Determination of the rhIGF-I/rhIGFBP-3 Dose; Administered as a Continuous Infusion, Required to Establish and Maintain Longitudinal SErum IGF-I Levels within Physiological Levels in Premature Infants, to prevent Retinopathy of PrematurityA Phase II, Randomized Controlled, Assessor-Blind, Dose-Confirming, Pharmacokinetic, Safety and Efficacy, Multicenter Study - ROP Phase-II

开始日期2015-07-21

申办/合作机构-

NCT01777542 / Completed临床2期IIT

Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With Recombinant Human IGF-1(Mecasermin [rDNA] Injection)

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]

100 项与美卡舍明生物类似药（Ipsen SA）相关的临床结果

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100 项与美卡舍明生物类似药（Ipsen SA）相关的转化医学

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100 项与美卡舍明生物类似药（Ipsen SA）相关的专利（医药）

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567

项与美卡舍明生物类似药（Ipsen SA）相关的文献（医药）

2024-01-01·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.

2023-01-01·PloS one

Automation, live-cell imaging, and endpoint cell viability for prostate cancer drug screens.

Article

作者: Martinez, Maria J ; Schürer, Stephan ; Burnstein, Kerry L ; Lyles, Rolando D Z ; Sherman, Benjamin

Androgen deprivation therapy (ADT) is the standard of care for high risk and advanced prostate cancer; however, disease progression from androgen-dependent prostate cancer (ADPC) to lethal and incurable castration-resistant prostate cancer (CRPC) and (in a substantial minority of cases) neuroendocrine prostate cancer (NEPC) is common. Identifying effective targeted therapies is challenging because of acquired resistance to established treatments and the vast heterogeneity of advanced prostate cancer (PC). To streamline the identification of potentially active prostate cancer therapeutics, we have developed an adaptable semi-automated protocol which optimizes cell growth and leverages automation to enhance robustness, reproducibility, and throughput while integrating live-cell imaging and endpoint viability assays to assess drug efficacy in vitro. In this study, culture conditions for 72-hr drug screens in 96-well plates were established for a large, representative panel of human prostate cell lines including: BPH-1 and RWPE-1 (non-tumorigenic), LNCaP and VCaP (ADPC), C4-2B and 22Rv1 (CRPC), DU 145 and PC3 (androgen receptor-null CRPC), and NCI-H660 (NEPC). The cell growth and 72-hr confluence for each cell line was optimized for real-time imaging and endpoint viability assays prior to screening for novel or repurposed drugs as proof of protocol validity. We demonstrated effectiveness and reliability of this pipeline through validation of the established finding that the first-in-class BET and CBP/p300 dual inhibitor EP-31670 is an effective compound in reducing ADPC and CRPC cell growth. In addition, we found that insulin-like growth factor-1 receptor (IGF-1R) inhibitor linsitinib is a potential pharmacological agent against highly lethal and drug-resistant NEPC NCI-H660 cells. This protocol can be employed across other cancer types and represents an adaptable strategy to optimize assay-specific cell growth conditions and simultaneously assess drug efficacy across multiple cell lines.

2021-02-01·European journal of endocrinology1区 · 医学

Effectiveness and safety of rhIGF1 therapy in patients with or without Laron syndrome

1区 · 医学

Article

作者: Polak, Michel ; Sert, Caroline ; Bang, Peter ; Perrot, Valerie ; Woelfle, Joachim

Objective:

The European Increlex® Growth Forum Database Registry monitors the effectiveness and safety of recombinant human insulin-like growth factor-1 (rhIGF1; mecasermin, Increlex®) therapy in patients with severe primary IGF1 deficiency (SPIGFD). We present data from patients with and without a reported genetic diagnosis of Laron syndrome (LS).

Design:

Ongoing, open-label, observational registry (NCT00903110).

Methods:

Children and adolescents receiving rhIGF1 therapy from 10 European countries were enrolled in 2008–2017 (n = 242). The treatment-naïve/prepubertal (NPP) cohort (n = 138) was divided into subgroups based on reported genetic diagnosis of LS (n = 21) or non-LS (n = 117). Multivariate analysis of the NPP-non-LS subgroup was conducted to identify factors predictive of growth response (first-year-height standard deviation score (SDS) gain ≥ 0.3). Assessments included change in height and weight over 5 years and adverse events (AEs).

Results:

Height SDS gain from baseline was greater in the NPP-LS than the NPP-non-LS subgroup after 1 years’ treatment (P < 0.05). In the NPP-non-LS subgroup, 56% were responders; young age at baseline was a positive independent predictive factor (P < 0.001). NPP-non-LS-responders and the NPP-LS subgroup had a similar mean age (6.07 years vs 7.00 years) at baseline and height SDS gain in year 1 (0.64 vs 0.70), although NPP-non-LS-responders were taller (P < 0.001) at baseline. BMI SDS changes did not differ across subgroups. Treatment-emergent AEs were experienced by 65.3% of patients; hypoglycaemia was most common.

Conclusions:

In most NPP children with SPIGFD, with or without LS, rhIGF1 therapy promotes linear growth. The safety profile was consistent with previous studies.

项与美卡舍明生物类似药（Ipsen SA）相关的新闻（医药）

2024-04-01

·CPHI制药在线

从Odevixibat在国内报产，浅谈益普生管线布局

关注并星标CPHI制药在线近日，Albireo/益普生（Ipsen）递交的「Odevixibat胶囊」的5.1类进口申请获CDE受理。Odevixibat是一种每日一次的强效非体循环回肠胆汁酸转运蛋白（IBAT）抑制剂，可以阻断肠胆汁酸经过肠肝循环回流入肝，从而减轻肝脏内及循环内的胆汁酸浓度。目前，Odevixibat（商品名：Bylvay）已在美国获批2项适应症：3个月以上所有亚型的进行性家族性肝内胆汁淤积（PFIC）患者的瘙痒（2021.07）；12个月龄及以上Alagille综合征(ALGS)患者的胆汁淤积性瘙痒症（2023.12）。此外，该药还被开发用于治疗胆道闭锁。 PFIC和ALGS均属于罕见病，Odevixibat是首个获批治疗PFIC的药物，其市场表现并不亮眼，2022年销售额仅有2400万美元。不过随着对罕见病的重视，Odevixibat被企业看好。 Odevixibat由Albireo开发。2021年10月，Jadeite Medicines超1.35亿美元获得该药在日本的开发和商业化授权。2023年3月，益普生通过收购Albireo将该药纳入囊中，收购金额约为9.52亿美元。今年2月，益普生与Jadeite Medicines签订协议，获得Odevixibat在日本的开发和商业化授权。在国内，Odevixibat于2023年11月被CDE纳入优先审评，用于治疗6个月或以上患者的进行性家族性肝内胆汁淤积症（PFIC）。PFIC是由于编码胆汁转运蛋白的基因发生不同突变导致的罕见常染色体隐性遗传病，典型表现是胆汁淤积引起的黄疸和瘙痒。根据编码胆汁转运蛋白基因的不同,PFIC可分为6个亚型,分别由ATP8B1、ABCB11、ABCB4、TJP2、NR1H4和MY05B基因突变引起。目前国内PFIC治疗缺乏特异性药物，临床上首选促胆汁酸排泄药熊去氧胆酸。利福平、消胆胺、胆汁酸螯合剂考来烯胺等也可用于治疗PFIC。期待Odevixibat早日在国内获批，给PFIC患者提供一种新选择。此外，Odevixibat还在国内被开发用于治疗胆道闭锁，目前针对该适应症的临床试验已进入3期阶段。 Odevixibat并不是全球获批的唯一一款IBAT抑制剂，目前全球还批准了另外两款，即氯马昔巴特（maralixibat）和依洛昔巴特(elobixibat)。其中氯马昔巴特于2021年9月被FDA批准用于治疗一岁及以上ALGS患者的胆汁淤积性瘙痒，2024年3月又被FDA批准用于五岁及以上PFIC患者的胆汁淤积性瘙痒症状。依洛昔巴特于2018年1月在日本获批治疗便秘。此外，目前全球还有几款在研IBAT抑制剂，如GSK的linerixibat。linerixibat被开发用于治疗原发性胆汁性胆管炎（PBC）的胆汁淤积性瘙痒，目前已进入3期临床。益普生的罕见病布局益普生是一家专注于肿瘤、罕见病和神经科学领域的全球生物制药公司，在罕见病领域已斩获多款药物，如Increlex（美卡舍明）、Somatuline（兰瑞肽，治疗肢端肥大症和垂体巨人症）、Sohonos（palovarotene，治疗进行性骨化性纤维发育不良）。其中Increlex被批准用于治疗儿童和青少年的严重原发性胰岛素生长因子1缺乏症导致的生长障碍。Sohonos是益普生超13亿美元收购Clementia获得，2023年8月被FDA批准用于减少8岁及以上女性和10岁及以上男性进行性骨化纤维发育不良(FOP)成人和儿科患者的新异位骨化体积。近年来，益普生不断扩张其在罕见病领域的布局： 2021年7月，益普生与IRLAB签署一项授权协议，引进创新多巴胺D3受体拮抗剂mesdopetam的全球开发和推广权益。该药用于治疗帕金森病患者在接受左旋多巴治疗之后出现的运动障碍。 2021年8月，益普生与Exicure就新型球形核酸（SNA）达成合作，共同研究、开发和商业化用于治疗亨廷顿病和天使综合征的SNA。 2021年12月，益普生与GENFIT达成4.8亿欧元的长期战略合作协议，获得后者治疗原发性胆汁性胆管炎（PBC）潜在首创PPAR-α/δ双重激动剂Elafibranor的全球独家开发、生产、商业化权益。目前，Elafibranor治疗二线治疗原发性胆汁性肝硬化（PBC）的新药申请正在欧美接受审查。 2023年1月，益普生斥资9.52亿美元收购Albireo，获得包括Bylvay®（odevixibat）在内的多款药物。益普生的肿瘤领域布局肿瘤药是制药领域最大的细分市场，也是药企积极布局的热门方向。除了罕见病，益普生近年来也积极拓展肿瘤管线： 2021年7月，益普生和BAKX Therapeutics签署8.52亿美元的独家全球合作协议，获得后者Bcl-2抑制剂BKX-001的研究、开发、制造和商业化授权。BKX-001被开发用于治疗白血病、淋巴瘤及实体瘤。 2021年10月，益普生和Accent Therapeutics (Accent)签署一项全球独家合作协议，共同研究、开发、生产和商业化后者的METTL3项目。METTL3是一种RNA修饰蛋白 (RMP)，已被临床前验证是AML的新治疗靶点。 2022年6月，益普生斥资2.47亿美元收购Epizyme，获得包括EZH21抑制剂Tazverik(tazemetostat)和 SETD2抑制剂EZM0414在内的多款药物。 2022年8月，益普生和Marengo Therapeutics达成战略合作，共同将Marengo公司的两款临床前选择性T细胞受体（TCR）激活剂推进临床，交易金额达16亿美元。而且，今年益普生肿瘤领域已迎来新突破，其肿瘤药物Onivyde（伊立替康脂质体注射液）于2024年2月被FDA批准联合5-氟尿嘧啶/亚叶酸+奥沙利铂（NALIRIFOX方案），一线治疗成人转移性胰腺导管腺癌（mPDAC）。总结罕见病是医药领域的小钢炮市场，是益普生等国内外药企布局的热门方向。不过整体来看，益普生在罕见病领域的斩获并不多，且其罕见病产品Increlex、Somatuline销售额持续下滑。肿瘤业务是益普生最大的收入来源，但2023年其前三季度营收也较2022前三季度有所降低。期待益普生斥巨资在罕见病、肿瘤领域的布局早日见到成效，有更多的新产品获批上市。【智药研习社近期课程预告】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~

临床3期并购优先审批引进/卖出申请上市

2023-08-18

·药时代

“石头人”们的首款药物！2次被拒批，这药能上市，真的不容易……

正文共：2467字预计阅读时间：8分钟作者 | 辰公子文章来源 | 药时代公众号2023年8月16日，益普生（Ispen）官网宣布，其创新疗法Sohonos（palovarotene）已获FDA批准上市，用于治疗进行性肌肉骨化症（FOP），降低患者新骨化组织的产生，适用人群为FOP成人患者、8岁以上的女性儿童患者和10岁以上的男性儿童患者。这是该领域的首个且唯一的能延缓疾病进展的药物。现实版“美杜莎诅咒”FOP俗称“石头人”，是一种超级罕见病。由于患者体内参与骨骼和软骨发育的基因出现了问题，导致骨骼形成异常，无论是肌肉、筋膜、肌腱、韧带还是其他结缔组织，都会逐渐钙化、变硬。炎症会使骨化行为加速，而任何外伤都会导致肌肉肿胀发炎，所以患者在平时生活中要尽量避免磕碰，但这依旧不能阻止疾病的进展，毕竟就连感冒引起的炎症，也可能会引发额外的骨化。FOP患者确诊时的中位年龄是5岁，大约30岁以后就会丧失独立进食和进水的能力，需要全职护理，最后患者基本死于胸腔周围骨化引发的呼吸及心脏问题。中位预期寿命仅为56岁。患者的疾病进程一般是先脚趾变形变大，然后是脖子、脊椎和肩膀活动受限，最后肌肉消失变成“石头人”，就像中了现实版的“美杜莎诅咒”一样。palovarotene的获批上市对他们来说是一场拯救，但这场拯救实在是来之不易。13亿美元收购，带来近7亿欧元减值palovarotene的上市之路极为坎坷。这款药物最早由罗氏开发，用于治疗肺部疾病，但在800多名患者身上进行过试验后，结果不甚理想，被罗氏雪藏。随后，Clementia公司注意到了palovarotene在治疗骨骼疾病方面的潜力，于是在2017年7月与罗氏签订协议，获得了该产品的全球权益。一年半后， Clementia被Ipsen以约13亿美元的价格并购，palovarotene也来到了Ipsen手下。由于palovarotene是当时Clementia唯一有价值的核心资产，所以我们也可以认为，Ipsen是以13亿美元的价格买下了这款产品。现在看来，FOP作为发病率仅为1.3人/百万人的超级罕见病，在全球仅有不到一千名患者，这个收购价格明显有些偏高了。但在当时，palovarotene是有两个适应症同时开发，除了超级罕见病FOP外，还在开发多发性骨软骨瘤（MO）适应症，这虽然也是个罕见病，但发病率比FOP高了20倍，是个大得多的市场。然而并购不到一年，palovarotene的开发便出现了问题，由于在儿童患者中发现了早期骨生长板闭合的严重不良事件，FDA叫停了其在FOP和MO中进行的2项临床试验的儿科部分，前途顿时一片黯淡。根据分析师的预测，如果问题不能解决，适应症范围将严重缩小，导致产品的峰值销售额从18.5亿美元降低至2.7亿美元。Ipsen的股价当即下跌超过20%。经过慎重的考量后，Ipsen决定放弃MO的开发，专注于FOP适应症。然而祸不单行，转过年来的第一季度，Ispen就面临着更糟糕的局面——经中期分析发现，palovarotene的3期临床试验不太可能到达到主要终点。临床叫停、适应症缩减、试验濒临失败……连续遇挫直接给Ispen造成了6.67亿欧元的减值。但这还不算完。3次递交申请，终于获批上市尽管临床试验出现了很大的问题，但Ipsen还是努力找出了解决办法。Ipsen最初对palovarotene的预期是——希望其能彻底阻止骨化进程，并将其设为了试验的主要终点。但根据中期分析，palovarotene无法阻止正常骨骼系统之外的骨骼形成，所以这条路被堵死了。不过，经事后分析发现，palovarotene 与“骨骼系统外新骨平均年化形成”下降 62% 相关，即：虽然骨化不能被阻止，却可以被延缓。说明药还是有用的。基于该项事后分析，Ipsen于2021年5月向FDA递交了NDA申请，并获得了优先审评券。可好景不长，3个月后Ipsen便主动撤回了NDA申请，因为FDA告诉他们，现有数据不足以支持批准，还需要“额外的分析和数据评估”。虽然不用重做试验，重新递交申请不需要等待太长的时间，但撤回申请还是意味着已有的审批工作前功尽弃，获批进度继续推后。在满足FDA需求的过程中，Ipsen也在推进产品的全球化，并获得了加拿大的积极认可，在2022年1月于加拿大获批上市，算是解了燃眉之急。当年6月，Ipsen重整旗鼓，再次向FDA递交了NDA申请。FDA非常谨慎，计划召开专家咨询委员会讨论，如果会议获得了积极结果，那么palovarotene获批上市的概率还是很大的。结果就在会议召开前一周，FDA突然取消了该专家咨询委员会的召开计划。会议没了，说明FDA的态度不犹豫了，那么结果会是什么呢？当然是被拒绝，否则也不用等到现在。2022年12月23日，Ipsen收到FDA的完整回复函（CRL）。FDA表示，数据还是不够。Ipsen只好继续整理数据，他们的效率很高，转过年来的3月就第三次向FDA递交了NDA申请。FDA再次发布了召开专家咨询委员会的计划。这次的会议终于没有“中道崩殂”，在2023年6月28日顺利召开。会上，专家以10:4的票数认可了palovarotene是治疗FOP的有效方法，以11:3的高票支持其获益大于风险。2个月后，palovarotene获得了FDA的上市批准。结语虽然palovarotene在FDA这里的结局是美好的，但Ipsen仍然有遗憾，因为就在一个月前，欧洲EMA拒绝了该产品的上市申请。4年时间里，palovarotene经历了太多，临床试验遭到毁灭性打击，适应症大幅缩减，3次递交上市申请，被FDA和EMA两度拒绝……对很多大型药企来说，MO适应症被堵上之后，就注定了该产品无法收回成本，及时止损是最可能的选择。但Ipsen还是紧咬着牙，一步一步把产品推进上市。毕竟，做药得有利润支持，但不能只为了利润，相信Ipsen的开发人员会面带微笑，在未来的日子里，想象着美国400名患者、全球近千名患者及其家人发出的欢声笑语。参考资料：1、Ipsen官网2、36/100万的罕见病！新药Palovarotene有望治疗FOP-医学界罕见病频道3、www.fiercebiotech.com4、其他公开资料诺和诺德中国首届研发日——畅想没有严重慢性疾病的未来【9月6日，药时代直播间，不见不散！】点击这里，与药时代一起快乐学习！

并购

2023-08-17

·BioPharmaDive

FDA clears Ipsen bone drug despite questions about its benefits

Dive Brief:After years of fits and starts, Ipsen won Food and Drug Administration approval to sell the first treatment for a rare disease known as fibrodysplasia ossificans progressiva, or FOP.The FDA cleared the drug, palovarotene, for females who are least 8 years old and males at least 10 years old, Ipsen said Wednesday. Its designed to reduce the formation of bone in tissue that is characteristic of the debilitating condition.Ipsen will sell the medicine under the brand name Sohonos at an average list price of $624,000 a year, though the cost will vary depending on insurance coverage, patient needs and support programs. A company spokeswoman said Ipsen is committed to making the treatment accessible to the approximately 400 people in the U.S. who have the disease.Dive Insight:The road to the U.S. market has been a winding one for Sohonos and Ipsen. Roche originally developed the medicine as a treatment for chronic obstructive pulmonary disease, but it failed in testing. The Canadian biotech Clementia Pharmaceuticals then licensed the medicine and in 2014 won orphan drug and fast track designations from the FDA to help speed development for FOP.The initial studies went well enough to attract the attention of Ipsen, which agreed in 2019 to buy Clementia for as much as $1.3 billion. But clinical trials delivered mixed results for Ipsen, and the FDA rejected the drug in late 2022. Ipsen tried again in March with new analyses of previous research, a gambit that doesnt always fly with regulators.An FDA advisory committee backed the drug in June, even though agency reviewers raised questions about its effectiveness and the potential for the treatment to cause inflammatory flare-ups. The final approval granted Wednesday came with a priority review voucher, which Ipsen can sell or use to speed the review process for another of its own drugs. The vouchers can fetch around $100 million when sold.Regulators in other parts of the world have had mixed views of the treatment. Canada approved the medicine in early 2022 and it also has conditional approval in the United Arab Emirates. But the European Commission last month decided against authorization.Even though FOP is exceedingly rare affecting only about 900 people around the world the condition has also drawn the interest of Regeneron Pharmaceuticals, which is testing a potential competing therapy in a Phase 3 trial. Ipsen also has a different option in Phase 2 that was acquired through the Clementia purchase. '

上市批准临床3期快速通道孤儿药临床2期

100 项与美卡舍明生物类似药（Ipsen SA）相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	美卡舍明生物类似药（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
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

10 条进展最快的记录,

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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
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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临床结果

适应症

分期

评价

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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


ESPE2022	N/A	insulin receptor gene	-	rhIGF-I	醖築選壓願鹽簾觸餘選(醖糧網齋窪壓構鑰壓蓋) = 糧鬱繭齋製築構鏇膚顧簾簾憲餘艱願鹹淵憲網 (鹽淵觸積積襯衊衊築築 ) 更多	-	2022-09-15
				metreleptin	願膚網構壓衊鬱顧鑰膚(餘夢膚願構積繭醖獵衊) = 膚醖艱夢艱鑰遞鏇構鏇構糧構願壓艱鹽範構窪 (餘淵壓願製製鏇積醖選 ) 更多
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 ®)	鹽網範繭膚糧艱憲製鬱(觸窪遞繭顧窪蓋網範鬱) = 膚糧繭繭醖餘鑰糧糧鏇鑰鏇糧製網餘積窪鏇鑰 (簾鏇鬱衊窪範願遞觸糧 )	-	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))	獵網壓齋鑰獵範鹽夢齋(齋觸鏇簾範膚製繭憲糧) = 壓觸壓願構構淵鹹鹽選膚簾糧積遞淵艱艱繭壓 (衊網願蓋繭簾糧鬱網顧, 觸醖鑰鑰艱觸築醖製膚 ~ 廠積鏇蓋淵觸網網鏇構) 更多	-	2022-05-12
				Normal Saline (Normal Saline)	獵網壓齋鑰獵範鹽夢齋(齋觸鏇簾範膚製繭憲糧) = 積衊窪衊範築鬱構衊艱膚簾糧積遞淵艱艱繭壓 (衊網願蓋繭簾糧鬱網顧, 簾鬱衊鬱鬱艱襯窪醖衊 ~ 範獵醖齋夢衊構餘鹹顧) 更多
NCT00903110 (European Increlex® Growth Forum Database Registry, Pubmed \| Eur J Endocrinol)	N/A	Laron综合征	242	rhIGF1 therapy	獵繭遞鑰憲鬱廠願膚鏇(齋製積齋糧廠蓋簾壓鏇) = experienced by 65.3% of patients; hypoglycaemia was most common 積獵鑰築鹹觸壓鹽獵製 (範鹹積遞獵築製餘顧網 )	-	2021-02-01
NCT01207908 (CTgov)	临床1/2期	杜氏肌营养不良症	44	IGF-1 (IGF-1)	蓋構艱繭獵願廠齋網網(鏇壓獵窪繭壓壓齋憲廠) = 衊簾憲淵齋齋範鏇製憲觸鹽簾膚製構鹽選糧衊 (壓選鑰選蓋鑰築衊築淵, 醖顧衊範選簾蓋壓糧簾 ~ 壓膚憲膚衊糧製醖範壓) 更多	-	2021-01-20
				steroid+Prednisone+Deflazacort (Standard Steroid Treatment Alone)	蓋構艱繭獵願廠齋網網(鏇壓獵窪繭壓壓齋憲廠) = 網蓋蓋夢繭蓋壓蓋鑰範觸鹽簾膚製構鹽選糧衊 (壓選鑰選蓋鑰築衊築淵, 鬱夢積築餘築獵憲顧鹽 ~ 壓襯壓選簾築窪構壓築) 更多
EU-IGFD (ESPE2016)	N/A	-	221	Increlex	衊獵鹽遞範蓋遞選夢醖(積獵蓋繭醖窪衊鹽憲選) = 13% vs 11% 觸構願鑰糧襯網鏇齋遞 (膚網製糧蓋選餘淵鏇網 ) 更多	-	2016-09-10
				Increlex
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])	憲範艱醖憲淵築獵齋築(衊獵鑰選夢積鬱構顧鹹) = 鏇願選醖襯觸遞膚鹹壓選艱鏇襯觸膚構遞醖遞 (淵窪糧遞壓餘襯糧鹹製, 繭膚壓範觸齋廠廠艱遞 ~ 網糧製簾餘願醖簾襯淵) 更多	-	2015-12-15
EU-IGFD (ESPE2015)	N/A	LS	200	Increlex	衊蓋製積製夢蓋艱壓築(範糧觸構廠鏇糧糧膚膚) = 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) 鑰糧蓋淵觸餘顧遞鏇淵 (獵鹽廠襯廠獵衊齋艱製 )	-	2015-10-01