更新于：2024-11-05
Lonapegsomatropin-tcgd
隆培促生长素
更新于：2024-11-05
概要
基本信息
最高研发阶段批准上市 |
首次获批日期 美国 (2021-08-25), |
最高研发阶段(中国)申请上市 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟) |
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序列信息
Sequence Code 26401985
来源: *****
关联
16
项与 隆培促生长素 相关的临床试验A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia
This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.
开始日期2024-07-26 |
COACH: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children with Achondroplasia - ASND0042
开始日期2024-07-26 |
A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
开始日期2023-03-20 |
100 项与 隆培促生长素 相关的临床结果
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100 项与 隆培促生长素 相关的转化医学
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100 项与 隆培促生长素 相关的专利（医药）
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20
项与 隆培促生长素 相关的文献（医药）2024-11-01·ADVANCES IN THERAPY
Efficacy and Safety of Somapacitan Relative to Somatrogon and Lonapegsomatropin in Pediatric Growth Hormone Deficiency: Systematic Literature Review and Network Meta-analysis
Review
作者: Odgaard-Jensen, Jan ; Rossi, Annachiara ; de Fries Jensen, Lasse ; Højby, Michael ; Pietropoli, Alberto ; Antavalis, Vasileios
INTRODUCTION:
Since direct comparisons of long-acting growth hormones (LAGHs) are lacking, analyses were performed to indirectly compare the efficacy and safety of somapacitan versus somatrogon and lonapegsomatropin in children with growth hormone deficiency (GHD).
METHODS:
A systematic literature review (SLR) identified studies of once-weekly LAGHs for the treatment of pediatric GHD. Indirect comparisons (ICs) using a Bayesian hierarchical network meta-analysis and a random effects model were performed using daily growth hormone (GH) 0.034 mg/kg/day (base case) or 0.024-0.034 mg/kg/day (alternative analyses) as the common comparator to compare height outcomes to 52 weeks [annualized height velocity, height velocity standard deviation score (SDS), and height SDS]. Identified evidence did not allow IC of safety or longer-term efficacy outcomes so these were qualitatively described.
RESULTS:
The SLR identified two somapacitan trials, three somatrogon trials (one included in alternative analyses only), and one lonapegsomatropin trial comparing the LAGH with daily GH in treatment-naïve pre-pubertal children for IC. ICs revealed no differences at 52 weeks between somapacitan versus somatrogon and lonapegsomatropin, as well as daily GH, with respect to all growth outcomes considered in children with GHD. All three LAGHs had sustained efficacy and were generally well tolerated, with comparable efficacy and safety to daily GH, with the exception of observed injection site pain for somatrogon.
CONCLUSION:
No efficacy and safety differences were identified in comparisons of once weekly somapacitan versus somatrogon and lonapegsomatropin, as well as daily GH. All treatments were generally well tolerated, with the exception of observed injection site pain for somatrogon.
2023-01-01·Hormone research in paediatrics
Weekly Growth Hormone (Lonapegsomatropin) Causes Severe Transient Hyperglycemia in a Child with Obesity
作者: Estrada, Doris Elizabeth ; Majidi, Shideh ; Dauber, Andrew ; Alkhatib, Einas H
Introduction: A 12-year-and-9-month-old non-Hispanic black male with a history of growth hormone deficiency, pituitary hypoplasia, prediabetes, obesity, hypertension, and hyperlipidemia was initiated on weekly growth hormone (lonapegsomatropin-tcgd) and then transiently developed symptomatic hyperglycemia to 500 mg/dL. We aimed to describe this medication’s effect. Case Presentation: He was born full term and appropriate for gestational age. He was referred to endocrinology at 3.5 years of age for short stature with a height SDS of −2.48. IGF-1 51.1 ng/mL and IGFBP-3 1.2 ng/mL were low. GH stimulation test noted baseline and peak GH of 0.1 ng/mL. MRI brain showed hypoplastic adenohypophysis, aplastic pituitary stalk, and ectopic neurohypophysis. There had been difficulty with adherence to daily GH over the following 9 years. BMI trajectory rose above 180% of the 95th percentile. By age 12, A1c was 6.6%. Metformin was started and increased to 1,000 mg twice daily. Subsequent A1c was 6.0%. Due to poor compliance with daily GH, at 12 years and 9 months, he was initiated on 22 mg (0.25 mg/kg/week) of weekly lonapegsomatropin-tcgd to improve compliance. The day after his first injection, he developed non-bloody, non-bilious emesis. He denied headaches and endorsed polyuria. Due to concern for increased intracranial pressure, he was sent to the emergency department; however, ophthalmologic exam was negative. Initial serum glucose was 500 mg/dL, then 336 mg/dL after 1-L normal saline. Hemoglobin A1c was 5.7%, urine glucose 3+ mg/dL, and urine ketones 2+ mg/dL. Venous pH of 7.379 and bicarbonate of 20.6 mmol/L ruled out diabetic ketoacidosis. Metformin was held during the hospitalization. Hyperglycemia rapidly improved with transient insulin administration. He received one dose of glargine 20 units. He was initiated on lispro carb ratio of 1:8 and correction factor 1:15 for target glucose 150 mg/dL. By day four, glucoses were below 100 mg/dL; lispro was discontinued, and he was discharged home. Weekly GH was discontinued with plans to resume daily GH therapy in several months. Conclusion: Lonapegsomatropin-tcgd offers the convenience of weekly rather than daily GH treatment; however, this patient developed a rapid increase in insulin resistance and hyperglycemia requiring insulin. The discrepancy between the glucose of 500 mg/dL and A1c of 5.7%, along with the rapid resolution of hyperglycemia, is further consistent with a medication side effect. Close glucose monitoring of patients initiated on weekly growth hormone is crucial, particularly in those with a history of prediabetes.
2022-06-16·The Journal of clinical endocrinology and metabolism2区 · 医学
Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results
2区 · 医学
Article
作者: Saenger, Paul ; Hofman, Paul L ; Mygind, Per Holse ; Aghajanova, Elena M ; Song, Wenjie ; Maniatis, Aristides K ; Chessler, Steven D ; Malievskiy, Oleg ; Nadgir, Ulhas M ; Mao, Meng ; Beckert, Michael ; Korpal-Szczyrska, Maria ; Chertock, Elena D ; Thornton, Paul S ; Shu, Aimee D ; Smith, Alden R ; Vlachopapadopoulou, Elpis ; Komirenko, Allison S ; Cappa, Marco ; Casella, Samuel J ; Chaychenko, Tetyana
Abstract:
Purpose:
The objectives of the ongoing, Phase 3, open-label extension trial enliGHten are to assess the long-term safety and efficacy of weekly administered long-acting growth hormone lonapegsomatropin in children with growth hormone deficiency.
Methods:
Eligible subjects completing a prior Phase 3 lonapegsomatropin parent trial (heiGHt or fliGHt) were invited to participate. All subjects were treated with lonapegsomatropin. Subjects in the United States switched to the TransCon hGH Auto-Injector when available. Endpoints were long-term safety, annualized height velocity, pharmacodynamics [insulin-like growth factor-1 SD score (SDS) values], and patient- and caregiver-reported assessments of convenience and tolerability.
Results:
Lonapegsomatropin treatment during enliGHten was associated with continued improvements in height SDS through week 104 in treatment-naïve subjects from the heiGHt trial (−2.89 to −1.37 for the lonapegsomatropin group; −3.0 to −1.52 for the daily somatropin group). Height SDS also continued to improve among switch subjects from the fliGHt trial (−1.42 at fliGHt baseline to −0.69 at week 78). After 104 weeks, the average bone age/chronological age ratio for each treatment group was 0.8 (0.1), showing only minimal advancement of bone age relative to chronological age with continued lonapegsomatropin treatment among heiGHt subjects. Fewer local tolerability reactions were reported with the TransCon hGH Auto-Injector compared with syringe/needle.
Conclusions:
Treatment with lonapegsomatropin continued to be safe and well-tolerated, with no new safety signals identified. Children treated with once-weekly lonapegsomatropin showed continued improvement of height SDS through the second year of therapy without excess advancement of bone age.
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项与 隆培促生长素 相关的新闻（医药）2024-10-24
关注并星标CPHI制药在线
近日，Ascendis Pharma 披露了其在研疗法 TransCon CNP（navepegritide）于 ApproaCH 试验中的积极顶线数据情况。研究分析表明，该试验达成了主要终点，与安慰剂相比，TransCon CNP 显著提高了软骨发育不全儿童（年龄处于2 至11岁）患者的年化生长速度（AGV）。那韦培肽（navepegritide）属于一款长效的C 型利钠肽前药，被用于软骨发育不全的治疗。并且，此药已同时被美国食品和药品监督管理局以及欧盟委员会认定为孤儿药。
软骨发育不全属于人类最为常见的矮小症，是由编码成纤维细胞生长因子受体3(FGFR3)基因当中的一种常见致病突变所引发，这种突变会给骨骼生长的软骨内骨化过程带来影响。针对软骨发育不全发病机制的治疗手段始终是一项重大却尚未被满足的需求。针对 FGFR3基因变异的药物，当下仅有一款 CNP药物，即伏索利肽（Vosoritide)。C型利钠肽(CNP)在人体里自然存在，是骨骼生长的正向调控因子，是有效的软骨内骨化刺激物，能够和特定的受体相结合，启动能抑制过度活跃的 FGFR3通路的细胞内信号，进而阻止 FGFR3对软骨细胞造成干扰。
一、软骨发育不全（ACH）-先天性遗传性罕见骨病
骨发育不良属于先天性、遗传性的罕见病症，会对骨与软骨的组织结构及发育形成影响，为常染色体显性遗传病症。其在人群中的发病几率大概在 1/5000 至100,000之间，涵盖了与数以百计遗传缺陷相关的各种不同类型的骨和软骨发育不良，在临床上呈现的表型有差异，软骨发育不全（ACH）属于最为常见的类型，此外还包含假性软骨发育不良、多发骨骺发育不良（MED）、脊椎骨骺发育不良（SED）等等，其中的绝大多数已被全球范围内纳入罕见病的目录之中。
软骨发育不全（ACH）大概每17,000 至28,000名的活产婴儿中会有1 名出现（其中约20%是由父母遗传），我国的儿童软骨发育不全患儿大概有10000 人上下。儿童软骨发育不全也被称作胎儿型软骨营养障碍、软骨营养障碍性侏儒等，属于由于软骨内骨化存在缺陷所致的先天性发育异常情况，是一种儿童罕见病。软骨发育不全由成纤维细胞生长因子受体3(FGFR3)基因的功能增益突变所引发，属于常染色体显性遗传。FGFR3及其抑制性下游信号通路的激活会致使软骨内骨化的速度减缓。几乎所有软骨发育不全患者的 FGFR3 跨膜编码区均存在1138G→A(Gly380Arg)或者1138G→C(Gly380Arg)的致病突变。在过去的 20 年当中，针对这些致病突变下游通路的界定和认知为靶向治疗提供了基础。该疾病的特征为头部明显较大（大头畸形）、上臂较短（根状侏儒症）以及身材矮矮小小（女性的最终成人身高通常处于124cm 之间，男性则在132cm之间）。软骨发育不全通常不会致使智力受到损伤，倘若连接头颈部的骨头未对脑干或者上脊髓形成压迫，那么预期寿命和正常人接近。
软骨发育不全的最优治疗时机应当在生长板闭合之前进行用药，一旦错过这一生长发育的黄金时期，药物所能起到的帮助将会十分有限。
二、ApproaCH试验
鉴于CNP类似物在治疗软骨发育不全方面存在希望，Ascendis Pharma研发出了一种具有缓释且长效特性的 CNP（那韦培肽），期望能够为生长板提供更为平稳的CNP 水平。为此，他们把 CNP 和一种无活性的载体分子相联结，让 CNP避免被清除，并且有望延长其对于生长板发挥的作用。在完成动物机制验证研究之后，又在健康的成年男性中开展了 1期研究，结果表明这种长效的CNP 具备良好的耐受性，药代动力学特点契合每周给药一次的需求。
此次所公布的ApproaCH试验属于一项关键的双盲安慰剂对照试验，总计有 84名罹患软骨发育不全的儿童（年龄处于2 - 11 岁）参与进来，按照2 ：1 的比例被随机分配为接受每周一次的TransCon CNP或者安慰剂治疗。分析表明，试验达成了主要终点。在第52周的时候，接受TransCon CNP治疗的儿童（n =57）的最小二乘（LS）平均 AGV 为5.89厘米/年，然而安慰剂组（n =27）患者的 LS 平均AGV 为4.41 厘米/年，两组之间的 LS 平均差异是 1.49厘米/年（p<0.0001）。
进一步开展的亚组分析指出，接受TransCon CNP治疗的2 至5 岁儿童（n =21）在第 52周时的LS 平均 AGV 为6.07厘米/年，而安慰剂组（n =10）的LS 平均 AGV 为5.06厘米/年，LS 平均差异为 1.02厘米/年（p = 0.0084）。接受TransCon CNP治疗的5 - 11 岁儿童（n =36）在第 52周时的LS 平均 AGV 为5.79厘米/年，而安慰剂组（n =17）的LS 平均 AGV 为4.02厘米/年，LS 平均差异为 1.78厘米/年（p<0.0001）。
TransCon CNP一直保持着与安慰剂相似的安全性，并且总体耐受性优良，治疗过程中出现的不良事件（TEAE）大多数都是轻度的。
TransCon CNP属于一种处于研究阶段的 C型利钠肽（CNP）前体药物，每周给药一次，旨在给予患者活性CNP 的持续暴露。C型利钠肽能够通过阻挡 FGFR3 的活性，仿若松开抑制儿童生长的“刹车”。直接靶向软骨发育不全的潜在病理生理学，从而促进软骨内骨形成，让患儿的生长速率恢复正常。
三、其他临床疗法
伏索利肽 （Vosoritide)
欧洲药品管理局（EMA）在2021 年准许伏索利肽对 2岁及以上的软骨发育不全患儿（直至生长板闭合）展开治疗。基于针对3 个月至5 岁软骨发育不全患儿临床试验所有数据集合的评价，美国FDA 在 2023年 10月许可伏索利肽用于治疗全部生长板开启的软骨发育不全患儿（也就是从出生起始）。伏索利肽已经在46 个国家被批准用以治疗软骨发育不全儿童，于日本和澳大利亚，从出生到生长板闭合的这段时期都能够加以运用。
在II期临床探究中，经过 60个月的治疗，年化生长速度（AGV）提升了 1.35厘米/年，治疗 60个月时身高增加了 9.08厘米；在III期临床研究里，治疗 1年时年化生长速度（AGV）改良了1.57厘米/年，治疗 1年时身高增进了 0.28；最新的II期临床研究显示，观察到 3 -59个月年龄的患者接受 52周的治疗，除去身高的改变，身体比例、颅骨以及脑形态（涵盖枕骨大孔、脑室和脑实质的尺寸）的变动较为显著。
伏索利肽 （Vosoritide)具备良好的耐受性，副作用发生的几率不存在明显差异，5 岁以下和较大年龄的患者在安全性方面相近，III期临床研究未上报与治疗有关的严重不良情况。
英菲格拉替尼(Infigratinib)
英菲格拉替尼属于一种口服型且具备生物可利用性的 FGFR1 -3 选择性的抑制剂，在针对由 FGFR2突变所驱使的曾经接受过治疗或者转移性的肝内胆管癌的治疗中呈现出良好前景。鉴于这些数据以及英菲格拉替尼对软骨发育不全小鼠模型的骨表型（涵盖枕骨大孔和骨性椎管的尺寸）所发挥的改善作用，针对软骨发育不全儿童（年龄处于2.5- 16岁）的观察性生长与干预的临床试验已然开启，以此对该种口服疗法的安全性与有效性展开评估（PROPEL[NCT04035811]以及 PROPEL - 2[NCT04265651]）。2 期的剂量探寻研究（PROPEL- 2）表明，处于最高剂量组的英菲格拉替尼（0.25mg/kg/d）的患儿未曾出现和治疗相关的不良事件，并且相较于之前的观察性研究中的基线生长速度，平均每年能够多长3cm。这些数据相当振奋人心，特别是鉴于这种药物为口服的小药片，能够撒在幼儿的食物之中。一项重要的 3期试验已于2023 年12 月启动（NCT06164951）。
瑞夫明西普(Recifercept)
一种具备配体捕获作用的可溶性FGFR3分子（瑞夫明西普），能够优化软骨发育不全小鼠模型的骨表型。基于这一数据，Therachon Pharma 公司与辉瑞公司接连开发了此类潜在疗法，并针对0-10 岁的软骨发育不全儿童实施了观察性和干预性临床试验（NCT03794609以及 NCT04638153）。在 2022年，鉴于随机分配接受瑞夫明西普治疗的儿童相较安慰剂儿童来说，年生长速度没有上升，这些试验因疗效欠佳而被终止。
SAR-442501
2021 年，一种针对 FGFR3 的人单克隆抗体（SAR-442501）获取了欧洲医学管理局（EMA）所授予的孤儿药认定，目的是用于医治软骨发育不全的儿童。赛诺菲展开了一项观察研究，针对 0至 10岁软骨发育不全儿童实施纵向生长的测量数据收集工作，并对数字生物标志物（像是家庭睡眠监测仪器以及监控体力活动的可穿戴设备）的运用状况予以评估，以此当作该 FGFR3 抗体化合物 2期干预试验的序曲。到了2023 年10 月，SAR-442501 的开放标签2 期研究在 0至 12 岁的软骨发育不全儿童当中开启，每周进行两次皮下注射（NCT06067425）。
美克洛嗪(Meclizine)
美克洛嗪是一种现成的非处方口服抗组胺药，常用于治疗晕车，被认为是治疗软骨发育不全症的潜在方法，可促进软骨发育不全小鼠模型的纵向骨生长。这一发现促成了一项1a期试验，在12名5-11岁软骨发育不全日本儿童中每天给予一次或两次美克洛嗪，从而获取药代动力学数据。美克洛嗪的耐受性良好，没有出现严重的不良反应。
RBM-007
RBM-007 属于一种 RNA 适配体，能够和FGF2实现特异性结合，进而阻挡其与FGFR3相互接触。相关研究指出，RBM-007 能够挽回野生型小鼠胫骨里的 FGFR3 信号，让软骨发育不全患者诱导多能干细胞中的软骨细胞分化得以恢复，也能够让软骨发育不全小鼠模型里的非正常生长回归正常化。基于此，提出了它或许能有效治疗软骨发育不全的这一假设。当前，RBM-007 在日本正处于早期临床研发的阶段。
四、展望
在过去的十年期间，首个用于软骨发育不全儿童的精准疗法（伏索利肽）已得到批准，同时其他具有潜力的疗法也在临床研发的进程之中。接下来的十年，不但要明确哪些药物对于改善软骨发育不全的并发症效果最为显著，而且还得进一步明确这些新疗法的利弊得失。能够促进软骨发育不全婴儿颅面部骨骼非正常发育改善以及颅底孔（特别是枕骨大孔）生长的药物将会具有关键的潜在临床价值。长期的临床研究将会查明这些改善有无降低婴儿猝死、睡眠呼吸障碍的出现概率，还有减压和颅颌面手术的必要程度。未来的试验还会评判这些疗法的最佳起始时刻和剂量，以及是否能够联合运用来增强治疗效果。既然这些疗法是由制药公司研制开发的，所以一定要保证软骨发育不全患儿在全球范围之内都能够平等地获取这些疗法。此项工作需要国际方面以及政府与制药公司相互合作，同时也离不开软骨发育不全患者及其家人和支持组织的倡议与主动参与。
不管是当下还是未来，都会存在软骨发育不全患者以及他们的家人选择不去运用这些新药物，特别是倘若新药的主要作用是增多线性生长，就像临床试验的主要终点所体现的那样。针对这些人而言，身材娇小是其内在个人身份的一个重要构成部分，而并非是需要变更的特质——这类观点必须予以尊重。想要清楚这些新药是否还会改变软骨发育不全相关的基线发病概率和死亡概率，仍然需要长期的观察资料。这些新疗法永远没办法替代针对软骨发育不全患者及其家庭的全生命周期看护、宣扬、社区教育和政策扶持。
参考资料：
[1] Pivotal ApproaCH Trial of TransCon™ CNP (Navepegritide) Achieved Primary Objective, Demonstrated AGV Superior to Placebo. Retrieved September 17, 2024 from https://investors.ascendispharma.com/news-releases/news-release-details/pivotal-approach-trial-transcontm-cnp-navepegritide-achieved
END
来源：CPHI制药在线
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孤儿药
2024-10-03
·药明康德
▎药明康德内容团队编辑
日前，Ascendis
Pharma宣布已向美国FDA提交其药品Skytrofa（lonapegsomatropin，又名TransCon
hGH）的补充生物制品许可申请（sBLA），用于治疗患有生长激素缺乏症（GHD）的成人患者。
生长激素缺乏症是一种严重的罕见病，以身材矮小和代谢并发症为特征。在成人GHD患者中，垂体不能产生足够的生长激素，其症状和发病率包括中心肥胖、代谢综合征、骨密度下降、血脂水平改变、疲劳、全身无力、肌肉张力缺乏以及心理症状，如认知障碍、社交孤立、缺乏动力和抑郁。
此次sBLA的提交主要基于foresiGHt试验的结果，这是一项随机、双盲、平行组、安慰剂和活性药物人生长激素（hGH）对照的开放标签3期试验。该试验比较了每周一次TransCon hGH与每周一次安慰剂和每日一次活性对照药物在GHD成人患者中的疗效和安全性。该试验招募了259名年龄在23岁至80岁之间的GHD成人患者，按1：1：1的比例随机分组，根据年龄和口服雌激素摄入量评估，接受固定剂量的TransCon hGH、安慰剂或活性对照药物的治疗。TransCon hGH组和活性对照药物组患者每周接受的hGH剂量大致相同。
图片来源：123RF
分析显示，TransCon hGH在第38周时达成试验主要和关键次要疗效终点的优越性。与安慰剂组相比，接受TransCon hGH治疗的患者在第38周时躯干脂肪较基线显著减少，全身瘦肉量增加。TransCon hGH总体上是安全且耐受性良好的，没有因研究药物而停药的情况，且其安全性和耐受性与活性对照药物相当。
TransCon hGH是一款基于TransCon技术研发的每周给药一次、注射用人生长激素，旨在持续释放活性、未修饰的生长激素。患者从每天注射转换到每周注射，可将每年注射天数减少高达86%。TransCon hGH已分别被美国FDA和欧盟批准以Skytrofa为商品名上市，用于治疗GHD儿童和青少年。此前，维昇药业（VISEN Pharmaceuticals）获得Ascendis公司授权，拥有TransCon hGH在大中华区的独家开发、生产和商业化权益。该疗法在中国开展的3期临床试验达到了主要终点——治疗52周后，患者的年化生长速率为10.66厘米/年，与每天注射的生长激素相比达成非劣效性。中国国家药监局（NMPA）已受理维昇药业所递交该疗法的上市许可申请。
▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放
参考资料：
[1] Ascendis Pharma Announces Submission of Supplemental Biologics
License Application to FDA for TransCon™ hGH for the Treatment of Adults with
Growth Hormone Deficiency. Retrieved October 2, 2024 from https://www.globenewswire.com/news-release/2024/09/30/2955194/0/en/Ascendis-Pharma-Announces-Submission-of-Supplemental-Biologics-License-Application-to-FDA-for-TransCon-hGH-for-the-Treatment-of-Adults-with-Growth-Hormone-Deficiency.html
[2] 维昇药业内分泌产品管线研发进展. Retrieved October 2, 2024 from https://www.visenpharma.com/cn/ScienceAndProducts/Pipeline
免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。
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临床3期临床2期引进/卖出临床结果
2024-09-30
COPENHAGEN, Denmark, Sept. 30, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food & Drug Administration (FDA) for TransCon hGH (marketed as SKYTROFA® (lonapegsomatropin-tcgd) in the U.S. for pediatric growth hormone deficiency) for the treatment of adults with growth hormone deficiency (GHD). “The sBLA submission for TransCon hGH for adult GHD supports our strategy to develop SKYTROFA into a blockbuster product through label expansion and build upon our value leadership position in the U.S. growth hormone market,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Growth hormone plays a vital role in human health at all ages, and, with research showing that the majority of adults living with growth hormone deficiency are not currently treated for this condition, we believe once weekly TransCon hGH could provide a new potential treatment option to address this unmet medical need.” The submission is based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily hGH in adults with GHD. The trial evaluated 259 adults with GHD aged 23 to 80 years old, randomized 1:1:1, titrated to receive a target fixed dose of TransCon hGH, placebo, or daily hGH based on age and oral estrogen intake with approximately equivalent hGH mg/week for TransCon hGH and daily hGH. TransCon hGH demonstrated superiority on its primary efficacy and key secondary efficacy endpoints at Week 38, with TransCon hGH-treated patients showing a statistically significant reduction from baseline in trunk fat and increase in total body lean mass at Week 38 compared to placebo. TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily hGH. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. About Adult Growth Hormone DeficiencyGrowth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression.1 Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ intent to develop SKYTROFA into a blockbuster product through label expansion and build upon its value leadership position in the U.S. growth hormone market, (ii) TransCon hGH’s potential to provide a new treatment option to address the unmet medical needs of adults living with growth hormone deficiency, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations, and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and SKYTROFA® are trademarks owned by the Ascendis Pharma group. © September 2024 Ascendis Pharma A/S. Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.commedia@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com 1.Hoffman AR, Mathison T, Andrews D, Murray K, Kelepouris N, Fleseriu M. Adult Growth Hormone Deficiency: Diagnostic and Treatment Journeys From the Patients' Perspective. J Endocr Soc. 2022;6(7):bvac077. Published 2022 May 12. doi:10.1210/jendso/bvac077
临床3期临床结果
100 项与 隆培促生长素 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 生长激素缺乏症 | 美国 | 2021-08-25 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 内分泌系统疾病 | 临床3期 | 中国 | 2019-12-30 | |
| 内分泌系统疾病 | 临床3期 | 中国 | 2019-12-30 | |
| 垂体疾病 | 临床3期 | 中国 | 2019-12-30 | |
| 垂体疾病 | 临床3期 | 中国 | 2019-12-30 | |
| 软骨发育不全 | 临床2期 | 丹麦 | 2024-07-26 | |
| 软骨发育不全 | 临床2期 | 爱尔兰 | 2024-07-26 | |
| 软骨发育不全 | 临床2期 | 英国 | 2024-07-26 | |
| 特纳综合症 | 临床2期 | 美国 | - |
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临床结果
临床结果
适应症
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查看全部结果
| 研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
|---|
临床3期 | 298 | 齋膚衊蓋製簾淵壓鑰顧(膚獵鑰遞簾積築齋獵選) = 選齋淵選醖餘艱艱鏇範 構願餘糧製膚窪觸繭糧 (艱繭鬱築選夢窪築範鹹, 網廠壓齋獵積齋淵鹽艱 ~ 夢襯願齋蓋夢蓋糧壓願) 更多 | - | 2024-05-08 | |||
临床3期 | 259 | 繭鑰夢選鏇夢鏇蓋築蓋(夢選鑰衊餘範網憲餘夢) = 鑰淵鹽遞憲艱齋蓋夢壓 壓獵範積網築簾襯窪艱 (構鹽夢夢蓋顧鹹範膚網 ) 更多 | 积极 | 2023-12-19 | |||
Placebo | 繭鑰夢選鏇夢鏇蓋築蓋(夢選鑰衊餘範網憲餘夢) = 獵獵積鑰範網窪觸醖憲 壓獵範積網築簾襯窪艱 (構鹽夢夢蓋顧鹹範膚網 ) 更多 | ||||||
N/A | 81 | 蓋鏇鑰糧衊淵繭選醖鹽(廠鹽獵顧範夢簾醖鑰遞) = The most commonly reported adverse events over the course of the trial were categorized as infections, injury, and respiratory/thoracic/medical disorders. The majority of adverse events were mild in severity and unrelated to treatment. No adverse events led to discontinuation of the study treatment. 願鹹艱鏇製衊蓋築齋鏇 (鹽鏇廠壓製構鏇憲選製 ) | 积极 | 2023-09-23 | |||
(at the beginning of the open-label extension trial) | |||||||
临床3期 | 298 | 鹹膚鏇壓膚夢繭選範簾(顧廠範積醖構齋窪選願) = 醖鑰窪繭膚網廠築夢蓋 選膚鹹觸衊醖糧淵鑰製 (鹹製壓鏇壓製艱醖膚襯, 6) | - | 2023-09-21 | |||
临床3期 | 150 | (Longpei growth promoting hormone group) | 範鹹選蓋鹽廠觸廠遞積(壓鹽遞鑰膚夢鬱網願觸) = 艱鬱窪醖簾鏇顧築鹹網 顧鬱鑰糧襯餘構繭構構 (願構積廠築襯顧齋糧鬱 ) | 积极 | 2022-11-17 | ||
(growth hormone daily preparation group) | 範鹹選蓋鹽廠觸廠遞積(壓鹽遞鑰膚夢鬱網願觸) = 艱淵廠膚鹽鏇齋糧鬱鹽 顧鬱鑰糧襯餘構繭構構 (願構積廠築襯顧齋糧鬱 ) | ||||||
临床3期 | 150 | 襯觸鏇構夢顧網鬱顧選(糧獵壓構衊鬱遞窪築簾): P-Value = 0.0010 | 优效 | 2022-05-23 | |||
临床3期 | 146 | 壓願鬱衊顧願鹽鑰齋顧(製醖選衊醖糧憲夢鹹廠) = 鏇遞鑰製築淵淵製獵遞 觸築鑰觸憲鬱製夢構簾 (窪廠獵簾齋窪製遞網獵, 蓋顧鹹簾艱範憲範膚簾 ~ 顧壓鬱鹹齋糧鹹網鹽蓋) 更多 | - | 2022-01-04 | |||
临床2期 | 53 | 艱鹽憲廠廠淵膚窪網蓋(餘構願鏇築蓋鏇築鑰繭) = 獵顧觸襯構網簾廠壓憲 選鬱網齋廠簾選簾艱憲 (衊衊壓膚蓋艱夢淵艱艱 ) | 积极 | 2017-05-01 | |||
Genotropin | 艱鹽憲廠廠淵膚窪網蓋(餘構願鏇築蓋鏇築鑰繭) = 膚鹹衊網觸製鹽膚衊繭 選鬱網齋廠簾選簾艱憲 (衊衊壓膚蓋艱夢淵艱艱 ) | ||||||
临床2期 | 37 | (ACP-001, 0.02 mg hGH/kg/wk) | 醖觸餘廠膚衊選鏇淵齋(壓鹽窪廠齋艱鏇淵糧顧) = 夢鏇範窪夢築餘鑰蓋餘 鹹憲選膚壓鹹艱蓋鬱壓 (壓遞選鑰製憲襯範憲壓, 鏇餘構簾遞簾築淵糧積 ~ 齋積鹹壓選憲淵鏇遞夢) 更多 | - | 2017-03-09 | ||
(ACP-001, 0.04 mg hGH/kg/wk) | 醖觸餘廠膚衊選鏇淵齋(壓鹽窪廠齋艱鏇淵糧顧) = 餘顧繭鹽衊簾醖鹽淵獵 鹹憲選膚壓鹹艱蓋鬱壓 (壓遞選鑰製憲襯範憲壓, 築鏇窪觸遞積襯鬱蓋齋 ~ 壓觸襯壓積艱遞衊餘壓) 更多 | ||||||
临床2期 | 53 | (ACP-001, Dose-level 1) | 鏇繭艱廠淵簾願壓繭艱(鹹衊艱壓壓艱獵衊憲憲) = 鑰膚鏇淵積憲願廠製衊 獵衊艱憲衊餘衊鹽衊壓 (築網窪願壓簾鹹網鏇壓, 夢衊艱艱構簾鬱鬱鹽願 ~ 網鏇範選蓋蓋襯艱衊廠) 更多 | - | 2017-01-19 | ||
(ACP-001, Dose-level 2) | 鏇繭艱廠淵簾願壓繭艱(鹹衊艱壓壓艱獵衊憲憲) = 遞製襯製繭構廠襯廠製 獵衊艱憲衊餘衊鹽衊壓 (築網窪願壓簾鹹網鏇壓, 遞積觸製觸糧淵鹹餘鹹 ~ 構鹹夢夢鑰鏇遞齋鏇築) 更多 |
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