Investigators are recruiting children for a clinical trial using the medication recombinant human IGF-1 (a.k.a. mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT). While IGF-1 is approved by the Food & Drug Administration (FDA) for certain use in children, it is considered an investigational drug in this trial because it has not previously been used to treat RTT. Information from this study will help determine if IGF-1 effectively treats RTT but will not necessarily lead to FDA approval of IGF-1 as a treatment for RTT.

开始日期2013-01-01

申办/合作机构

The Children's Hospital Corp.

[+1]

NCT01525901

/ Completed临床2期IIT

A Double-Blind Placebo-Controlled Crossover Trial of Insulin-Like Growth Factor-1 (IGF-1) in Children and Adolescents With 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)

The purpose of this study is to pilot the use of Insulin-Like Growth Factor-1 (IGF-1) treatment in 22q13 Deletion Syndrome (Phelan-McDermid Syndrome) caused by SHANK3 gene deficiency in order to evaluate safety, tolerability, and efficacy. IGF-1 is an injection under the skin that contains human IGF-1. IGF-1 is approved by the FDA under the brand name Increlex for the treatment of children with short stature due to primary IGF-1 deficiency. It is being used off-label in the current study and is not FDA approved, nor has it yet been studied in humans for the treatment of SHANK3 deficiency.

开始日期2012-02-01

申办/合作机构

Icahn School of Medicine at Mount Sinai

[+1]

NCT01207908

/ Completed临床1/2期IIT

IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy

The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).

开始日期2010-11-01

申办/合作机构

Children's Hospital & Medical Center

[+2]

100 项与美卡舍明生（Ipsen SA）相关的临床结果

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100 项与美卡舍明生（Ipsen SA）相关的转化医学

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项与美卡舍明生（Ipsen SA）相关的文献（医药）

2024-07-01·Ophthalmology

Glycemic Trends in Patients with Thyroid Eye Disease Treated with Teprotumumab in 3 Clinical Trials

Article

作者: Holt, Robert J ; Wester, Sara Tullis ; Kim, Sun ; Smith, Terry J ; Hsu, Kate ; Bhattacharya, Rajib K ; Cavida, Dustin ; Barbesino, Giuseppe ; Fu, Qianhong

PURPOSE:

Assess incidence, severity, and glucose excursion outcomes in thyroid eye disease (TED) patients receiving the insulin-like growth factor-1 receptor inhibitor teprotumumab from 3 clinical trials.

DESIGN:

Analysis of pooled glycemic data over time.

PARTICIPANTS:

Eighty-four teprotumumab- and 86 placebo-treated active TED patients from the phase 2 and phase 3 (OPTIC) controlled clinical trials and 51 teprotumumab-treated patients from the OPTIC extension (OPTIC-X) trial.

METHODS:

Eight intravenous infusions were given over 21 weeks. Phase 2 serum glucose was measured at weeks 1, 4, 15, and 21, with fasting measurements at weeks 1 and 4. Serum glucose was measured at each study visit in OPTIC and OPTIC-X, with fasting measurements at weeks 1 and 4 (in patients without diabetes) or all visits (in patients with diabetes). In all studies, hemoglobin A1c (HbA1c) was measured at baseline, 12, and 24 weeks plus weeks 36 and 48 in OPTIC-X.

MAIN OUTCOME MEASURES:

Serum glucose and HbA1c.

RESULTS:

In the phase 2 and 3 studies, 9 hyperglycemic episodes occurred in 8 teprotumumab patients; mean HbA1c level increased 0.22% from baseline to week 24 (to 5.8%; range, 5.0%-7.9%) versus 0.04% in patients receiving the placebo (to 5.6%; range, 4.6%-8.1%). At study end, 78% (59/76) of teprotumumab patients and 87% (67/77) of patients receiving placebo had normoglycemic findings. Normoglycemia was maintained in 84% (57/68) of patients receiving teprotumumab and 93% (64/69) of patients receiving placebo. Among baseline prediabetic patients, 43% (3/7) remained prediabetic in both groups, and 29% (2/7) of teprotumumab patients and 14% (1/7) of patients receiving placebo had diabetic findings at week 24. OPTIC-X patients trended toward increased fasting glucose and HbA1c whether initially treated or retreated with teprotumumab. Fasting glucose commonly rose after 2 or 3 infusions and stabilized thereafter. Most hyperglycemic incidents occurred in patients with baseline prediabetes/diabetes but were controlled with medication. No evidence was found for progression or increased incidence of hyperglycemia with subsequent doses.

CONCLUSIONS:

Serious glycemic excursions are uncommon in patients with normoglycemia before teprotumumab therapy. Patients with controlled diabetes or impaired glucose tolerance can be treated safely if baseline screening, regular monitoring of glycemic control, and timely treatment of hyperglycemia are practiced.

FINANCIAL DISCLOSURE(S):

Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

2024-02-07·ACS applied materials & interfaces

In Vitro Assessment of Bacterial Adhesion and Biofilm Formation on Novel Bioactive, Biodegradable Electrospun Fiber Meshes Intended to Support Tendon Rupture Repair

Article

作者: Giovanoli, Pietro ; Zinkernagel, Annelies S ; Buschmann, Johanna ; Miescher, Iris ; Calcagni, Maurizio ; Orlietti, Mariano ; Schweizer, Tiziano A ; Snedeker, Jess G ; Rieber, Julia ; Andreoni, Federica ; Meier Buergisser, Gabriella ; Tarnutzer, Andrea

The surgical repair of a ruptured tendon faces two major problems: specifically increased fibrous adhesion to the surrounding tissue and inferior mechanical properties of the scar tissue compared to the native tissue. Bacterial attachment to implant materials is an additional problem as it might lead to severe infections and impaired recovery. To counteract adhesion formation, two novel implant materials were fabricated by electrospinning, namely, a random fiber mesh containing hyaluronic acid (HA) and poly(ethylene oxide) (PEO) in a ratio of 1:1 (HA/PEO 1:1) and 1:4 (HA/PEO 1:4), respectively. Electrospun DegraPol (DP) treated with silver nanoparticles (DP-Ag) was developed to counteract the bacterial attachment. The three novel materials were compared to the previously described DP and DP with incorporated insulin-like growth factor-1 (DP-IGF-1), two implant materials that were also designed to improve tendon repair. To test whether the materials are prone to bacterial adhesion and biofilm formation, we assessed 10 strains of Staphylococcus aureus, Staphylococcus epidermidis, Pseudomonas aeruginosa, and Enterococcus faecalis, known for causing nosocomial infections. Fiber diameter, pore size, and water contact angle, reflecting different degrees of hydrophobicity, were used to characterize all materials. Generally, we observed higher biofilm formation on the more hydrophobic DP as compared to the more hydrophilic DP-IGF-1 and a trend toward reduced biofilm formation for DP treated with silver nanoparticles. For the two HA/PEO implants, a similar biofilm formation was observed. All tested materials were highly prone to bacterial adherence and biofilm formation, pointing toward the need of further material development, including the optimized incorporation of antibacterial agents such as silver nanoparticles or antibiotics.

2023-12-21·JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Frequency and Predictive Factors of Hypoglycemia in Patients Treated With rhIGF-1: Data From the Eu-IGFD Registry

Article

作者: Bossowski, Artur ; Woelfle, Joachim ; Argente, Jesús ; Perrot, Valerie ; Ramon-Krauel, Marta ; Sert, Caroline ; Maghnie, Mohamad ; Polak, Michel ; Bang, Peter ; Mazain, Sarah

Abstract:

Context:

The European Increlex® Growth Forum Database (Eu-IGFD) is an ongoing surveillance registry (NCT00903110) established to collect long-term safety and effectiveness data on the use of recombinant human insulin-like growth factor-1 (rhIGF-1, mecasermin, Increlex) for the treatment of children/adolescents with severe primary insulin-like growth factor-1 deficiency (SPIGFD).

Objective:

This analysis of Eu-IGFD data aimed to identify the frequency and predictive factors for hypoglycemia adverse events (AEs) in children treated with rhIGF-1.

Methods:

Data were collected from December 2008 to May 2021. Logistic regression was performed to identify predictive risk factors for treatment-induced hypoglycemia AEs. Odds ratios (ORs) are presented with 95% CIs for each factor.

Results:

In total, 306 patients were enrolled in the registry; 84.6% were diagnosed with SPIGFD. Patients who experienced ≥ 1 hypoglycemia AE (n = 80) compared with those with no hypoglycemia AEs (n = 224) had a lower mean age at treatment start (8.7 years vs 9.8 years), a more frequent diagnosis of Laron syndrome (27.5% vs 10.3%), and a history of hypoglycemia (18.8% vs 4.5%). Prior history of hypoglycemia (OR 0.25; 95% CI: [0.11; 0.61]; P = .002) and Laron syndrome diagnosis (OR 0.36; 95% CI: [0.18; 0.72]; P = .004) predicted future hypoglycemia AEs. Total hypoglycemia AEs per patient per treatment year was 0.11 and total serious hypoglycemia AEs per patient per treatment year was 0.01.

Conclusion:

Hypoglycemia occurs more frequently in patients with prior history of hypoglycemia and/or Laron syndrome compared with patients without these risk factors, and these patients should be carefully monitored for this AE throughout treatment.

项与美卡舍明生（Ipsen SA）相关的新闻（医药）

2025-02-18

·医药健闻

益普生2024年实现稳健业绩增长，确认2025年全年指引

美通社消息，全球特药领域生物制药公司益普生2月13日公布了2024年全年的财务业绩。以固定汇率计算，2024财年总销售额增长9.9%，或如财务报告显示为8.7%。所有治疗领域的强劲业绩驱动了增长，包括：罕见病产品组合增长67.4%，神经科学领域增长9.2%，肿瘤领域增长7.3%；索马杜林®（兰瑞肽）销售额增长5.6%，除索马杜林外，所有其他产品的销售额实现了两位数增长，增长率为12.2%。 2024财年核心营业利润为11.09亿欧元，增长率为10.8%，核心营业利润率占总销售额的32.6%。益普生首席执行官David Loew表示：“益普生在2024年实现强劲业绩，并推进了研发管线发展，为可持续增长奠定了坚实的基础。随着Iqirvo和蓓尔唯在全球的成功推出、Onivyde在美国的上市以及多项业务拓展交易增加了多项创新资产，我们完全有能力执行我们的战略路线图。今年，我们期待达到关键里程碑，包括长效神经毒素（LANT）的首次数据发表，并进一步扩充和推进我们在所有三个治疗领域的研发管线，为患者带来前景广阔的新药。” 研发管线进展 2024年，实现了重大监管里程碑，包括Onivyde®（伊立替康）获得FDA批准用于一线胰腺导管腺癌（PDAC），以及Iqirvo®（Elafibranor）分别在美国和欧洲获得加速批准。此外，Kayfanda®（Odevixibat）已在欧盟获批用于治疗Alagille综合征（ALGS）。公司还积极开展了Cabometyx®（Cabozantinib）治疗晚期神经内分泌肿瘤（NETs）患者的Ⅲ期研究CABINET，研究结果在2024年欧洲肿瘤内科学会（ESMO）大会上公布，并在《新英格兰医学杂志》上发表。已提交IPN01194（一种ERK抑制剂）的IND申请，预期将该潜在药物推进到临床开发阶段，并针对晚期实体瘤患者开展I/IIa期试验。益普生进一步提升了研发管线的深度和广度，增加了五项具有全球权益和新模式的临床前创新疗法，并与Day One Biopharmaceuticals就晚期肿瘤领域产品Tovorafenib（一种治疗儿童低级别胶质瘤的口服RAF抑制剂）达成许可协议（除美国外）。与Sutro Biopharma和Foreseen Biotechnology签署了两项肿瘤领域抗体偶联药物（ADC）全球许可协议。完成了与Marengo Therapeutics在肿瘤领域的合作关系续期，将新一代精密T细胞接合器TriSTAR纳入合作范围，最近在第四季度与Biomunex就临床前新型T细胞接合器（TCE）签署了一项全球许可协议。本年度还与Skyhawk Therapeutics签署了一项合作协议，开发用于治疗罕见神经系统疾病的RNA调节小分子药物。益普生在2024年撤销了多项投资，包括：将Increlex®（Mecasermin注射液）出售给Eton Pharmaceuticals，以及出售罕见儿科疾病优先审评凭证。环境、社会和治理 2024年，益普生采取了重要步骤，以实施可持续发展战略的宏伟目标。公司继续将可持续发展融入到整个运营过程中。从减少环境足迹到推进患者药物可及性，再到培养强大的企业文化，公司进一步承诺推动患者、员工、社区和地球的进步。公司实现了范围1和范围2温室气体排放量减少45%，范围3温室气体排放量减少25%，完全符合2030年目标（与2019年基线相比）。为使供应商和第三方参与益普生的可持续发展蓝图，做出了巨大努力，包括首次举办“益普生供应商可持续发展日”活动。经过密集转型项目，目前益普生全球99.8%的电力来自可再生能源。通过“未来车队”（Fleet for Future）项目，公司继续推进可持续运输，截至2024年，公司总车队中有43%的车辆为电动汽车。继续致力于领导团队的性别平衡，目前女性在全球领导团队中的占比为55%。 2025年即将达到的里程碑益普生预计将在2025年达到产品组合的几个关键里程碑，包括： Cabometyx®（CABINET试验）-欧洲针对晚期神经内分泌肿瘤（NETs）的注册决策，包括胰腺（pNETs）和胰外（epNETs）神经内分泌肿瘤； Tovorafenib（FIREFLY-1试验）-针对儿童低级别胶质瘤的欧洲注册提交； Fidrisertib（FALKON试验）-进行性骨化性纤维发育不良（FOP）关键性Ⅱb期试验的数据发表； LANT（LANTIC试验）-概念验证数据发表，评价其在美容方面的潜力；这些里程碑巩固了益普生致力于推进创新疗法，为全球患者扩大治疗选择的承诺。 2025年财务指引益普生为2025财年制定了以下财务指引，其中排除了潜在的后期（Ⅲ期临床开发或之后）业务开发交易的任何影响：以固定汇率计算，总销售额增长超过5.0%。基于2025年1月的平均汇率水平，预计汇率将对总销售额产生约1%的有利影响。核心营业利润率超过总销售额的30.0%，其中包括预期的早期和中期外部创新机会产生的额外研发费用。关于销售收入以及核心经营利润的财务指引是基于除索玛杜林之外的产品组合的加速增长以及假设索玛杜林在美国和欧洲会受到更加激烈的仿制品竞争的负面影响。联系美通社 +86-10-5953 9500 info@prnasia.com

临床3期抗体药物偶联物财报临床2期临床申请

2025-02-17

·美通社

在所有治疗领域强劲业绩的推动下，益普生2024年实现稳健业绩增长并确认2025年全年指引

以固定汇率计算 [1] ，2024财年总销售额增长9.9%，或如财务报告显示为8.7%。所有治疗领域的强劲业绩驱动了增长，包括：罕见病产品组合增长67.4%，神经科学领域增长9.2%，肿瘤领域增长7.3%；索马杜林 ® （兰瑞肽）销售额增长5.6%，除索马杜林外，所有其他产品的销售额实现了两位数增长，增长率为12.2% 2024财年核心营业利润为11.09亿欧元，增长率为10.8%，核心营业利润率占总销售额的32.6% 2024年继续扩充研发管线，并获得重要注册批准，增加了几项具有全球权益和创新模式的临床前疗法，及一项后期资产预计2025年将实现四个关键注册和临床里程碑，包括长效神经毒素（LANT）的概念验证数据发表基于除索马杜林外产品组合的销售额加速增长，并假设美国和欧洲的仿制药竞争加剧会对索马杜林销售产生负面影响，以固定汇率计算，2025年的财务指引 [2] 包括总销售额增长大于5.0% [3] ，核心营业利润率大于总销售额的30.0% 巴黎 2025年2月17日 /美通社/ -- 全球特药领域生物制药公司益普生（Euronext: IPN; ADR: IPSEY）2月13日公布了2024年全年的财务业绩。 2024 财年和 2023 财年合并业绩摘录 [4] ： 2024 财年 2023 财年变化百分比 % 百万欧元百万欧元实际 CER [1] 总销售额 3,400.6 3,127.5 8.7 % 9.9 % 核心营业利润 1,109.4 1,001.0 10.8 % 核心营业利润率 32.6 % 32.0 % +0.6pts 核心合并净利润 857.8 765.5 12.1 % 每股核心收益（全面摊薄） €10.27 €9.15 12.3 % 国际财务报告准则（ IFRS ）经营利润 496 .7 [5] 816 .0 -39. 1 % 国际财务报告准则经营利润率 14.6 % 26.1 % -11.5pts 国际财务报告准则合并净利润 347.3 [5] 647.2 -46.3 % 国际财务报告准则每股收益（全面摊薄） €4.15 [5] €7.73 -46.3 % 每股股息 [6] €1.40 [7] €1.20 16.7 % 自由现金流 774.4 710.9 8.9 % 期末净现金 160.3 65.1 n/a 益普生首席执行官David Loew表示："益普生在2024年实现强劲业绩，并推进了研发管线发展，为可持续增长奠定了坚实的基础。随着Iqirvo和蓓尔唯在全球的成功推出、Onivyde在美国的上市以及多项业务拓展交易增加了多项创新资产，我们完全有能力执行我们的战略路线图。今年，我们期待达到关键里程碑，包括长效神经毒素（LANT）的首次数据发表，并进一步扩充和推进我们在所有三个治疗领域的研发管线，为患者带来前景广阔的新药。" 研发管线进展 2024年，我们实现了重大监管里程碑，包括Onivyde ® （伊立替康）获得FDA批准用于一线胰腺导管腺癌（PDAC），以及Iqirvo ® （Elafibranor）分别在美国和欧洲获得加速批准。此外，Kayfanda ® （Odevixibat）已在欧盟获批用于治疗Alagille综合征（ALGS）。公司还积极开展了Cabometyx ® （Cabozantinib）治疗晚期神经内分泌肿瘤（NETs）患者的Ⅲ期研究CABINET，研究结果在2024年欧洲肿瘤内科学会（ESMO）大会上公布，并在《新英格兰医学杂志》上发表。已提交IPN01194（一种ERK抑制剂）的IND申请，预期将该潜在药物推进到临床开发阶段，并针对晚期实体瘤患者开展I/IIa期试验。益普生进一步提升了研发管线的深度和广度，增加了五项具有全球权益和新模式的临床前创新疗法，并与Day One Biopharmaceuticals就晚期肿瘤领域产品Tovorafenib（一种治疗儿童低级别胶质瘤的口服RAF抑制剂）达成许可协议（除美国外）。与Sutro Biopharma和Foreseen Biotechnology签署了两项肿瘤领域抗体偶联药物（ADC）全球许可协议。完成了与Marengo Therapeutics在肿瘤领域的合作关系续期，将新一代精密T细胞接合器TriSTAR纳入合作范围，最近在第四季度与Biomunex就临床前新型T细胞接合器（TCE）签署了一项全球许可协议。本年度还与Skyhawk Therapeutics签署了一项合作协议，开发用于治疗罕见神经系统疾病的RNA调节小分子药物。益普生在2024年撤销了多项投资，包括：将Increlex ® （Mecasermin注射液）出售给Eton Pharmaceuticals，以及出售罕见儿科疾病优先审评凭证。环境、社会和治理 2024年，益普生采取了重要步骤，以实施可持续发展战略的宏伟目标。公司继续将可持续发展融入到整个运营过程中。从减少环境足迹到推进患者药物可及性，再到培养强大的企业文化，公司进一步承诺推动患者、员工、社区和地球的进步。我们为可持续发展作出的努力在多项环境倡议中得到了认可。公司实现了范围1和范围2温室气体排放量减少45%，范围3温室气体排放量减少25%，完全符合2030年目标（与2019年基线相比）。为使供应商和第三方参与益普生的可持续发展蓝图，我们做出了巨大努力，包括首次举办"益普生供应商可持续发展日"活动。经过密集转型项目，目前益普生全球99.8%的电力来自可再生能源。通过"未来车队"（Fleet for Future）项目，公司继续推进可持续运输，截至2024年，公司总车队中有43%的车辆为电动汽车。我们继续致力于领导团队的性别平衡，目前女性在全球领导团队中的占比为55%。 2025 年即将达到的里程碑益普生预计将在2025年达到产品组合的几个关键里程碑，包括： Cabometyx ® （CABINET试验）-欧洲针对晚期神经内分泌肿瘤（NETs）的注册决策，包括胰腺（pNETs）和胰外（epNETs）神经内分泌肿瘤 Tovorafenib（FIREFLY-1试验）-针对儿童低级别胶质瘤的欧洲注册提交 Fidrisertib （FALKON试验）-进行性骨化性纤维发育不良（FOP）关键性Ⅱb期试验的数据发表 LANT [8] （LANTIC试验）-概念验证数据发表，评价其在美容方面的潜力这些里程碑巩固了益普生致力于推进创新疗法，为全球患者扩大治疗选择的承诺。 2025 年财务指引益普生为2025财年制定了以下财务指引，其中排除了潜在的后期（Ⅲ期临床开发或之后）业务开发交易的任何影响：以固定汇率计算，总销售额增长超过5.0%。基于2025年1月的平均汇率水平，预计汇率将对总销售额产生约1%的有利影响。核心营业利润率超过总销售额的30.0%，其中包括预期的早期和中期外部创新机会产生的额外研发费用。关于销售收入以及核心经营利润的财务指引是基于除索玛杜林之外的产品组合的加速增长以及假设索玛杜林在美国和欧洲会受到更加激烈的仿制品竞争的负面影响。欲获得完整信息，请点击 https://www.ipsen.com/press-releases/ipsen-delivers-solid-results-in-2024-driven-by-strong-performance-across-all-therapeutic-areas-and-provides-guidance-for-2025-3025567/ 查看益普生于2024年2月13日发布的报告。 [1] 以固定汇率（CER）计算，即通过应用前一时期使用的汇率重新计算相关时期的业绩，排除任何外汇影响。 [2] 排除潜在后期（Ⅲ期临床开发或后续开发）外部创新交易的任何影响。 [3] 基于2024年1月的平均汇率水平，预计汇率将对总销售额产生约1%的有利影响。 [4] 合并业绩摘录。公司审计师对简明合并财务报表进行了有限审查。 [5] 包括与Sohonos相关的2.79亿欧元（或2.33欧元/股）减值损失，反映了患者接受率降低后修正销售额减少。 [6] 次年支付的与本财年有关的股息。 [7] 由Ipsen S.A.董事会决定，将于2025年5月21日的年度股东大会上提出。 [8] 长效神经毒素关于益普生益普生是一家全球性的生物制药公司，专注于在肿瘤、罕见病和神经科学三个治疗领域为患者提供革新型药物。我们的研发管线由外部创新推动，并由近100年的开发经验以及位于美国、法国和英国的全球中心提供支持。我们的团队遍布全球40多个国家，在世界各地均有合作伙伴，这使我们能够为100多个国家的患者提供药物。益普生在巴黎（泛欧交易所：IPN）上市并通过赞助的I级美国存托凭证项目（ADR：IPSEY）在美国上市交易。更多信息，请访问 ipsen.com 。关于益普生中国益普生集团于1992年进入中国，2019年在上海设立创新中心，是全球四个研发中心之一。益普生于2021年在上海成立中国区总部，并于2022年根据集团业务变动，同步剥离多元健康业务，专注于特药领域，针对三大疾病领域（肿瘤、罕见病、神经科学），益普生上海创新中心将持续推出创新治疗方案以满足中国患者亟待解决的治疗需求。益普生— 有关前瞻性声明的警示说明本文所含前瞻性声明、目的和目标基于集团的管理战略、当前观点和假设。此类声明涉及已知和未知的风险和不确定性，可能导致实际结果、业绩或事件与本文所预期的大不相同。上述所有风险均可能影响集团在未来实现其财务目标的能力，财务目标是基于现今可用信息在合理的宏观经济条件下设定的。"相信"、"期望"和"期待"等词语和类似表述的使用是为了明确前瞻性声明，包括集团对未来事件的期望，此类事件包括监管文件和决定。此外，本文所述目标的制定未考虑外部增长假设和潜在未来收购，而这两者可能会使指标发生变化。目的的依据是集团认为合理的数据和假设。目标取决于将来可能发生的条件或事实，而不仅取决于历史数据。考虑到某些风险和不确定性的发生，实际结果可能与这些指标有很大出入，明显表现为在早期研发阶段或临床试验中有前景的产品可能最终永远不会投放市场或达到其商业目标，尤其是由于注册或竞争原因。集团必须面对或可能面对来自仿制药的竞争，这可能会转化为市场份额的损失。此外，研发过程涉及多个阶段，每个阶段都涉及重大风险，即集团可能无法实现其目的并被迫放弃就自身已投入大量资金的产品所做的努力。因此，集团不能确定在临床前试验中获得的有利结果是否会在随后的临床试验中得到确认，也不能确定临床试验的结果是否足以证明相关产品的安全性和有效性。不能保证产品将获得必要的注册批准或该产品将被证明在商业上是成功的。如果基本假设证明不准确或确实出现风险或不确定性，则实际结果可能与前瞻性声明中的结果有很大不同。其他风险和不确定性包括但不限于一般行业条件和竞争；一般经济因素，包括利率和货币汇率波动；制药行业法规和医疗保健立法的影响；全球医疗成本抑制趋势；竞争对手获得技术进步、新产品和专利；新产品研发中固有的挑战，包括获得监管部门的批准；集团准确预测未来市场状况的能力；生产困难或延误；国际经济的金融不稳定和主权风险；对集团专利的有效性和创新产品的其他保护的依赖；以及可能面临的诉讼，包括专利诉讼和/或监管诉讼。此外，集团依赖第三方来研发和销售某些产品，这些产品可能会产生大额特许权使用费用；这些合作伙伴的行为可能会对集团的活动和财务结果造成损害。集团不能确定其合作伙伴将履行各自的义务。集团可能无法从这些协议中获得任何利益。集团任何合作伙伴违约均会产生低于预期的收益。此类情况可能会对集团的业务、财务状况或业绩产生负面影响。集团明确声明，除非适用法律有所要求，否则其不承担更新或修订本新闻稿中所包含的任何前瞻性声明、目标或假设的任何义务，以反映此类声明所依据的事件、条件、假设或情况的任何变化，也不会就此作出任何承诺。集团的业务受制于其在法国金融市场监管机构备案的注册文件中概述的风险因素。该文件中所列风险和不确定性并非详尽无遗，建议读者参阅集团网站（ www.ipsen.com ）上集团的2021年注册文件。

财报

2025-02-13

·ipsen.com

Ipsen delivers solid results in 2024, driven by strong performance across all therapeutic areas, and provides guidance for 2025

FY 2024 total sales growth of 9.9% at CER1, or 8.7% as reported, with growth driven by strong performance across all therapeutic areas, including a 67.4% increase in the Rare Diseases portfolio, 9.2% in Neuroscience, and 7.3% in Oncology; Somatuline® (lanreotide) sales grew by 5.6%, while all other products, excluding Somatuline, achieved double-digit sales growth at 12.2% FY 2024 core operating income of €1,109m, growing 10.8% as reported, with core operating margin of 32.6% of total sales Continued pipeline expansion in 2024, with significant regulatory approvals, addition of several preclinical therapies with global rights and innovative modalities, and a late-stage asset Four key regulatory and clinical milestones expected in 2025, including the Proof-of-Concept data readout for the Long-Acting Neurotoxin (LANT) Financial guidance2 for 2025 including total sales growth greater than 5.0%3 at CER, and core operating margin greater than 30.0% of total sales, based on accelerated sales growth of the ex-Somatuline portfolio and assuming negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe PARIS, FRANCE, 13 February 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the full year 2024. “Ipsen delivered solid results and advanced its pipeline in 2024, laying a strong foundation for sustained growth,” said David Loew, Chief Executive Officer, Ipsen. “With the successful global rollout of Iqirvo and Bylvay, and the U.S. launch of Onivyde, alongside multiple business development deals adding several innovative assets, we are well positioned to execute our strategic roadmap. This year, we look forward to achieving key milestones, including the first data readout for the Long-Acting Neurotoxin (LANT), and further expand and progress our pipeline across all three therapeutic areas to bring promising new medicines to patients.” Pipeline Progress Significant regulatory milestones were achieved in 2024, including FDA approval of Onivyde® (irinotecan) for first-line pancreatic ductal adenocarcinoma (PDAC), along with accelerated U.S. approval and European approval for Iqirvo® (elafibranor), respectively. Additionally, Kayfanda® (odevixibat) was approved for Alagille syndrome (ALGS) in the E.U. The company also opted-in for the CABINET Phase III study of Cabometyx® (cabozantinib) in patients with advanced neuroendocrine tumors (NETs), with study results presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in the New England Journal of Medicine. An IND application was filed for IPN01194, an ERK inhibitor, advancing the potential medicine into clinical development with a Phase I/IIa trial in advanced solid tumors. Ipsen improved further the depth and breadth of its pipeline by adding five preclinical innovative therapies with global rights and new modalities, and an ex-U.S. licensing agreement with DayOne Biopharmaceuticals for the late-stage oncology asset tovorafenib, an oral RAF inhibitor for pediatric low-grade glioma. Two global licensing agreements for Antibody Drug Conjugate (ADC) in oncology with Sutro Biopharma and Foreseen Biotechnology were signed. An extension of the oncology partnership with Marengo Therapeutics to include TriSTAR, a next-generation precision T-cell engager was completed, as well as more recently, in the fourth quarter, a global licensing agreement with Biomunex for a preclinical novel T-cell engager (TCE). A collaboration with Skyhawk Therapeutics to develop RNA-modulating small molecules for rare neurological diseases was also signed during the year. Ipsen executed several divestments in 2024, including the sale of Increlex® (mecasermin injection) to Eton Pharmaceuticals and the sale of its rare pediatric disease Priority Review Voucher. Environmental, Social and Governance Ipsen took important steps in 2024 in delivering its ambitious sustainability strategy. The company continued to integrate sustainability across its operations. From reducing its environmental footprint to advancing patient access and fostering a strong workplace culture, the company increased its commitment to driving progress for patients, employees, communities, and the planet. Our sustainability efforts were recognized across multiple environment initiatives. The company achieved a 45% reduction in Scopes 1 & 2 greenhouse gas emissions and a 25% reduction in Scope 3, fully in line with its 2030 targets (versus 2019 baseline). Significant efforts were made to engage suppliers and third parties in Ipsen’s sustainability roadmap including the first-ever “Ipsen Supplier Sustainability Day”. Following an intensive transformation project, 99.8% of Ipsen’s global electricity now comes from renewable sources. Through the Fleet for Future project, the company continues to advance sustainable transportation, with 43% of its total company’s fleet now electric vehicles as of 2024. We remain committed to gender balance in leadership, with women now representing 55% of the Global Leadership Team. 2025 Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in Europe for advanced neuroendocrine tumors (NETs), including pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors Tovorafenib (FIREFLY-1 trial) – Regulatory submission in Europe for pediatric low-grade glioma Fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP) LANT88(LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics These milestones reinforce Ipsen’s commitment to advancing innovative therapies and expanding treatment options for patients worldwide. 2025 Financial Guidance Ipsen has set for FY 2025 the following financial guidance, which excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in January 2025, a favorable effect on total sales of around 1% from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance on total sales and core operating margin is based on accelerated sales growth of the ex-Somatuline portfolio and assumes negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. Consolidated financial statements The Board of Directors approved the consolidated financial statements on 12 February 2025. The consolidated financial statements have been audited and the Statutory Auditors’ report is in the process of being published. Ipsen’s comprehensive audited financial statements will be available in due course on ipsen.com (regulated-information section). Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its Q1 2025 sales on April 16th, 2025. Notes All financial figures are in € millions (€m), unless otherwise noted. The performance shown in this announcement covers the twelve-month period to 31 December 2024 (FY 2024) and the three-month period to 31 December 2024 (Q4 2024), compared to the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), respectively, unless stated otherwise. The commentary is based on the performance in FY 2024, unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts Investors Media 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions.3 Based on the average level of exchange rates in Jan 2024, a favorable effect on total sales of about 1% from currencies is expected. 4 Extract of consolidated results. The Company’s auditors performed a limited review of the condensed consolidated financial statements.5 Including an impairment loss of €279m (or €2,33 /share) related to Sohonos, reflecting lower revised sales following lower patient uptake.6 Dividend related to the current financial year to be paid the following year.7 Decided by the Ipsen S.A. Board of Directors and to be proposed at the annual shareholders’ meeting on 21 May 2025. 8 Long-acting neurotoxin Attachment Ipsen PR_FY 2024 – Results announcement_13022025 FY 2024 total sales growth of 9.9% at CER1, or 8.7% as reported, with growth driven by strong performance across all therapeutic areas, including a 67.4% increase in the Rare Diseases portfolio, 9.2% in Neuroscience, and 7.3% in Oncology; Somatuline® (lanreotide) sales grew by 5.6%, while all other products, excluding Somatuline, achieved double-digit sales growth at 12.2% FY 2024 core operating income of €1,109m, growing 10.8% as reported, with core operating margin of 32.6% of total sales Continued pipeline expansion in 2024, with significant regulatory approvals, addition of several preclinical therapies with global rights and innovative modalities, and a late-stage asset Four key regulatory and clinical milestones expected in 2025, including the Proof-of-Concept data readout for the Long-Acting Neurotoxin (LANT) Financial guidance2 for 2025 including total sales growth greater than 5.0%3 at CER, and core operating margin greater than 30.0% of total sales, based on accelerated sales growth of the ex-Somatuline portfolio and assuming negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe PARIS, FRANCE, 13 February 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the full year 2024. “Ipsen delivered solid results and advanced its pipeline in 2024, laying a strong foundation for sustained growth,” said David Loew, Chief Executive Officer, Ipsen. “With the successful global rollout of Iqirvo and Bylvay, and the U.S. launch of Onivyde, alongside multiple business development deals adding several innovative assets, we are well positioned to execute our strategic roadmap. This year, we look forward to achieving key milestones, including the first data readout for the Long-Acting Neurotoxin (LANT), and further expand and progress our pipeline across all three therapeutic areas to bring promising new medicines to patients.” Pipeline Progress Significant regulatory milestones were achieved in 2024, including FDA approval of Onivyde® (irinotecan) for first-line pancreatic ductal adenocarcinoma (PDAC), along with accelerated U.S. approval and European approval for Iqirvo® (elafibranor), respectively. Additionally, Kayfanda® (odevixibat) was approved for Alagille syndrome (ALGS) in the E.U. The company also opted-in for the CABINET Phase III study of Cabometyx® (cabozantinib) in patients with advanced neuroendocrine tumors (NETs), with study results presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in the New England Journal of Medicine. An IND application was filed for IPN01194, an ERK inhibitor, advancing the potential medicine into clinical development with a Phase I/IIa trial in advanced solid tumors. Ipsen improved further the depth and breadth of its pipeline by adding five preclinical innovative therapies with global rights and new modalities, and an ex-U.S. licensing agreement with DayOne Biopharmaceuticals for the late-stage oncology asset tovorafenib, an oral RAF inhibitor for pediatric low-grade glioma. Two global licensing agreements for Antibody Drug Conjugate (ADC) in oncology with Sutro Biopharma and Foreseen Biotechnology were signed. An extension of the oncology partnership with Marengo Therapeutics to include TriSTAR, a next-generation precision T-cell engager was completed, as well as more recently, in the fourth quarter, a global licensing agreement with Biomunex for a preclinical novel T-cell engager (TCE). A collaboration with Skyhawk Therapeutics to develop RNA-modulating small molecules for rare neurological diseases was also signed during the year. Ipsen executed several divestments in 2024, including the sale of Increlex® (mecasermin injection) to Eton Pharmaceuticals and the sale of its rare pediatric disease Priority Review Voucher. Environmental, Social and Governance Ipsen took important steps in 2024 in delivering its ambitious sustainability strategy. The company continued to integrate sustainability across its operations. From reducing its environmental footprint to advancing patient access and fostering a strong workplace culture, the company increased its commitment to driving progress for patients, employees, communities, and the planet. Our sustainability efforts were recognized across multiple environment initiatives. The company achieved a 45% reduction in Scopes 1 & 2 greenhouse gas emissions and a 25% reduction in Scope 3, fully in line with its 2030 targets (versus 2019 baseline). Significant efforts were made to engage suppliers and third parties in Ipsen’s sustainability roadmap including the first-ever “Ipsen Supplier Sustainability Day”. Following an intensive transformation project, 99.8% of Ipsen’s global electricity now comes from renewable sources. Through the Fleet for Future project, the company continues to advance sustainable transportation, with 43% of its total company’s fleet now electric vehicles as of 2024. We remain committed to gender balance in leadership, with women now representing 55% of the Global Leadership Team. 2025 Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in Europe for advanced neuroendocrine tumors (NETs), including pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors Tovorafenib (FIREFLY-1 trial) – Regulatory submission in Europe for pediatric low-grade glioma Fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP) LANT88(LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics These milestones reinforce Ipsen’s commitment to advancing innovative therapies and expanding treatment options for patients worldwide. 2025 Financial Guidance Ipsen has set for FY 2025 the following financial guidance, which excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in January 2025, a favorable effect on total sales of around 1% from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance on total sales and core operating margin is based on accelerated sales growth of the ex-Somatuline portfolio and assumes negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. Consolidated financial statements The Board of Directors approved the consolidated financial statements on 12 February 2025. The consolidated financial statements have been audited and the Statutory Auditors’ report is in the process of being published. Ipsen’s comprehensive audited financial statements will be available in due course on ipsen.com (regulated-information section). Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its Q1 2025 sales on April 16th, 2025. Notes All financial figures are in € millions (€m), unless otherwise noted. The performance shown in this announcement covers the twelve-month period to 31 December 2024 (FY 2024) and the three-month period to 31 December 2024 (Q4 2024), compared to the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), respectively, unless stated otherwise. The commentary is based on the performance in FY 2024, unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts Investors Media 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions.3 Based on the average level of exchange rates in Jan 2024, a favorable effect on total sales of about 1% from currencies is expected. 4 Extract of consolidated results. The Company’s auditors performed a limited review of the condensed consolidated financial statements.5 Including an impairment loss of €279m (or €2,33 /share) related to Sohonos, reflecting lower revised sales following lower patient uptake.6 Dividend related to the current financial year to be paid the following year.7 Decided by the Ipsen S.A. Board of Directors and to be proposed at the annual shareholders’ meeting on 21 May 2025. 8 Long-acting neurotoxin Attachment Ipsen PR_FY 2024 – Results announcement_13022025

临床2期上市批准引进/卖出临床3期

100 项与美卡舍明生（Ipsen SA）相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	美卡舍明生（Ipsen SA）	H01AC03	DB01277

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
成长不全	加拿大	Ipsen Biopharmaceuticals, Inc.	2020-12-17
原发性胰岛素样生长因子-1 缺乏症	加拿大	Ipsen Biopharmaceuticals, Inc.	2020-12-17
侏儒症	英国	Ipsen SA	2008-12-07
生长障碍	美国	Ipsen Biopharmaceuticals, Inc.	2005-08-30
生长激素缺乏症	美国	Ipsen Biopharmaceuticals, Inc.	2005-08-30
胰岛素样生长因子I缺乏症	美国	Ipsen Biopharmaceuticals, Inc.	2005-08-30

未上市

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适应症	最高研发状态	国家/地区	公司	日期
克罗恩病	临床3期	美国	Tercica, Inc.	2008-10-01
Laron综合征	临床3期	美国	Ipsen SA	1990-01-01
杜氏肌营养不良症	临床2期	美国	Ipsen SA	2010-11-01
糖尿病	临床2期	美国	Tercica, Inc.	-
多发性硬化症	药物发现	美国	Genentech, Inc.	-

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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


NCT00571727 (CTgov)	临床2/3期	Laron综合征	92	Mecasermin	蓋夢構窪餘遞簾構鹽製(顧顧顧糧鏇鬱繭醖構築) = 膚鏇鬱蓋範遞窪鏇範製願鏇顧齋選鬱顧選蓋蓋 (齋觸築願簾窪蓋壓鑰鹹, 遞構鹽鹽網淵壓廠鑰製 ~ 膚網艱鹹艱築構鬱簾選) 更多	-	2023-07-06
NCT00903110 (ESPE2022)	N/A	胰岛素样生长因子I缺乏症 GH levels \| IGF binding protein-3 (IGFBP-3) \| GH binding protein (GHBP) ... 更多	306	mecasermin (Increlex ®)	膚醖餘願衊糧鑰壓憲壓(遞鏇窪願繭壓襯餘獵淵) = 窪衊製鹹遞獵鑰願遞積膚積願夢齋艱獵鬱獵餘 (築膚夢蓋壓夢遞繭簾窪 )	-	2022-09-15
ESPE2022	N/A	insulin receptor gene	-	rhIGF-I	糧選築艱鏇遞觸艱蓋鏇(憲齋繭襯積獵顧壓鹹糧) = 憲遞餘獵製製廠衊齋獵艱構鑰顧網顧鹽構積醖 (餘鑰壓淵鏇遞遞構獵蓋 ) 更多	-	2022-09-15
ESPE2022	N/A	insulin receptor gene	-	metreleptin	鹽餘衊築鏇膚齋廠築壓(觸鬱鬱廠醖齋鏇獵選鑰) = 鏇簾憲憲艱鏇繭構構構淵構獵製襯積願築鏇顧 (窪糧網壓鹽築築鑰憲餘 ) 更多	-	2022-09-15
NCT01525901 (CTgov)	临床2期	Phelan-McDermid 综合征	19	Insulin-Like Growth Factor-1 (IGF-1) (Insulin-Like Growth Factor-1 (IGF-1))	鑰網憲艱醖糧築製餘顧(衊網鹽鹽艱繭顧膚齋鹽) = 鏇膚顧衊願蓋選鬱壓壓築餘鹹範鏇範壓觸齋顧 (膚壓醖窪襯糧襯鹹簾糧, 範網繭淵艱餘選繭鹹築 ~ 遞鹽憲醖鹽願選選願製) 更多	-	2022-05-12
NCT01525901 (CTgov)	临床2期	Phelan-McDermid 综合征	19	Normal Saline (Normal Saline)	鑰網憲艱醖糧築製餘顧(衊網鹽鹽艱繭顧膚齋鹽) = 鹽選醖憲積鬱觸積製憲築餘鹹範鏇範壓觸齋顧 (膚壓醖窪襯糧襯鹹簾糧, 鏇艱獵獵鹹膚淵衊範醖 ~ 襯蓋餘築築繭願鏇糧餘) 更多	-	2022-05-12
NCT00903110 (European Increlex® Growth Forum Database Registry, Pubmed \| Eur J Endocrinol)	N/A	Laron综合征	242	rhIGF1 therapy	網鬱積鑰餘鹹廠網鏇夢(願繭壓繭醖獵製築積醖) = experienced by 65.3% of patients; hypoglycaemia was most common 積壓觸遞鬱蓋積顧鬱築 (齋窪夢夢鹽鹹廠鑰獵觸 )	-	2021-02-01
NCT01207908 (CTgov)	临床1/2期	杜氏肌营养不良症	44	IGF-1 (IGF-1)	艱廠鏇簾鹹築蓋憲廠鏇(網壓膚製簾網鏇膚鑰繭) = 壓觸鑰顧簾製餘鑰淵夢壓遞襯網獵築構選鏇艱 (憲餘範衊顧構糧糧願廠, 構築鬱積願壓餘襯淵選 ~ 製繭獵簾憲窪蓋壓蓋衊) 更多	-	2021-01-20
NCT01207908 (CTgov)	临床1/2期	杜氏肌营养不良症	44	steroid+Prednisone+Deflazacort (Standard Steroid Treatment Alone)	艱廠鏇簾鹹築蓋憲廠鏇(網壓膚製簾網鏇膚鑰繭) = 醖糧簾選衊構製醖鏇觸壓遞襯網獵築構選鏇艱 (憲餘範衊顧構糧糧願廠, 範顧壓鏇獵選積餘憲遞 ~ 壓繭鏇窪膚廠淵製鑰壓) 更多	-	2021-01-20
NCT00764699 (CTgov)	临床2/3期	克罗恩病	3	rhIGF (Increlex)	簾淵願醖醖鑰廠鹹範鏇(艱夢鹹齋鹹鬱鬱憲齋鹹) = 築餘觸廠繭網壓廠膚鏇衊遞觸壓鬱選製遞廠願 (鑰齋齋願遞鹽餘夢窪艱, 夢艱糧願齋構鑰簾網餘 ~ 艱願餘願觸糧餘齋簾膚) 更多	-	2018-03-02
NCT00572156 (CTgov)	临床2期	侏儒症 \| 胰岛素样生长因子I缺乏症	106	NutropinAq® (Somatropin [rDNA origin])	繭醖鹽憲簾顧醖夢繭獵(願顧構獵膚鹹餘積壓餘) = 夢簾夢衊獵襯鏇鏇積觸壓壓淵獵鏇獵醖網簾壓 (淵觸膚醖範膚齋糧壓艱, 鏇構獵襯糧顧夢廠構憲 ~ 範繭夢廠積範繭獵鹹繭) 更多	-	2015-12-15
NCT00903110 (EU-IGFD, ESPE2015)	N/A	原发性胰岛素样生长因子-1 缺乏症 LS	200	Increlex	構夢鬱齋鹽選選築壓憲(廠鏇鬱廠構鑰蓋簾顧窪) = As of 2 October 2014, 61 hypoglycaemic events (27 suspected, 26 verified, eight not specified) were reported in 34/200 patients of the safety population (17.0%), making them the most frequently reported targeted AE. Eight serious AEs of hypoglycaemia were reported in five patients. In three patients, episode(s) occurred following fasting or exercise without food intake. In patients with hypoglycaemia, diagnosis of Laron syndrome (LS) was more common (35.3% vs 10.2%, P <0.001) and they tended to be younger at first Increlex ® intake (median age: 8.9 vs 10.8 years, P =0.165) and to have more often prior history of hypoglycaemia (11.8% vs 4.8%, P =0.133). In the multivariate analysis, only LS was identified as predictive factor for hypoglycaemia (OR (CI 95%): 0.21; (0.09; 0.50)). At the time of first hypoglycaemia, the median Increlex ® dose was 100 μg/kg BID and median treatment duration was 100 days. Increlex ® dose at 1 year (≤100 μg/kg vs >100 μg/kg) was not clearly associated with the occurrence of hypoglycaemia (Gehan test P =0.16) 範襯獵選製獵獵積構鏇 (築顧網觸獵繭觸鹹艱鹹 )	-	2015-10-01
NCT00330668 (CTgov)	临床3期	成长不全 \| 胰岛素样生长因子I缺乏症	114	rh IGF-1 (mecasermin)	糧構積鹹鹹衊艱蓋襯鬱(顧鏇膚構淵顧衊鹽繭遞) = 夢繭窪廠網顧蓋選醖繭壓襯願窪餘蓋淵網築鹹 (艱積壓願鑰鏇顧鹹製廠, 憲範獵膚廠築鬱鑰壓願 ~ 蓋糧網壓醖糧顧餘製願) 更多	-	2011-06-27