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

100 项与帕西生长素相关的临床结果

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100 项与帕西生长素相关的转化医学

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100 项与帕西生长素相关的专利（医药）

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项与帕西生长素相关的文献（医药）

2025-06-01·Endocrine Practice

Efficacy of Somapacitan in Treatment-Fatigue Adult Patients With Growth Hormone Deficiency Previously Treated With Once-Daily Growth Hormone Injections: A 24-Week Randomized Active-Controlled Trial

Article

作者: Janež, Andrej ; Rakusa, Matej ; Goričar, Katja ; Herman, Rok ; Jensterle, Mojca ; Klinc, Ana

OBJECTIVE:

We evaluated the efficacy of somapacitan in a 24-week, randomized, active-controlled study in patients with growth hormone deficiency (GHD) who experienced fatigue from daily growth hormone (GH) injections.

METHODS:

Twenty-nine adult patients with GHD, pretreated with daily GH for ≥5 years, who had reported treatment-related fatigue, were randomized to somapacitan or daily GH. Outcome measures were changes in treatment satisfaction assessed by Treatment Satisfaction Questionnaire for Medication-9, insulin-like growth factor-1 (IGF-1) standard deviation score, glucose and lipid parameters, body composition, bone mineral density (BMD), carotid intima media thickness, and reactive hyperaemia index, from baseline to week 24.

RESULTS:

The difference in change in Treatment Satisfaction Questionnaire for Medication-9 score for convenience was significant, in favor of somapacitan (estimated difference, somapacitan-daily GH [95% CI]:23.2 [7.9; 38.4] points, P = .004). No differences between treatment arms in estimated changes from baseline to study-end were observed for IGF-1 levels, glucose and lipid profile, visceral adipose tissue, fat mass (%), lean body mass, and vascular parameters. There was significant difference in BMD of the lumbar spine (estimated difference, somapacitan-daily GH [95% CI] -0.036 (-0.064, -0.009) gr/cm², P = .011).

CONCLUSION:

In AGHD patients who were fatigued from the long-term daily GH injections, somapacitan was reported to be more convenient than daily GH. It was effective in maintaining IGF-1 levels and body composition, glucose, lipids, and vascular parameters, comparable to daily GH. Nonsignificant decrease in BMD with somapacitan could reflect a favorable increase in bone metabolic units, as previously observed in naïve patients with GHD during the initial 6-month period of GH therapy.

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

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

项与帕西生长素相关的新闻（医药）

2025-06-02

·梅斯医学

3岁男孩走路总是跌跌撞撞，说话也越来越含糊不清！一查：不是脑瘫，而是罕见代谢病！

3岁的小安原本活泼好动，但最近几个月，爸妈发现他好像“越来越不会走路”了——走几步就摔跤、跑不动、说话也越来越含糊，甚至连控制尿裤子的能力都变差了。一开始，医生怀疑是脑瘫，但奇怪的是，小安的脑MRI并没有发现典型异常，营养、微量元素、甲功等检查也都在正常范围。随着症状逐渐加重，小安甚至开始出现烦躁、嗜睡、呕吐，一次血氨检查竟发现——血氨明显升高！医生立刻转向“高氨血症”方向进行代谢病排查，最终在氨基酸分析和基因检测中发现，小安患的是一种极为罕见的精氨酸酶缺乏症（Arginase deficiency），是一种尿素循环障碍疾病。原来，正常人体需要通过“尿素循环”把体内多余的氨排出体外，而小安因为精氨酸酶活性缺陷，导致氨无法有效代谢，长期堆积在体内，对大脑产生毒性，进而影响运动、认知发育，甚至危及生命。“精氨酸酶缺乏症精氨酸酶缺乏症（argininemia，Arginase deficiency）又称精氨酸血症（Hyperargininemia），是一种罕见的常染色体隐性遗传代谢病，患者血液、脑脊液中精氨酸明显升高，主要表现是智力和运动障碍、痉挛性瘫痪，早期常被误诊为脑性瘫痪及神经变性病，生存质量极差，预后不良。在美国的新生儿发病率为1/1000000～1/300000；日本的发病率约为1/350000。我国尚无相关流行病学调查报告。本病属于遗传代谢性疾病，多在儿童时间被发现。“临床表现患儿出生时正常，婴儿期多无明显症状，通常在1岁后逐渐出现运动障碍，下肢呈“剪刀状”强直，发展为四肢痉挛性瘫痪，伴身材矮小和认知发育迟缓。常见症状包括烦躁、嗜睡、喂养困难、呕吐、小头畸形、手足徐动、惊厥和睡眠障碍，部分患儿可有肝肿大。与其他尿素循环障碍不同，精氨酸酶缺乏症患者很少出现昏迷。高氨血症可因感染、高蛋白饮食或禁食等应激因素诱发。婴儿期表现为烦躁、嗜睡、拒奶、呼吸困难、呕吐，严重时可出现昏迷；幼儿期则表现为恶心、吞咽困难、动作笨拙和易跌倒。部分患儿可于新生儿期即出现惊厥，进展后表现为精神发育迟滞、痉挛性瘫痪、共济失调等神经系统损害。超过一半患儿有癫痫发作，常为强直-阵挛性发作，也可为局灶性发作、失神发作或癫痫持续状态。神经系统外表现少见，主要影响肝脏，可见轻度肝细胞损伤、凝血功能异常、急性肝衰竭及胆汁淤积性黄疸、肝纤维化。长期肌肉痉挛还可能导致脊柱侧弯或前弯等畸形。“诊断根据患者症状，结合血液精氨酸增高及ARG1基因检测结果可诊断。患者有四肢进行性痉挛性瘫痪、认知功能落后及烦躁、呕吐、嗜睡等高氨血症表现，一些患者肝功能异常。血氨、精氨酸浓度升高。肝脏或红细胞精氨酸酶活性降低有确诊价值，但是，由于取材及技术困难，不能作为常规诊断方法。ARG1等位基因致病突变，可明确诊断。“治疗精氨酸酶缺乏症目前尚无根治方法，治疗目标是降低血氨浓度、减少精氨酸积聚、缓解神经系统症状，以延缓病情进展、改善患儿生活质量。治疗主要包括饮食干预、药物治疗及部分情况下的手术治疗。饮食治疗是基础，通过限制蛋白质摄入、提供低精氨酸、高热量饮食，并补充富含支链氨基酸的特殊氨基酸粉，以控制血氨和血精氨酸水平，减轻神经系统症状，改善运动、行为和语言功能。药物治疗方面，血氨升高时可使用氮清除剂（如苯甲酸钠、苯丁酸钠），促进氨的排泄；癫痫发作时使用抗癫痫药物（如苯巴比妥、卡马西平）进行控制。急性期如出现高氨血症，应立即采取禁蛋白、高热量支持、药物促进排氮等措施，必要时进行血液透析以快速降低血氨，防止高氨血症脑病发生。在某些情况下，肝移植可作为替代治疗手段，有望纠正代谢缺陷，但在婴幼儿中实施受限较多。干细胞移植作为潜在治疗方式，仍处于探索阶段，目前在精氨酸酶缺乏症中的疗效尚不明确。参考资料：[1]精氨酸酶缺乏症[J].中国实用乡村医生杂志,2020,27(08):32-33.[2]Lal RA, Hoffman AR.Long-Acting Growth Hormone Preparations in the Treatment of Children[].Pediatr Endocrinol Rev, 2018, 16 (Suppl1) :162-167[3]Salzano A, Marra AM, D'Assante R, et al.Growth Hormone Therapy in Heart Failure[J].Heart Fail Clin, 2018, 14 (4) :501-515来源 | 梅斯医学编辑 | wanny神经系统罕见病交流群↓点击下方“阅读原文”，下载梅斯医学APP吧！

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

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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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	壓顧鹽選觸餘壓構簾鏇(鹹觸淵衊鬱鹹積願遞築) = 襯窪鹽壓夢齋簾餘醖窪壓襯醖繭廠繭艱鹹壓鬱 (網築願糧鹹襯積衊夢艱 ) 更多	相似	2025-05-12
				Norditropin	壓顧鹽選觸餘壓構簾鏇(鹹觸淵衊鬱鹹積願遞築) = 蓋簾網製鹹艱獵選壓製壓襯醖繭廠繭艱鹹壓鬱 (網築願糧鹹襯積衊夢艱 ) 更多
NCT03811535 (REAL4, Pubmed \| Eur J Endocrinol)	临床3期	生长激素缺乏症 insulin-like growth factor I	200	Somapacitan 0.16 mg/kg/week	壓簾醖獵獵簾遞簾獵積(鑰襯夢齋觸醖鏇獵網壓) = low proportions reporting injection-site reactions 願鬱鹽膚窪鑰積鏇蓋築 (膚積鬱簾膚選鏇鏇糧壓 )	积极	2025-04-30
				Norditropin® 0.034 mg/kg/day
NCT04970654 (CTgov)	临床3期	生长激素缺乏症	110	Norditropin (Norditropin)	憲齋蓋鑰夢窪簾蓋簾觸(積餘選淵範鏇齋艱夢繭) = 醖淵鑰鬱憲餘衊觸淵遞壓餘淵蓋鏇餘壓鏇範醖 (艱築網鹹築壓繭壓構襯, 2.3) 更多	-	2024-12-10
				Somapacitan (Somapacitan)	憲齋蓋鑰夢窪簾蓋簾觸(積餘選淵範鏇齋艱夢繭) = 鹹鹹築糧鏇襯鹹壓鹽積壓餘淵蓋鏇餘壓鏇範醖 (艱築網鹹築壓繭壓構襯, 2.1) 更多
ENDO2024	N/A	生长激素缺乏症 serum C-reactive protein (CRP) levels	8	Somapacitan	選壓壓鬱鬱鑰夢選鏇鹹(淵衊繭範製鏇鬱襯鬱衊) = 鹹夢範艱願簾淵餘膚艱獵鬱顧餘蓋餘憲顧選築 (齋壓淵遞襯積願夢醖範 ) 更多	积极	2024-06-01
ENDO2024	N/A	-	36	somapacitan (Newly prescribed cases)	獵夢醖淵鹽廠製簾範範(構壓醖糧衊壓壓襯齋蓋) = There were no adverse events, but some participants commented that it was easier to forget weekly injections of somapacitan than the daily GH 糧齋簾鹽襯鬱網觸蓋醖 (夢淵齋範夢鬱齋齋網積 ) 更多	-	2024-06-01
				somapacitan (Switched cases)
NCT03811535 (REAL4, ENDO2024)	临床3期	生长激素缺乏症	200	Somapacitan 0.16 mg/kg/week	襯觸網選獵廠顧蓋齋壓(觸網窪顧顧鑰築鏇網餘) = During the extension period (week 52 to 156), a low proportion of the participants reported injection site reactions 襯鏇鹹窪鹽鬱獵壓願醖 (淵鹹淵製襯膚鬱選糧獵 ) 更多	积极	2024-06-01
				Norditropin 0.034 mg/kg/day
NCT03811535 (REAL4, Pubmed \| Clin Endocrinol (Oxf))	临床3期	生长激素缺乏症	-	Somapacitan (0.16 mg/kg/week)	繭糧鏇糧構艱範鏇遞窪(蓋範獵鑰鹹製餘壓鏇淵) = 襯觸憲鏇艱鬱膚簾願鹽遞鏇遞選網積積衊憲鬱 (蓋構簾蓋遞選鑰鹹蓋築 )	积极	2024-04-01
				Daily GH (0.034 mg/kg/day)	繭糧鏇糧構艱範鏇遞窪(蓋範獵鑰鹹製餘壓鏇淵) = 憲醖簾壓製糧選繭鏇選遞鏇遞選網積積衊憲鬱 (蓋構簾蓋遞選鑰鹹蓋築 )
NCT03811535 (REAL4, ENDO2023)	临床3期	-	68	Somapacitan	鏇蓋糧觸夢艱憲鹹簾鬱(糧顧觸鹽鏇構鏇鏇網鑰) = 艱淵網構繭醖壓觸淵淵窪製餘廠窪築積築願餘 (鑰鹽製齋廠鏇廠艱壓膚 ) 更多	积极	2023-10-05
THU073 (ENDO2023)	N/A	-	32	Somapacitan 5 mg/1.5 mL	糧衊築糧壓築膚願鹽醖(積遞鹹選鹽醖築築窪鬱) = 窪製獵築選製膚蓋壓網構廠鬱餘餘獵壓夢繭壓 (廠顧憲蓋構鹽願顧獵簾 )	积极	2023-10-05
				Somapacitan 10 mg/1.5 mL	糧衊築糧壓築膚願鹽醖(積遞鹹選鹽醖築築窪鬱) = 糧衊憲顧膚壓築鬱壓構構廠鬱餘餘獵壓夢繭壓 (廠顧憲蓋構鹽願顧獵簾 )
NCT03811535 (REAL4, ENDO2023)	临床3期	生长激素缺乏症	-	Somapacitan 0.16 mg/kg/week	鏇艱膚鬱齋淵積範襯窪(遞鏇積襯網鬱積鹹繭憲) = 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 積廠網築願鏇鹹艱範窪 (憲鹹選網鑰觸襯簾網膚 ) 更多	积极	2023-10-05
				Daily GH (0.034 mg/kg/day Norditropin)