This clinical study aims to evaluate the safety and efficacy of repeated dosing of recombinant human parathyroid hormone (rhPTH[1-84]) in Japanese participants with chronic hypoparathyroidism for a 26-week period.

开始日期2022-08-31

申办/合作机构

Shire Plc

ChiCTR2300067899 / Recruiting临床4期IIT

Clinical study of recombinant human parathyroid hormone in the treatment of osteoradionecrosis of the jaws

开始日期2022-06-30

申办/合作机构-

NCT05137730 / Completed临床1期

A Randomized, Open-label, Single-center, Single-dose Study to Evaluate the Relative Bioavailability of a New Formulation Compared With the Approved Formulation of Recombinant Human Parathyroid Hormone (rhPTH[1-84]) and to Assess Dose Linearity of the New Formulation in Healthy Subjects

The main aim of Part I of this study is to evaluate the relative bioavailability of a new formulation compared with the approved formulation when a single dose of rhPTH(1-84) is given to healthy volunteers. Bioavailability is the ability of a drug to be absorbed and used by the body. In Part II, the main aim is to assess the dose linearity of the new formulation.
Participants will receive 2 doses in Part I and 4 doses in Part II.
Participants need to visit their doctor approximately 14 days and 30 days after the last dose of study drug.

开始日期2021-11-29

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

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100 项与甲状旁腺素(Shire Plc) 相关的临床结果

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100 项与甲状旁腺素(Shire Plc) 相关的转化医学

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100 项与甲状旁腺素(Shire Plc) 相关的专利（医药）

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9,046

项与甲状旁腺素(Shire Plc) 相关的文献（医药）

2024-04-01·Arthroscopy : the journal of arthroscopic & related surgery : official publication of the Arthroscopy Association of North America and the International Arthroscopy Association

Recombinant Human Parathyroid Hormone Biocomposite Promotes Bone-to-Tendon Interface Healing by Enhancing Tenogenesis, Chondrogenesis, and Osteogenesis in a Rabbit Model of Chronic Rotator Cuff Tears

Article

作者: Park, Suk-Hee ; Kim, Yeong Seo ; Han, Jian ; Rhee, Sung Min ; Jeong, Hyeon Jang ; Oh, Joo Han ; Han, Sheng Chen ; Jin, Yong Jun

PURPOSE:

To investigate the effect of recombinant human parathyroid hormone (rhPTH) biocomposite on bone-to-tendon interface (BTI) healing for surgical repair of a chronic rotator cuff tear (RCT) model of rabbit, focusing on genetic, histologic, biomechanical and micro-computed tomography (CT) evaluations.

METHODS:

Sixty-four rabbits were equally assigned to the 4 groups: saline injection (group A), nanofiber sheet alone (group B), rhPTH-soaked nanofiber sheet (nanofiber sheet was soaked with rhPTH, group C), and rhPTH biocomposite (rhPTH permeated the nanofiber sheet by coaxial electrospinning, group D). The release kinetics of rhPTH (groups C and D) was examined for 6 weeks in vitro. Nanofiber scaffolds were implanted on the surface of the repair site 6 weeks after the induction of chronic RCT. Genetic and histologic analyses were conducted 4 weeks after surgery. Furthermore, genetic, histologic, biomechanical, micro-CT, and serologic analyses were performed 12 weeks after surgery.

RESULTS:

In vivo, group D showed the highest collagen type I alpha 1 (COL1A1), collagen type III alpha 1 (COL3A1), and bone morphogenetic protein 2 (BMP-2) messenger RNA (mRNA) expression levels (all P < .001) 4 weeks after surgery; however, there were no differences between groups at 12 weeks postsurgery. After 12 weeks postsurgery, group D showed better collagen fiber continuity and orientation, denser collagen fibers, more mature bone-to-tendon junction, and greater fibrocartilage layer formation compared with the other groups (all P < .05). Furthermore, group D showed the highest load-to-failure rate (28.9 ± 2.0 N/kg for group A, 30.1 ± 3.3 N/kg for group B, 39.7 ± 2.7 N/kg for group C, and 48.2 ± 4.5 N/kg for group D, P < .001) and micro-CT outcomes, including bone and tissue mineral density, and bone volume/total volume rate (all P < .001) at 12 weeks postsurgery.

CONCLUSIONS:

In comparison to rhPTH-soaked nanofiber sheet and the other control groups, rhPTH biocomposite effectively accelerated BTI healing by enhancing the mRNA expression levels of COL1A1, COL3A1, and BMP-2 at an early stage and achieving tenogenesis, chondrogenesis, and osteogenesis at 12 weeks after surgical repair of a chronic RCT model of rabbit.

CLINICAL RELEVANCE:

The present study might be a transitional study to demonstrate the efficacy of rhPTH biocomposites on BTI healing for surgical repair of chronic RCTs as an adaptable polymer biomaterial in humans.

2024-04-01·Bioactive materials

A parathyroid hormone related supramolecular peptide for multi-functionalized osteoregeneration

Article

作者: Wang, Ying ; Guo, Xiaodong ; Chen, Tianhong ; Wang, Junwu ; Wang, Yi ; Hu, Yingkun ; Hao, Zhuowen ; Li, Jingfeng ; Yang, Zhiqiang ; Chen, Renxin ; Li, Hanke ; Chen, Jiayao ; Feng, Qinyu ; Lv, Xuan

Supramolecular peptide nanofiber hydrogels are emerging biomaterials for tissue engineering, but it is difficult to fabricate multi-functional systems by simply mixing several short-motif-modified supramolecular peptides because relatively abundant motifs generally hinder nanofiber cross-linking or the formation of long nanofiber. Coupling bioactive factors to the assembling backbone is an ideal strategy to design multi-functional supramolecular peptides in spite of challenging synthesis and purification. Herein, a multi-functional supramolecular peptide, P1R16, is developed by coupling a bioactive factor, parathyroid hormone related peptide 1 (PTHrP-1), to the basic supramolecular peptide RADA16-Ⅰ via solid-phase synthesis. It is found that P1R16 self-assembles into long nanofibers and co-assembles with RADA16-Ⅰ to form nanofiber hydrogels, thus coupling PTHrP-1 to hydrogel matrix. P1R16 nanofiber retains osteoinductive activity in a dose-dependent manner, and P1R16/RADA16-Ⅰ nanofiber hydrogels promote osteogenesis, angiogenesis and osteoclastogenesis in vitro and induce multi-functionalized osteoregeneration by intramembranous ossification and bone remodeling in vivo when loaded to collagen (Col) scaffolds. Abundant red blood marrow formation, ideal osteointegration and adapted degradation are observed in the 50% P1R16/Col scaffold group. Therefore, this study provides a promising strategy to develop multi-functional supramolecular peptides and a new method to topically administrate parathyroid hormone or parathyroid hormone related peptides for non-healing bone defects.

2024-03-01·Bone

Cortical thickness adaptation to combined mechanical loading and parathyroid hormone treatments is site specific and synergistic in the mouse tibia model

Article

作者: Pickering, Edmund ; Delisser, Peter ; Martelli, Saulo ; Miller, Corey J ; Meakin, Lee B ; Dall'Ara, Enrico ; Pivonka, Peter ; Trichilo, Silvia

In this study, we aimed to quantify the localised effects of mechanical loading (ML), low (20 μg/kg/day), moderate (40 μg/kg/day) or high (80 μg/kg/day) dosages of parathyroid hormone (PTH), and combined (PTHML) treatments on cortical bone adaptation in healthy 19-week old female C57BL/6 mice. To this end, we utilise a previously reported image analysis algorithm on μCT data of the mouse tibia published by Sugiyama et al. (2008) to measure changes in cortical area, marrow cavity area and local cortical thickness measures (ΔCt.Ar, ΔMa.Ar, ΔCt.Th respectively), evaluated at two cross-sections within the mouse tibia (proximal-middle (37 %) and middle (50 %)), and are compared to a superposed summation (P + M) of individual treatments to determine the effectiveness of combining treatments in vivo. ΔCt.Ar analysis revealed a non-linear, synergistic interactions between PTH and ML in the 37 % cross-section that saturates at higher PTH dosages, whereas the 50 % cross-section experiences an approximately linear, additive adaptation response. This coincided with an increase in ΔMa.Ar (indicating resorption of the endosteal surface), which was only counteracted by combined high dose PTH with ML in the middle cross-section. Regional analysis of ΔCt.Th changes reveal localised cortical thinning in response to low dose PTH treatment in the posteromedial region of the middle cross-section, signifying that PTH does not provide a homogeneous adaptation response around the cortical perimeter. We observe a synergistic response in the proximal-middle cross-section, with regions of compressive strain experiencing the greatest adaptation response to PTHML treatments, (peak ΔCt.Th of 189.32, 213.78 and 239.30 μm for low, moderate and high PTHML groups respectively). In contrast, PTHML treatments in the middle cross-section show a similar response to the superposed P + M group, with the exception of the combined high dose PTHML treatment which shows a synergistic interaction. These analyses suggest that, in mice, adding mechanical loading to PTH treatments leads to region specific bone responses; synergism of PTHML is only achieved in some regions experiencing high loading, while other regions respond additively to this combined treatment.

项与甲状旁腺素(Shire Plc) 相关的新闻（医药）

2024-06-21

·赛柏蓝

「百亿大品种」，齐鲁、济川、国为拿下首仿

编者按：本文来自药智数据，赛柏蓝转载，编辑凯西上周（6月10日~6月16日），共60个品种通过/视同通过一致性评价。其中，齐鲁制药有限公司（以下简称“齐鲁制药”）的氯苯唑酸葡胺软胶囊、济川药业集团有限公司（以下简称“济川药业”）的盐酸非索非那定干混悬剂、成都国为生物医药有限公司（以下简称“国为生物”）的骨化三醇注射液等为首仿药物。同期，68个品种申报一致性评价。 01 重磅首仿齐鲁、济川、国为 NMPA官网显示，齐鲁制药的氯苯唑酸葡胺软胶囊、济川药业的盐酸非索非那定干混悬剂、国为生物的骨化三醇注射液均于6月11日获得上市批件。氯苯唑酸葡胺软胶囊齐鲁制药的氯苯唑酸葡胺软胶囊为国内首仿+首家过评，该药物是一款罕见病用药，原研为辉瑞开发的氯苯唑酸（商品名：VYNDAQEL；维达全）。维达全是一款转甲状腺素蛋白稳定剂口服制剂，于2020年2月在中国获批上市，用于治疗转甲状腺素蛋白淀粉样变性多发性神经病（ATTR-PN）I期症状性成人患者，以延缓其周围神经功能损害。 ATTR-PN是一种遗传性罕见致死性神经退行性疾病，常常累及多系统的疾病，主要表现为对称性由肢体远端向近端进展的神经功能障碍，可累及运动、感觉及自主神经，并可伴有心脏、肾脏、眼部等其他脏器受损。ATTR-PN为成年患者发病，约在起病后10年左右，逐渐进展至终末期。由于ATTR-PN的临床特点易与其他临床常见疾病相混淆，加之病例罕见令临床医生对其缺乏认识，获得病理和基因诊断的机会也有限，导致该病的诊断延误。据公开资料，维达全自获批上市以来年销售额持续上升，2023年其全球销售额超过30亿美元（超220亿人民币）。齐鲁制药的氯苯唑酸葡胺软胶囊成功获批上市，有望为国内ATTR-PN患者带来更多选择。盐酸非索非那定干混悬剂济川药业的盐酸非索非那定干混悬剂为国内首个获批干混悬剂型非索非那定的药企，该产品适用于缓解6个月以上儿童和成人的季节性过敏性鼻炎、慢性特发性荨麻疹的相关症状。盐酸非索非那定是一种第二代H1受体拮抗剂，具有良好的抗组胺作用。据报道，此次济川药业获批的干混悬剂型既有固体制剂（颗粒）的特点，又有液体制剂的优势，携带便捷、稳定性好、方便服用，特别适合于儿童和吞咽有困难的老人患者，有助于提高患者用药体验，且每个年龄段人群都有精准的用法用量，更好地指导患者合理用药。此外，该药物采用独特的掩味技术，达到了提升口感的目的。济川药业的盐酸非索非那定干混悬剂获批上市，为国内季节性过敏性鼻炎、慢性特发性荨麻疹患者，尤其儿童和老年患者带来了更多选择。骨化三醇注射液国为生物的骨化三醇注射液为国内首仿、国内独家，用于治疗慢性肾透析患者的低钙血症和/或继发性甲状旁腺功能亢进。公开数据显示，截至2022年底血液透析登记在透患者超过84万人，继发性甲状旁腺功能亢进（SHPT）发病率高达60%以上。骨化三醇是国内外权威指南推荐的SHPT一线用药，备受临床医生认可。国为生物从口服到注射的剂型改良显著提高了药物生物利用度，提升降甲状旁腺激素（PTH）疗效，从而提高血液透析患者用药依从性。国为生物表示，骨化三醇注射液的获批为患者提供了新的治疗选择，同时进一步丰富了其肾科相关产品线。 02 一致性评价通过情况 6月10日~6月16日期间，共60个品种通过/视同通过一致性评价。其中，浙江恒研医药科技有限公司、浙江赛默制药有限公司的利伐沙班细粒剂，成都欣捷高新技术开发股份有限公司、峨眉山通惠制药有限公司的盐酸异丙肾上腺素注射液等产品为企业独家品种。其他品种具体情况如下：表1：6月10日~6月16日，通过一致性评价的品种表2：6月10日~6月16日，视同通过一致性评价的品种 03 一致性评价受理情况 6月10日~6月16日，共68个品种申报一致性评价，具体情况如下：表3： 6月10日~6月16日，一致性评价申报情况资料来源：药智数据企业版——仿制药一致性评价分析系统数据库 END 内容沟通：郑瑶（13810174402）医药代表交流群扫描下方二维码加入银发经济市场机遇交流群扫描下方二维码加入左下角「关注账号」，右下角「在看」，防止失联

上市批准一致性评价

2024-05-13

·BiG生物创新社

占比43% ，“孤儿”已不孤：辉瑞、阿斯利康、强生、诺华如何布局罕见病？

作者｜自由译员Isabelle4月27日，FDA批准Beqvez（fidanacogene elaparvovec-dzkt）用于治疗成人出血性疾病B型血友病。这是辉瑞首个获得FDA批准的基因疗法，也是该适应症继CSL和uniQure的治疗药物Hemgenix后的第二款药物。Hemgenix于2022年获批，以350万美元的单价荣膺彼时全球最贵药物的称号。辉瑞本有机会使Beqvez的定价低于Hemgenix，但最终决定保持350万美元的单价。然而350万的价格已经并非全球最贵药物了——3月，协和麒麟和Orchard Therapeutics研发的基因疗法Lenmeldy上市，用于治疗罕见遗传病异染性脑白质营养不良，以425万美元的标价晋身天价药之王。关于Beqvez的定价，辉瑞表示，“考虑到潜在的长期收益可以抵消当今慢性病的治疗成本，Beqvez可以为卫生系统节约费用，所提价值主张令人信服。 ”2014年，辉瑞开启基因疗法项目，向Spark支付了2,000万美元预付款和2.6亿美元潜在里程碑付款。根据协议，Spark负责一期和二期开发，然后由辉瑞接手。但FDA并非第一个批准Beqvez的机构。1月，加拿大批准将Beqvez应用于基于腺相关病毒（AAV）载体的治疗。这两次批准都许可将Beqvez用于治疗中度至重度B型血友病患者，且这些患者对变异腺相关病毒（AAV）血清型Rh74的中和抗体呈阴性。B型血友病是一种罕见遗传病，患者无法生成凝血因子IX（FIX）的蛋白质，血液无法正常凝固。据世界血友病联盟称，全球患病人数不过38,000人。罕见病是辉瑞六大重点领域之一（其余五个分别是内科、炎症与免疫、疫苗、肿瘤、抗感染），目前辉瑞尚有8款罕见病管线，分别是：a.治疗杜氏肌营养不良的fordadistrogene movaparvovec，处于临床试验二期；b.治疗2-3岁男童杜氏肌营养不良的fordadistrogene movaparvovec，处于临床试验三期；5月1日更新的辉瑞管线c.治疗A型和B型血友病的marstacimab，目前已至注册阶段；d.治疗A型血友病的giroctocogene fitelparvovec，目前处于临床试验三期：e.治疗成人生长激素缺乏症的NGENLATM，目前处于临床试验三期：f.治疗镰刀型细胞贫血症的inclacumab和►osivelotor，目前处于临床试验三期；g.治疗小儿镰刀型细胞贫血症的Oxbryta®，目前处于临床试验三期。 5月1日更新的辉瑞管线 01 阿斯利康：市场药荒，并购正当时3月，阿斯利康购买了法国药企Amolyt Pharma，以获得eneboparatide，这是一种治疗甲状旁腺功能减退的药物，目前处于临床试验三期。甲状旁腺功能减退症是指甲状旁腺不能分泌足够的甲状旁腺激素。这种激素对调节血液中的钙和磷酸盐水平很重要。该病对女性的影响尤其严重。可以通过补充钙和维生素D治疗，但大剂量补充钙可能会引起便秘等肠胃问题。Amolyt提供的治疗方法有所不同，eneboparatide是一种靶向绑定在甲状旁腺激素1型受体上的肽药，天然的甲状旁腺激素也是绑定在这一受体上的。Amolyt表示，该药可能无需每日补充钙和维生素D即可使血钙水平恢复正常，且可以保持骨密度，这对于有较高风险出现骨质疏松症等骨骼问题的病人很重要。Amolyt称最迟今年底可获得初步的三期数据。阿斯利康此次出手时机恰到好处，因为唯一已获得FDA批准的药物停产在即，市场正亟需一款甲状旁腺功能减退症新药。武田的Natpara自2015年获得FDA批准后，商业化之路一直坎坷。2019年，由于Natpara药筒产生的橡胶微粒可能污染注射药品，FDA要求武田召回Natpara。随后三年，武田一直试图重新上市Natpara，但都遭到了FDA的拒绝。2022年，武田表示，计划最迟于2024年停产Natpara。阿斯利康计划于三季度完成对Amolyt的收购，Amolyt将并入阿斯利康的罕见病分部Alexion。目前阿斯利康自身有18款罕见病管线，包括：● 7个LCM（全生命周期管理）项目：a 治疗地图样萎缩的danicopan；b 治疗造血干细胞移植相关血栓性微血管病、全身性重症肌无力、增殖性狼疮性肾炎、免疫球蛋白A（IgA）型肾病的Ultomiris；c 治疗心脏手术相关急性肾损伤的Ultomiris ARTEMIS；d 治疗视神经脊髓炎谱系疾病的Ultomiris CHAMPION-NMOSD。● 6个三期临床试验项目：a 治疗转甲状腺素蛋白淀粉样变性心肌病的acoramidis和ALXN2220；b 治疗淀粉样蛋白轻链淀粉样变性的anselamimab；c 治疗碱性磷酸酶过少的efzimfotase alfa；d 治疗全身性重症肌无力的gefurulimab；e 治疗阵发性夜间血红蛋白尿合并临床显著血管外溶血的Voydeya ALPHA。● 1个二期临床试验项目：治疗增殖性狼疮性肾炎或IgA型肾病的vemircopan。● 4 个一期临床试验项目：a 改善骨代谢的ALXN1910；b 治疗肾病的ALXN1920和ALXN2030；c 口服补体因子D抑制剂Mechanism。 02 强生：双药告捷，喜提重症肌无力突破疗法2月，强生公司宣布，旗下抗体疗法nipocalimab在全身型重症肌无力（gMG）关键的三期研究和干燥综合征（SjD）二期研究获得积极顶线成果。Nipocalimab可以阻断特定受体，阻止免疫系统攻击患者的健康细胞。目前，对于SjD，全球尚没有获批的疗法。自身免疫性疾病是指免疫球蛋白G（IgG）抗体攻击身体，引起疾病。在重症肌无力中，抗体攻击乙酰胆碱受体。在胎儿新生儿溶血病（HDFN）中，抗体攻击婴儿的红细胞。Nipocalimab可以绑定受体，使抗体无法被重新使用，从而减少抗体数量。重点是，Nipocalimab可以防止抗体穿过胎盘。所以，有可能仅靠一种疗法就能将致病性IgG抗体引起的所有疾病一网打尽。这些疾病可分为三大类：由致病性自身抗体引起的疾病、自身抗体影响妊娠的疾病、自身抗体引起的常见风湿病。强生称，有了nipocalimab，自己是唯一一家药物开发覆盖这三类疾病的公司。强生目前尚在开发nipocalimab用于治疗胎儿新生儿溶血病和特发性炎性肌病的管线。 03 诺华：首款口服，潜在重磅疗法获批去年12月，FDA批准了诺华的一款口服新药Fabhalta，用于治疗一种罕见且严重的血液病，称为阵发性睡眠性血红蛋白尿症（PNH）。 PNH是由补体系统（免疫系统的一部分）失调引起的，特征是补体系统会过早破坏红细胞，“溶血”会造成贫血、血栓等症状。针对PNH患者进行的临床研究证实Fabhalta有助于提高患者的血红蛋白水平。诺华期望Fabhalta未来十年可以成为重要的业务支柱。公司相信随着Fabhalta的使用扩展至其他补体系统相关疾病，如IgA肾病和C3肾小球病，年销售额最终可高达30亿美元。诺华进行了两次后期试验，分别招募的是接受过和未接受过C5抑制剂（另一种PNH药物）治疗的患者。第一次试验中，在无需输血的前提下，82%服用Fabhalta的受试者每分升血红蛋白稳定增加了2克以上，相比之下，继续用C5抑制剂治疗的患者血红蛋白没有增加。第二次试验的相关数据为78%。基于这两次试验的结果，FDA批准了Fabhalta。目前诺华还有8款罕见病管线，分别是：a.治疗脊髓性肌萎缩症的AVXS-101；b.治疗IgA肾病（此病在欧美算罕见病，在亚洲不算）的EXV811和FUB523；c.治疗C3肾小球病和非典型溶血性尿毒症的Fabhalta；d.治疗胃肠胰腺神经内分泌肿瘤的Lutathera；e.治疗淋巴管畸形的Vijoice；f.治疗多发性硬化的LOU064。以上项目均进行到三期临床试验。写在最后罕见病，又称“孤儿病”，世界卫生组织的定义是患病人数占总人口的0.65‰～1‰的疾病，我国的定义是新生儿发病率小于1/万、患病率小于1/万、患病人数小于14万的疾病。尽管每种病的患病人数少，但是罕见病种类繁多，全球确认的罕见病有7000多种，总患病人数有3亿。这是一个不可忽视、也不应该被忽视的群体。但现实是其中大部分病无药可治。企业研发罕见病药物，有两个绕不过去的困难：投入产出比和寻找足够的受试者。一方面每种疾病受众基数小，另一方面药物研发需要投入的成本一样不少，所以算经济账，企业研发罕见病并不合算，这也是为什么天价药王多出自罕见病药的原因。同时，也是因为受众基数小，企业需要费更多的周折才能找到足够的受试者。但是，尽管有这两重困难，上世纪八十年代末，只有5%的新药是罕见病药，而去年，这个数字已经上升到43%，大型药企的管线中都有罕见病药的身影，“孤儿病”正得到越来越多的重视。相信随着政策扶持、技术进步，会有越来越多的人负担得起治疗。毕竟，任何药物，都不应该是富人的专属；毕竟，我们生来平等，而生命，本无价。参考资料：Fierce Pharma: Pfizer scores FDA nod for hemophilia B gene therapy, will charge $3.5M per doseMedCity News: AstraZeneca Acquisition Brings Drug to Fill a Rare Disease Gap Left by TakedaBiophara Dive: Novartis gets FDA approval of closely watched rare disease drugPharmaVoice: J&J’s growing rare disease focus brings a potential multi-use treatment to the table辉瑞、阿斯利康、强生、诺华、Fabhalta官网 BiG 十周年预告本次BiG十周年庆典，将首次分为国内和国际篇章，以原创科学和临床需求为出发点，讨论创新技术平台和创新疗法（PROTACT/分子胶、双抗/ADC、siRNA/ASO、CGT、AI制药）的重大进展和发展前景，十年一药曙光现，大浪淘沙始见金！▼报名参会会议门票：审核制(免费)；含主论坛+分论坛，不含餐；优先biotech/Pharma、临床、院校科学家共建Biomedical创新生态圈！如何加入BiG会员？

临床2期临床3期孤儿药基因疗法引进/卖出

2024-05-13

·BioSpace

FDA Action Alert: Dynavax, Ascendis and BMS

Pictured: A scientist with pill bottles in front of FDA headquarters/Taylor Tieden for BioSpace May will be relatively slow for the FDA, with only four big target action dates. Among other announcements, the regulator is scheduled to release its verdicts on a hepatitis B vaccine for adults on hemodialysis and two potential indications for BMS's CAR-T therapy Breyanzi. Read below for more. Dynavax Proposes Hepatitis B Vaccine for Patients on Hemodialysis Today is the day by which the FDA is scheduled to publish its decision on Dynavax Technologies’ sBLA seeking to expand the label of its hepatitis B vaccine Heplisav-B for use in adults on hemodialysis. Heplisav-B won its initial U.S. approval in 2017 for the prevention of hepatitis B, caused by all known subtypes of the virus, in adults aged 18 years and above. According to its label, Heplisav is a recombinant protein vaccine comprised mainly of the hepatitis B surface antigen, which can prime the body to protect against the virus’ infection. Heplisav-B is designed to be administered in two doses, and is the only approved hepatitis B shot that completes the vaccination series in one month. The U.K.’s Medicines and Healthcare products Regulatory Agency granted Heplisav-B Marketing Authorization in February 2023. Dynavax reported that Heplisav-B brought in $213 million in net revenues in 2023, representing a 69% year-over-year increase. As of the end of December 2023, Heplisav-B controlled approximately 42% of the U.S. hepatitis B vaccine market, up from 35% at the end of 2022. In a statement accompanying the company’s full-year results, CEO Ryan Spencer said that Heplisav-B saw “record revenue growth” in 2023 and became the “market share leader in two largest growth segments.” These achievements will help Dynavax establish Hepliav-B as “the leading vaccine in the U.S. adult hepatitis B vaccine market,” he said. The company expects continued growth for Heplisav-B in 2024, anchored by the upcoming potential approval. Dynavax forecasts net product revenues of $265 million to $280 million for Heplisav-B this year. Ascendis Tries Again for Hypoparathyroidism Approval for TransCon PTH The FDA is scheduled on May 14 to release its verdict on Ascendis Pharma’s resubmission for its investigational drug TransCon PTH (palopegteriparatide), which is being proposed as a treatment for adults with hypoparathyroidism. Designed to be orally available and formulated to be long-acting, TransConPTH is a prodrug of the parathyroid hormone that works by restoring physiologic levels of the hormone for 24 hours each day. This mechanism of action could potentially allow it to counter hypoparathyroidism, a rare disorder characterized by the insufficiency of parathyroid hormone, in turn leading to low calcium and high phosphate levels. Patients with hypoparathyroidism experience weakness, muscle cramps and headaches, and the condition can lead to long-term complications such as calcium deposits in the eyes, brain and kidneys. Currently, hypoparathyroidism is managed through high-dose therapy with calcium and active vitamin D, which does not optimally control the condition. Ascendis first submitted a drug application for TransCon PTH in 2022, which the FDA rejected in May 2023, citing manufacturing concerns. The regulator did not identify problems with the drug’s safety or efficacy, and it did not ask for additional studies. Currently, TransCon PTH is approved in the U.K. and European Union, where it is marketed under the brand name Yorvipath. BMS Awaits Two Verdicts for CAR-T Therapy Breyanzi To close out the month, BMS is looking at two target action dates for its CAR-T therapy Breyanzi (lisocabtagene maraleucel): May 23 for relapsed or refractory follicular lymphoma (FL) and May 31 for relapsed or refractory mantle cell lymphoma (MCL). In FL, Breyanzi is backed by the Phase II single-arm TRASCEND FL study, which, with 213 enrolled patients, is the largest clinical trial so far to assess a CAR T therapy in relapsed or refractory indolent non-Hodgkin lymphoma, including FL. BMS presented data from TRANSCEND FL in December 2023, showing that Breyanzi elicited a 95.7% complete response rate in patients with high-risk relapsed or refractory FL when used in the second-line setting. Median progression-free survival had not been reached at the time. Meanwhile, BMS is backing Breyanzi’s MCL application with findings from the MCL cohort of the TRANSCEND NHL 001 study. Published in December 2023 in the Journal of Clinical Oncology, results from the pivotal Phase I trial demonstrated significant and clinically meaningful treatment responses in heavily pre-treated patients, most of whom achieved a complete response. Breyanzi is a CAR-T cell therapy that works by targeting the CD19 surface protein, which is commonly expressed on B cells. Once bound to CD19, Breyanzi proliferates, induces the release of proinflammatory cytokines and cell death in cancer cells. The FDA first approved Breyanzi in February 2021 for relapsed or refractory B-cell lymphoma. In March 2024, Breyanzi picked up two new approvals, one for chronic lymphocytic leukemia and the other for small lymphocytic leukemia. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

上市批准临床结果临床2期临床1期疫苗

100 项与甲状旁腺素(Shire Plc) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	甲状旁腺素(Shire Plc)	H05AA03	DB05829

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
甲状旁腺功能减退症	欧盟	Takeda Pharmaceuticals International AG	2017-04-24
甲状旁腺功能减退症	冰岛	Takeda Pharmaceuticals International AG	2017-04-24
甲状旁腺功能减退症	列支敦士登	Takeda Pharmaceuticals International AG	2017-04-24
甲状旁腺功能减退症	挪威	Takeda Pharmaceuticals International AG	2017-04-24
低钙血症	美国	Takeda Pharmaceuticals U.S.A., Inc.	2015-01-23
绝经期后骨质疏松	欧盟	NPS Pharma Holdings Ltd.	2006-04-24
绝经期后骨质疏松	冰岛	NPS Pharma Holdings Ltd.	2006-04-24
绝经期后骨质疏松	列支敦士登	NPS Pharma Holdings Ltd.	2006-04-24
绝经期后骨质疏松	挪威	NPS Pharma Holdings Ltd.	2006-04-24

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
骨质疏松症	临床3期	美国	Shire Plc	2004-01-10
骨质疏松症	临床3期	阿根廷	Shire Plc	2004-01-10
骨质疏松症	临床3期	墨西哥	Shire Plc	2004-01-10
骨折	临床3期	美国	Shire Plc	2000-04-27
骨折	临床3期	阿根廷	Shire Plc	2000-04-27
骨折	临床3期	巴西	Shire Plc	2000-04-27
骨折	临床3期	保加利亚	Shire Plc	2000-04-27
骨折	临床3期	加拿大	Shire Plc	2000-04-27
骨折	临床3期	以色列	Shire Plc	2000-04-27
骨折	临床3期	墨西哥	Shire Plc	2000-04-27

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临床结果

适应症

分期

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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


NCT03324880 (CTgov)	临床4期	甲状旁腺功能减退症	93	Placebo (Placebo)	襯夢鹹憲夢顧鏇選積艱(糧鬱鑰夢襯繭築鹹鹽構) = 齋鬱繭觸鹹壓鏇鹹鏇餘範鹽齋築夢夢壓簾繭觸 (積願遞憲襯醖鹹蓋膚簾, 範築壓鑰簾蓋製網壓積 ~ 鬱襯構願製窪淵繭鹽製) 更多	-	2023-06-09
				rhPTH (1-84) (rhPTH (1-84))	襯夢鹹憲夢顧鏇選積艱(糧鬱鑰夢襯繭築鹹鹽構) = 膚蓋鹽鑰壓餘蓋齋遞遞範鹽齋築夢夢壓簾繭觸 (積願遞憲襯醖鹹蓋膚簾, 蓋鹹繭蓋糧壓壓願餘憲 ~ 夢簾衊淵鏇艱衊築遞衊) 更多
Pubmed	N/A	甲状旁腺功能减退症	72	rhPTH(1-84)	蓋壓鏇繭壓觸觸願醖淵(鏇襯範餘衊壓餘網窪餘) = 廠膚鏇餘鏇積鹹鹹構構齋顧選願簾選淵積壓鹹 (窪範餘選齋壓醖夢鑰鬱 )	积极	2022-08-26
				rhPTH(1-84) (Control)	蓋壓鏇繭壓觸觸願醖淵(鏇襯範餘衊壓餘網窪餘) = 網糧淵鬱艱艱鑰壓鹹觸齋顧選願簾選淵積壓鹹 (窪範餘選齋壓醖夢鑰鬱, 1.67 mL/min/1.73 m^2 per year)
REPLACE (NCT00732615), RELAY (NCT01268098), RACE (NCT01297309) (Pubmed)	N/A	甲状旁腺功能减退症	113	rhPTH(1-84)	鏇獵衊淵齋獵鬱鑰鏇觸(夢蓋壓獵衊獵鬱齋簾觸) = 製範壓淵鬱觸鬱窪淵衊簾齋憲淵壓膚淵築夢鬱 (壓鑰鑰選顧廠觸築夢積 )	-	2022-06-11
				rhPTH(1-84) (Control)	鏇獵衊淵齋獵鬱鑰鏇觸(夢蓋壓獵衊獵鬱齋簾觸) = 憲構淵餘構鹽顧齋夢窪簾齋憲淵壓膚淵築夢鬱 (壓鑰鑰選顧廠觸築夢積 )
MO033 (ERA2022)	N/A	常染色体显性多囊肾病 parathyroid hormone \| vitamin D \| tubular reabsorption of phosphates ... 更多	87	Parathyroid hormone	蓋遞醖鑰網衊範願艱鬱(鬱鑰襯獵壓蓋積獵範壓) = 膚襯夢鑰獵遞鹽膚衊築鹹壓顧積鹹網築簾膚餘 (鏇襯膚簾積鑰鑰淵顧襯, 403 ~ 1184)	积极	2022-05-03
				Parathyroid hormone	蓋遞醖鑰網衊範願艱鬱(鬱鑰襯獵壓蓋積獵範壓) = 醖鏇鹹衊糧餘觸簾衊觸鹹壓顧積鹹網築簾膚餘 (鏇襯膚簾積鑰鑰淵顧襯, 258 ~ 576)
MO541 (ERA2022)	N/A	继发性甲状旁腺功能亢进症 PTH \| calcium \| phosphorus ... 更多	210	Parathyroid Hormone	製衊夢鹽壓願鹽範鏇選(鑰簾糧淵選獵製夢顧鹽) = 壓醖蓋積蓋襯範廠範繭構淵鑰鹹觸膚製獵繭壓 (壓獵衊膚醖願鹽鬱製繭, 667 ~ 1078)	积极	2022-05-03
				Parathyroid Hormone	製衊夢鹽壓願鹽範鏇選(鑰簾糧淵選獵製夢顧鹽) = 鑰廠觸鏇遞選艱選衊製構淵鑰鹹觸膚製獵繭壓 (壓獵衊膚醖願鹽鬱製繭, 225 ~ 400)
ESC2021	N/A	肺性高血压 PTH	20	PTH treatment	選獵範鑰獵繭製網醖網(繭網鏇構觸選構廠繭遞) = 壓淵鬱蓋衊網獵簾網廠遞齋蓋醖夢積鏇壓鑰築 (窪夢鏇顧襯襯襯蓋築繭 )	-	2021-08-27
				Parathyroid hormone	選獵範鑰獵繭製網醖網(繭網鏇構觸選構廠繭遞) = 觸齋鹹顧繭積構鏇餘製遞齋蓋醖夢積鏇壓鑰築 (窪夢鏇顧襯襯襯蓋築繭 )
NCT02910466 (CTgov)	临床4期	甲状旁腺功能减退症	39	rhPTH(1-84)	遞壓襯醖築醖網網鏇糧(廠壓夢艱襯簾糧築鹹鹹) = 壓獵鹹夢獵範壓鹽繭鬱齋願鬱淵窪願簾鏇顧觸 (鏇願齋觸衊繭獵鏇觸簾, 網範選顧遞衊鹽廠蓋齋 ~ 鑰顧製網廠廠夢獵糧憲) 更多	-	2021-07-28
NCT03364738 (PARALLAX, CTgov)	临床3期	甲状旁腺功能减退症	22	rhPTH(1-84)	鹽繭獵網夢餘膚蓋鹹網(膚廠鹹網製鹽淵襯醖糧) = 鹹餘膚鑰壓齋糧構鏇選遞鹽選廠鏇廠選憲顧鹽 (餘夢鹹壓網選鑰鹽願夢, 淵鑰憲鹽蓋壓衊觸製膚 ~ 齋鹽襯鑰鹹淵顧淵積鏇) 更多	-	2021-06-16
ASGCT2021	N/A	甲状旁腺功能减退症	-	Synthetic PTH hormone	夢獵糧積糧簾繭築壓顧(醖築遞憲簾蓋遞範構鏇) = 觸衊選鹹廠觸餘選積窪築願繭鬱蓋餘鹹獵顧繭 (網繭齋鑰憲窪襯願蓋製 )	-	2021-04-27
				Allogeneic cell-based therapy	夢獵糧積糧簾繭築壓顧(醖築遞憲簾蓋遞範構鏇) = 遞願壓憲醖築遞憲製鹽築願繭鬱蓋餘鹹獵顧繭 (網繭齋鑰憲窪襯願蓋製 )