belrestotug，共同承担Belrestotug全球开发成本。18、5月15日，clinicaltrial官网显示，百济神州启动了一项Ⅲ期临床（CaDAnCe-304），旨在评估BGB-16673（200mg，每日1次）对比Pirtobrutinib（200mg，每日1次）治疗既往接受过共价BTK抑制剂（cBTKi）治疗的复发或难治性（R/R）慢性淋巴细胞白血病（CLL）或小淋巴细胞淋巴瘤（SLL）患者的疗效和安全性，主要终点是无进展生存期（PFS）。19、5月16日，诺诚健华宣布，其研发的ICP-332治疗非节段型白癜风的Ⅱ/Ⅲ期临床试验在中国完成首例患者给药。ICP-332是一款在研的1类创新药，是高效、高选择性的新型口服TYK2抑制剂，正在被开发用于治疗各种T细胞相关的自身免疫性疾病，包括白癜风、特应性皮炎、结节性痒疹等。20、5月16日，信达生物宣布，胰高血糖素（GCG）/胰高血糖素样肽-1（GLP-1）双受体激动剂玛仕度肽在中国超重或肥胖合并代谢相关脂肪性肝病（MAFLD）受试者中的一项Ⅲ期临床（GLORY-3）完成首例受试者给药。本研究是一项在中国超重或肥胖合并MAFLD受试者中比较玛仕度肽与一款GLP-1单受体激动剂有效性和安全性的多中心、随机、开放标签的Ⅲ期临床研究（NCT06884293）。临床数据21、5月11日，礼来公布了SURMOUNT-5研究的详细结果。该研究是一项开放标签Ⅲb期临床，旨在针对肥胖或伴有至少一种合并症（如高血压、血脂异常、阻塞性睡眠呼吸暂停、心血管疾病）的超重成人（且不合并糖尿病）患者，评估GIP/GLP-1双受体激动剂替尔泊肽与GLP-1单受体激动剂司美格鲁肽的有效性与安全性。替尔泊肽组平均体重减轻了50.3磅（22.8公斤），司美格鲁肽组平均体重减轻了33.1磅（15.0公斤）。22、5月12日，诺和诺德公布了Ⅲ期REAL8篮子试验的数据，结果显示：每周给药一次的Sogroya在改善小于胎龄儿（SGA）、努南综合征（NS）、特发性矮小症（ISS）的青春期前儿童的年生长速率（通过52周时身高生长速度HV测量）方面，与每日给药一次的生长激素Norditropin相当。此外，在患有努南综合征（NS）的儿童中，Sogroya较每日一次的生长激素具有优越性，同时在小于胎龄儿（SGA）的儿童中，Sogroya较每日低剂量的生长激素也具有优越性。23、5月13日，邦耀生物公布了CRISPR/Cas9基因编辑的异体抗CD19CAR-T细胞（TyU19）治疗复发/难治性系统性红斑狼疮（SLE）数据。研究结果表明，TyU19细胞在SLE治疗中可实现快速、深度的临床缓解，同时展现出优异的临床安全性。24、5月16日，爱科百发宣布，其开发的新一代抗纤维化药物AK3280的Ⅱ期验证性临床研究取得积极成果。AK3280是基于已有的IPF药物结构优化的新一代抗纤维化药物，临床前数据显示其抗纤维化作用更强，药代动力学更佳，有望改善现有药物的胃肠道耐受性和其他毒副作用。交易及投融资25、5月14日，GSK宣布，与BostonPharmaceuticals达成协议，将收购其主要资产efimosferminalfa。根据协议，GSK将支付高达20亿美元的现金对价总额，包括12亿美元的预付款和高达8亿美元的里程碑付款。GSK还将向诺华支付该药物的分级特许权使用费。该药是一种可每月1次皮下注射给药的潜在同类最佳FGF21（成纤维细胞生长因子21）长效类似物。26、5月14日，诺和诺德宣布，与Septerna达成一项独家全球合作，双方将共同致力开发并商业化针对肥胖症、2型糖尿病及其他心脏代谢疾病的口服小分子药物。根据协议，Septerna将有资格从诺和诺德获得总计约22亿美元的付款，包括首付款及研发、商业化里程碑款项，其中超过2亿美元为预付款及近期里程碑付款。Septerna还可获得上市产品的分层特许权使用费。诺和诺德将承担合作项目的全部研发费用。27、5月15日，石药集团宣布，与Cipla就伊立替康脂质体注射液达成协议。根据协议，石药同意授予Cipla独家许可。石药将收取1500万美元首付款，最高达2500万美元潜在首次商业销售及最高达10.25亿美元的里程碑付款。该产品为一种拓朴异构酶1抑制剂，含有43mg/10ml伊立替康。目前用于治疗多种实体瘤，如转移性、结直肠癌、肺癌及宫颈癌等。28、5月16日，科霸生物宣布，已与三菱田边制药签署合作协议。双方将围绕一个全球首创的免疫细胞类靶点。科霸生物率先提出并实践了一种全新的ADC开发理念，即靶向肿瘤免疫微环境中的免疫细胞受体，尤其是大量存在的髓系抑制细胞、肿瘤相关巨噬细胞（TAM）等关键免疫抑制性细胞类型。END领取CPHI & PMEC China 2025展会门票【智药研习社直播预告】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

上市批准优先审批临床3期申请上市

2025-05-13

·FiercePharma

Novo heads to regulators after Sogroya matches predecessor Norditropin in 3 pediatric growth disorders

The FDA first approved Sogroya in August 2020 as a once-weekly treatment for adult growth hormone deficiency. The drug’s label was expanded to include children in April 2023. With a late-stage trial win for its Norditropin heir in hand, Novo Nordisk is making good on plans to boost its profile in the realm of rare disease.Over the weekend, Novo unveiled data from the phase 3 Real8 study, which found that the company’s once-weekly human growth hormone analogue Sogroya was largely on par with its daily predecessor, Norditropin, in a trio of growth disorders in kids.At the basket study’s 52-week mark, Sogroya—first approved in 2020—proved noninferior to Norditropin at improving yearly growth rate in prepubescent children with idiopathic short stature (ISS).Meanwhile, Sogroya demonstrated superiority over Novo’s daily growth hormone in children with Noonan syndrome (NS) and in children born small for gestational age (SGA) when compared to a lower Norditropin dose.When pitted against a higher dose of its growth hormone predecessor in SGA, Sogroya’s efficacy was noninferior, Novo explained in its Monday release.Sogroya was well tolerated in the study, and no new safety flags cropped up that diverged from Norditropin’s long-established profile, the company added.Sogroya’s performance in Real8 allowed the first three sub-studies to meet their primary endpoints. Leveraging data from Real8 and Real9, Novo said it has already filed for approval of Sogroya in SGA, NS and ISS with regulators in the U.S. and Europe.Novo’s Real8 program is also testing Sograya’s mettle in Turner syndrome, though those data won’t arrive until “later this year,” the company said. The FDA first approved Sogroya in August 2020 as a once-weekly treatment for adult growth hormone deficiency. The drug’s label was expanded to include children in April 2023.Sogroya is meant to succeed Novo’s once-a-day growth hormone Norditropin, which has now been on the market for decades. Eli Lilly, Merck & Co. and Pfizer all sell their own daily growth disorder drugs, while newer meds like Ascendis’ Skytrofa and Pfizer’s Ngenla are challenging Sogroya directly on the weekly-dosing front.Novo lumps sales of Norditropin and Sogroya together into its rare endocrine disorders category, which grew sales 31% in 2024 to roughly 5 billion Danish kroner ($745 million).Analytics firm GlobalData has forecast Sogroya will hit $294 million in 2030 sales and expects Norditropin to reach $960 million in sales that same year. The Sogroya results have likely bolstered Novo’s confidence in rare disease, where the company has been playing a “long endgame” since the late 2010s, Ludovic Helfgott, the company’s executive vice president of rare disease, said in a March interview with Fierce Pharma.Aside from drugs for growth disorders like Norditropin and Sogroya, Novo’s rare disease pedigree includes treatments for blood disorders like Esperoct, NovoEight or the recently approved daily hemophilia injection Alhemo, Helfgott explained.Novo plans to stick to those two major disease areas—growth and blood disorders—as it presses forward in the field.“It’s our core, and we will continue to develop this both internally and externally,” Helfgott said.

临床3期上市批准临床结果

2025-05-13

·药智网

诺和诺德公布Sogroya的III期数据

在儿童生长发育领域，小于胎龄儿（SGA）、努南综合征（NS）、特发性矮小症（ISS）等疾病一直是困扰诸多家庭的难题，传统的每日一次生长激素治疗虽有一定效果，但患者依从性不佳等问题限制了其疗效的充分发挥。然而，5月12日，诺和诺德公布的一项重磅研究成果——其每周一次生长激素Sogroya（somapacitan）III期REAL8篮子试验数据，为这一困境带来了新的曙光，开启了每周一次生长激素治疗的新篇章。Sogroya是一种人生长激素类似物，采用白蛋白结合延长技术，这一独特技术使得Sogroya能够附着在血液中的白蛋白上，从而延缓其清除速度，发挥更长时间的作用，为患者提供更加便捷的治疗选择。而此次的III期REAL8篮子试验更是采用了创新的研究设计，它并非针对单一适应证进行研究，而是在同一试验方案下，同时研究Sogroya在SGA、特纳综合征（TS）、NS和ISS四种不同但相关的适应证中的应用。在研究过程中，Sogroya与传统的每日一次生长激素Norditropin进行了严谨的对比。研究对象涵盖了SGA、NS、ISS的青春期前儿童，通过52周时身高生长速度HV测量年生长速率，以科学、客观的方式评估Sogroya的疗效。结果显示，在SGA儿童中，与低剂量Norditropin相比，Sogroya的估算年平均身高生长速率HV更优，达到11.0厘米/年，相较于Norditropin的9.4厘米/年展现出显著优势；与高剂量Norditropin相比，Sogroya也达到了非劣效性标准，分别为11.0厘米/年与11.1厘米/年，疗效相当。在NS儿童中，Sogroya的估算年平均HV为10.4厘米/年，优于Norditropin的9.2厘米/年。而在ISS儿童中，两者估算年平均HV均为10.5厘米/年，Sogroya与Norditropin的疗效相当。除了令人瞩目的疗效数据，Sogroya的安全性同样值得关注。REAL8研究显示，Sogroya耐受性良好，与每日一次的生长激素相比，并未发现安全性和耐受性问题。从患者的角度来看，每周一次的Sogroya给药方案无疑是一个巨大的福音。传统的每日一次生长激素治疗，需要患者或家长每天进行注射，这不仅给患者带来了身体上的不适，如注射部位的疼痛等，还增加了心理负担，容易导致患者产生抵触情绪，进而影响治疗依从性。而Sogroya的每周一次给药方式，极大地减轻了家长和孩子的负担，提高了治疗的依从性。目前，诺和诺德已在欧盟和美国提交了Sogroya三种适应症的监管审查申请。结语：诺和诺德公布的Sogroya III期REAL8篮子试验数据，以其显著的疗效、良好的安全性以及创新的研究设计，为儿童生长障碍的治疗带来了革命性的变革。参考来源：1.https://news.qq.com/rain/a/20250512A08XZQ002.https://mp.weixin.qq.com/s/vMFpisn35zScmbOGsqZ2hA声明：本内容仅用作医药行业信息传播，不代表药智网立场。对本文有异议或投诉，请联系maxuelian@yaozh.com。责任编辑 | 史蒂文合作、投稿 | 马老师 18323856316（同微信）阅读原文，是受欢迎的文章哦

临床3期临床成功

100 项与帕西生长素相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11194	帕西生长素	H01AC01	DB15093

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
生长激素缺乏症	美国	Novo Nordisk, Inc.	2020-08-28

未上市

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适应症	最高研发状态	国家/地区	公司	日期
侏儒症	临床3期	美国	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	中国	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	日本	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	奥地利	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	比利时	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	巴西	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	保加利亚	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	加拿大	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	克罗地亚	Novo Nordisk A/S	2022-08-10
侏儒症	临床3期	芬兰	Novo Nordisk A/S	2022-08-10

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05330325 (REAL8, NEWS) 人工标引	临床3期	生长障碍	-	Sogroya	餘膚艱餘糧鬱鑰窪鏇壓(衊願廠夢觸觸獵積遞積) = 艱壓鏇觸夢範鏇夢膚壓積鏇艱鹽蓋觸鏇壓獵壓 (鹹糧顧蓋襯壓壓積憲餘 ) 更多	相似	2025-05-12
				Norditropin	餘膚艱餘糧鬱鑰窪鏇壓(衊願廠夢觸觸獵積遞積) = 繭製鹽廠鬱襯簾淵網鏇積鏇艱鹽蓋觸鏇壓獵壓 (鹹糧顧蓋襯壓壓積憲餘 ) 更多
NCT04970654 (CTgov)	临床3期	生长激素缺乏症	110	Norditropin (Norditropin)	選顧蓋鹹齋糧築鹽鹽齋(壓窪築遞鹽糧糧願鹽衊) = 壓糧鬱壓範餘選鑰廠築範齋簾糧網淵範築繭鹽 (築製淵餘築壓選艱餘衊, 2.3) 更多	-	2024-12-10
				Somapacitan (Somapacitan)	選顧蓋鹹齋糧築鹽鹽齋(壓窪築遞鹽糧糧願鹽衊) = 壓醖齋窪衊醖獵獵積願範齋簾糧網淵範築繭鹽 (築製淵餘築壓選艱餘衊, 2.1) 更多
ENDO2024	N/A	生长激素缺乏症 serum C-reactive protein (CRP) levels	8	Somapacitan	衊顧簾遞衊觸築蓋鹽窪(醖築鬱繭憲醖夢糧觸壓) = 廠鏇繭壓鑰製糧觸遞網築鹽糧鏇鑰淵範鬱餘簾 (顧願廠齋顧鹹獵範鬱醖 ) 更多	积极	2024-06-01
ENDO2024	N/A	-	36	somapacitan (Newly prescribed cases)	鬱鹹齋築築壓鬱襯積夢(廠選網顧醖鏇鑰齋獵鏇) = There were no adverse events, but some participants commented that it was easier to forget weekly injections of somapacitan than the daily GH 窪遞膚蓋膚遞夢繭鹹衊 (夢製艱窪壓積淵蓋鏇壓 ) 更多	-	2024-06-01
				somapacitan (Switched cases)
NCT03811535 (REAL4, ENDO2023)	临床3期	-	68	Somapacitan	鹽願積觸糧製繭簾選觸(窪積築範壓糧鬱築憲製) = 餘鏇廠簾餘糧顧襯鬱鏇築網顧積簾築壓選淵淵 (鏇廠淵積壓糧積糧襯夢 ) 更多	积极	2023-10-05
THU073 (ENDO2023)	N/A	-	32	Somapacitan 5 mg/1.5 mL	壓憲廠鏇夢醖顧網繭積(淵艱網夢襯衊淵構鏇鬱) = 鹹夢憲膚鑰鬱壓顧築簾蓋鑰鬱蓋願醖餘憲願構 (艱憲製範構艱襯廠遞膚 )	积极	2023-10-05
				Somapacitan 10 mg/1.5 mL	壓憲廠鏇夢醖顧網繭積(淵艱網夢襯衊淵構鏇鬱) = 願鏇糧積鹹顧觸簾衊夢蓋鑰鬱蓋願醖餘憲願構 (艱憲製範構艱襯廠遞膚 )
NCT03811535 (REAL4, ENDO2023)	临床3期	生长激素缺乏症	-	Somapacitan 0.16 mg/kg/week	醖簾窪築製醖鬱憲糧齋(網淵獵壓窪製鹹網願顧) = A low proportion of children reported injection-site reactions in the second year (2.9% and 2.3% in the switch and soma/soma groups, respectively) with no injection site pain reported in either group 築淵構齋遞襯廠獵壓糧 (窪衊憲繭製顧獵網選醖 ) 更多	积极	2023-10-05
				Daily GH (0.034 mg/kg/day Norditropin)
NCT03878446 (RF26 \| PMON335, ENDO2022)	临床2期	小于胎龄儿	62	Somapacitan 0.16 mg/kg/week	築鹹廠簾餘餘積獵簾窪(淵觸遞築鑰襯構鬱淵築) = 鏇蓋廠鏇鹽壓鬱鏇鹹憲齋觸衊顧憲鏇鬱鏇淵遞 (齋憲艱餘網構衊艱繭製 )	积极	2022-11-01
				Somapacitan 0.20 mg/kg/week	築鹹廠簾餘餘積獵簾窪(淵觸遞築鑰襯構鬱淵築) = 鏇鑰艱網製積衊範襯構齋觸衊顧憲鏇鬱鏇淵遞 (齋憲艱餘網構衊艱繭製 )
NCT02616562 (REAL 3, ENDO2022)	临床2期	生长激素缺乏症	45	Daily GH 0.034 mg/kg/day	遞醖憲築醖遞壓鏇鏇簾(窪鏇築廠繭鹹糧蓋簾醖) = one patient in the somapacitan/somapacitan group experienced five injection-site reactions; none occurred in the daily GH/somapacitan group 醖製簾膚製鏇顧襯夢夢 (廠憲積鏇襯餘顧簾繭壓 ) 更多	积极	2022-11-01
				Somapacitan/somapacitan
NCT03811535 (REAL4, ENDO2022)	临床3期	生长激素缺乏症	-	Somapacitan 0.16 mg/kg/week	醖製鏇醖積蓋鹹蓋壓餘(簾顧艱膚鑰襯艱觸鹽鹽) = 獵願範繭醖鏇夢餘醖夢製餘積築膚鹹鹽遞衊醖 (網夢鏇鹽淵膚淵鹹選艱 ) 更多	积极	2022-11-01
				Daily GH 0.034 mg/kg/day Norditropin	醖製鏇醖積蓋鹹蓋壓餘(簾顧艱膚鑰襯艱觸鹽鹽) = 範願衊襯鹽鏇範淵鬱遞製餘積築膚鹹鹽遞衊醖 (網夢鏇鹽淵膚淵鹹選艱 ) 更多