A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease

The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement therapy (ERT).
Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.

开始日期2023-01-03

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT03702361

/ Completed临床4期IIT

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study

During the past two years, the investigator has performed succsefully an IIR wherein patients with GD, previously treated with velaglucerase alfa ERT were gradually switched to a 10 minutes (rapid) administration of the same ERT. The success was expressed as safety (no clinically meaningful AEs, no antibodies detected, home therapy), efficacy ("lack of deterioration") and patients' satisfaction. The latter was based not just on specific questionnaires and analog scales, but particularly by the patients' sharing the experience with other patients and consequently repeated requests by many to switch to a rapid administration of their ERT.
Therefore, the investigator is hereby proposing to investigate the safety and efficacy of a 10 minutes administration of velaglucerase alfa in a cohort of treatment-naive patients.
The current VPRIV label is restricted to a dosage of 60 units/kg body weight every other week (60 units/kg EOW) - this dose will be used throughout the study period. The enzyme will be provided by Shire, which will also provide a research grant for the conduction of the trial.

开始日期2018-09-04

申办/合作机构

Shaare Zedek Medical Center

NCT02574286

/ Completed临床4期

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease

The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment.

开始日期2016-06-29

申办/合作机构

Shire Plc

100 项与维拉苷酶α 相关的临床结果

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100 项与维拉苷酶α 相关的专利（医药）

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项与维拉苷酶α 相关的文献（医药）

2025-10-01·WASTE MANAGEMENT

Green coconut husk biochar- a sustainable reductant in ironmaking processes

Article

作者: Mahobia, Girija Shankar ; Sachan, Rehan ; Rao, Lakkoju Sankara ; Mishra, Biswajit

This study explores the potential of green coconut husk biochar (GCB) as an alternative reductant in ironmaking by evaluating its composition, calorific value, and reduction efficiency in comparison to non-coking coal and coke. Pyrolysis of coconut husk at 500 °C for 45 min produced biochar with 65 % fixed carbon, 28 % volatile matter, and low ash content (5.5 %). The gross calorific value (GCV) peaked at 27.4 MJ/kg. Reduction experiments revealed that GCB exhibited faster initial reduction rates at all tested temperatures than both coke and non-coking coal, attributed to its higher volatile and hydrogen content, which promote reducing gas generation. FTIR analysis confirmed the presence of aliphatic C-H bonds and partially aromatic structures, indicating a balance between reactivity and thermal stability. Given its enhanced reduction performance, GCB shows promise not only as a standalone reductant but also as a blending component with conventional coal to improve early-stage reduction kinetics. These findings support the viability of biowaste-derived GCB in promoting more sustainable and efficient ironmaking processes.

2025-10-01·ACTA PSYCHOLOGICA

Enhancing sustainable performance through green human resource management: Green competencies building and green passion playing as a joint moderation

Article

作者: Masud, Abdullah Al ; Islam, Mohammad Fakhrul ; Rahman, Md Kazi Hafizur ; Emon, Md ; Mim, Anika Tasnim ; Ahmed, Selim ; Lipy, Nusrat Sharmin

This study aims to investigate the moderating effect of green competencies building (GCB) and green passion (GP) on the relationship between green human resource management (GHRM) and sustainable performance (SP). Moreover, it aims to find out the joint moderating effect of GCB and GP on the relationship between GHRM and SP. An online survey was used to gather 410 samples from various manufacturing organizations in Bangladesh, and the data was analyzed using structural equation modeling (SEM). The study found that GCB and GP separately and jointly moderate the relationship between GHRM and SP. This study uniquely explores how green competencies and green passion, both individually and jointly, moderate the relationship between GHRM and sustainable performance.

2025-10-01·Indian Journal of Hematology and Blood Transfusion

Diffuse Large B-Cell Lymphoma – Experience from a Tertiary Care Hospital in Eastern India

Article

作者: Sarkar, Swapnodeep ; Dolai, Tuphan Kanti ; Ghosh, Kaustav ; Roy, Shipla

Abstract:

Diffuse large B-cell lymphoma (DLBCL) is the most common and a morphologically, molecularly, and clinically heterogeneous group of non-Hodgkin's lymphoma (NHL) whose outcomes have greatly improved after rituximab-based chemotherapy. The present Indian study is unique as we have analysed the data of DLBCL patients based on cell of origin, clinical features, and outcomes with rituximab-based chemotherapy. In this retrospective observational study from 2016-2023, the medical records of 580 DLBCL patients were analysed. The cell of origin was based on immunohistochemistry using the Hans algorithm. The median age of presentation was 55 years, with the Activated B-cell (ABC) type being the most common. Stage IV disease was observed in 41% of the patients. As per International Prognostic Index (IPI) risk stratification, 38% and 36% of the patients were in the low intermediate (LI) and high intermediate (HI) risk groups, respectively. Extranodal involvement, B symptoms, and bulky disease were seen in 26%, 46%, and 29% of the patients, respectively. R-CHOP chemotherapy was used in 75% of the patients. Complete response was seen in 73% and 43% in the germinal centre B-cell (GCB) type and ABC type, respectively. Other factors associated with lower rates of complete response were female sex, Stage II/IV, IPI-HI/H, Bulky disease, and non-R-CHOP chemotherapy use. The overall two-year PFS rate was 78%, with 87.6% in the GCB and 70.5% in the ABC type, respectively. Two-year overall survival was 81.4% (87.7% for GCB, 73.5% for ABC and 90.1% for others).

Clinical trial registration:

Nil.

项与维拉苷酶α 相关的新闻（医药）

2025-10-20

·有驾

遗传性疾病治疗药物概览：从范可尼贫血到甲基丙二酸血症

本文旨在为读者提供关于范可尼贫血(FA)、戈谢病(GD)、先天性肾上腺皮质增生症(CAH)、原发性肉碱缺乏症(PCD)以及甲基丙二酸血症(MMA)等病症的最新治疗或缓解药物信息。请注意，本文所提供的药物信息仅供参考，并非临床用药依据。若涉及相关病症，请务必及时就医并遵循专业医生的建议。 1.1 【羟甲烯龙的作用】羟甲烯龙属于蛋白同化激素类药物，它能够促进蛋白质的合成并抑制其异生，有助于降低血胆固醇、减少钙磷的排泄，并缓解骨髓抑制，进而促进发育、组织新生以及肉芽的形成。此外，该药物还能预防和对抗因长期使用肾上腺皮质激素所引发的肾上腺皮质功能减退。 1.2 【艾曲泊帕与RP-L102 】艾曲泊帕，一种口服血小板生成素，是葛兰素史克与Ligand Pharmaceuticals共同研究的成果。诺华公司在美国以其商品名Promacta进行制造和销售，同时在欧盟以Revolade商品名进行销售。此药物主要用于改善某些个体的中性粒细胞计数，常作为并发疾病期间的支持治疗。 RP-L102是Rocket制药公司研发的一种基于LVV的基因疗法。其治疗流程包括在体外利用CD33+筛选程序对造血干细胞进行筛选和分离，随后通过RP-102进行基因修饰。经过修饰的富含CD34+的造血细胞会被回输至患者体内，以恢复其正常功能。目前，该疗法正在欧盟进行1/2期临床试验研究。 2.1 【 Alglucerase和Elelyso 】戈谢病的治疗迎来了重大突破，在1991年4月，首个有效治疗药物Alglucerase（Ceredase）获得了FDA的批准。这款药物以人胎盘组织为原料，经过提取和精制，制备出葡糖脑苷脂酶，用于治疗戈谢病。然而，遗憾的是，它已从市场上撤出。 Elelyso（Taliglucerase-α）他利苷酶-alfa，这款由以色列Protalix公司和Pfizer（辉瑞）联合开发的重组葡萄糖脑苷脂酶生物制剂，专为治疗Ⅰ型戈谢病而设计。它不仅是植物细胞表达系统诞生的首个药物，更在美国FDA的严格审批下成为第一种获得批准的植物制剂药。 2.2 【 VPRIV与依利格鲁司特】 VPRIV（Velaglucerase-α）被称为维葡瑞，是一款专为戈谢病治疗设计的酶替代疗法药物，由Shire公司研发，并经过美国FDA和欧盟EMEA的优先审批，分别于2010年2月26日和2010年8月26日获得批准。它作为一种长期有效的治疗选择，为戈谢病患者带来了新的希望。依利格鲁司特（Cerdelga），由健赞公司（赛诺菲）研发，是一种专为Ⅰ型戈谢病治疗设计的口服药物。其独特的作用机制在于抑制特异性葡萄糖神经酰胺合酶，从而为患者提供了一种新的治疗选择。 2.3 【 Imiglucerase和Miglustat 】 Imiglucerase（商品名Cerezyme），由Genzyme公司制造，是治疗Ⅰ型和Ⅲ型戈谢病的重要药物。作为西利酶（Ceredase）的改良版，伊米苷酶于1994年5月获得FDA批准，并成功取代了Ceredase。 Miglustat（商品名Zavesca），由英国Oxford GlycoSciences研发并由瑞士生物科技企业Actelion销售，是一种口服小分子药物。当酶替代疗法对轻度至中度Ⅰ型戈谢病（GD1）患者无效时，此药物可作为替代治疗方案。其作用机制在于抑制葡萄糖脑苷脂的形成，这一治疗方法被称为底物还原疗法。 3.1 【氢化可的松与泼尼松龙】对于经典型CAH患者，终身使用糖皮质激素是标准治疗方案，其中氢化可的松是首选药物。在青春期，由于皮质醇的代谢率显著增强，因此一些专家建议在此阶段，特别是对于代谢旺盛的患者，应考虑采用氢化可的松进行治疗。在婴儿期和儿童期，氢化可的松或液体泼尼松龙常被优先考虑，因为它们与人体自然分泌的皮质醇结构相似，对骨骼生长板的影响相对较小。 3.2 【地塞米松及氟氢可的松】在骨骺闭合后，治疗目标主要转变为预防长期不良的临床特征。考虑到青少年患者的依从性可能较差，且在停止生长后，许多经典型CAH患者会转为长效糖皮质激素治疗。氟氢可的松是当前唯一可用的盐皮质激素药物，其常用剂量范围为每日0.05至2毫克。在用药过程中，需密切监测电解质、肾素以及血压水平，以确保剂量调整的合理性。 3.3 【 BBP-631的创新疗法】这是BridgeBio制药公司最新宣布的一种AAV基因疗法，旨在治疗先天性肾上腺发育不全（CAH）。经过多项动物研究证实，该疗法能有效且持久地将正常产生激素的肾上腺基因传递至患者体内。通过刺激肾上腺增生，使患者能够自主制造皮质醇和醛固酮，从而恢复体内的激素和类固醇平衡。这一创新治疗方法在目前的市场上尚属首次，且有望在2022年下半年启动早期临床数据采集工作。 4.1 【左旋肉碱的重要功能】左旋肉碱，又称左旋肉毒碱，是食物中的一种关键成分，广泛存在于自然界中，尤其在山羊肉中的含量高达2.1g/kg。人体自身也能合成左旋肉毒碱，成人体内大约含有20克左右，主要分布在心肌和骨骼肌中。1958年，Fritz发现左旋肉毒碱能够加速线粒体对脂肪的代谢速度，从而明确了它在脂肪氧化过程中的核心作用。 5.1 【维生素B12的参与与缺乏】在甲基丙二酰辅酶A的代谢过程中，维生素B12扮演着至关重要的角色。它参与了将辅因子甲基丙二酰-CoA转化为琥珀酰-CoA的关键步骤。一旦维生素B12的量不足以完成这一转化，未消耗的甲基丙二酰-CoA便会积累，最终引发甲基丙二酸血症。 5.2 【左旋肉碱及其他新疗法】左旋肉碱，在医学领域被称为“左卡尼汀”，是一种广泛应用于运动食品和功能补剂中的成分。全球范围内，这种物质被广泛销售，且所有MMA患者对左旋肉毒碱治疗都表现出反应。此外，左旋肉碱还有助于清除体内的酰基辅酶A，通过转化为可经尿液排出的酰基肉碱，从而在低蛋白饮食中发挥膳食补充剂的作用。 5.3 【 LB-001与HST5040 】 LB-001是LogicBio Therapeutics公司研发的单次给药基因组编辑疗法，旨在为甲基丙二酸血症（MMA）患儿提供新的治疗选择。近期公布了一项重大临床试验成果，标志着首次在儿童患者身上进行的基因编辑疗法取得了突破。 HST5040是由HemoShear公司研发的一款药物，旨在降低甲基丙二酸血症（MMA）和丙酸血症（PA）患者体内积累的有毒代谢物。HST5040作为一种小分子药物，能够轻松分布到患者体内的所有受影响组织，从而在全身范围内发挥作用。 5.4 【 mRNA-3705的探索】 Moderna公司已成功完成对首位患者的给药，这是在评估其研究性mRNA疗法mRNA-3705的安全性和耐受性的1/2期研究中的重要一步。该疗法针对的是甲基丙二酸血症（MMA），一种通过静脉输注给药的罕见代谢性疾病。研究将探索多达五种不同的给药方案，以确定每种方案的安全性和耐受性。在收集足够的安全性和药理学数据后，将选出最佳剂量，并允许更多患者加入可选择的扩展队列，接受进一步治疗。

优先审批基因疗法申请上市临床研究

2025-09-03

·ETPharma

GST waiver for 36 drugs; formulations under 5% slab

New Delhi: Executing the much-awaited overhaul of the Goods and Services Tax (GST) regime, the GST Council has brought a major relief for patients by reducing the indirect tax on medicines to 5 per cent, along with exempting as many as 36 life-saving medicines and other critical drugs. The GST council—a statutory body highest decision making body on indirect taxes led by the Union Finance Ministry at its 56th meeting in the national capital has recommended to reduce the GST on all drugs and medicines (finished formulations) from 12 per cent to 5 per cent under the new two-tier tax structure of 5 and 18 per cent slabs to be effective from September 22. Additionally the body has also recommended to exempt GST on 33 lifesaving drugs and on 3 other medicines used for treatment of cancer, rare diseases and other severe chronic diseases. Speaking at a press briefing after the council 10.5-hour long marathon meet, Finance Minister Nirmala Sitharaman said, “all recommendations have been cleared with zero reservations and have corrected various inverted duty structure problems, classification related issues and that this rationalization exercise will bring predictability and stability of GST.” “These reforms have been carried out with a focus on the common man, and after a rigorous evaluation process in most sectors including healthcare, the rates have come down drastically, she added. Previously, medicines in India were taxed at 5 per cent for life-saving and essential drugs, and at 12 per cent for others but following the announcement all medicines will attract a standard duty of 5 per cent with extensions for several medicines specified at the end of this article. Welcoming the move, Sudarshan Jain, Secretary General, IPA said, “the decision to exempt life-saving and cancer medicines from GST, is a step that will bring direct relief to patients and their families. and, the reduction in GST on a wide range of medicines from 12 per cent to 5 per cent will further help to ease the overall treatment burden and make essential therapies more affordable.” Meanwhile, for companies, the clarity on a 5 per cent slab and exemptions removes long-standing ambiguity, enabling transparent pricing and better market planning. The move will expand access in semi-urban and rural markets, ease litigation, and free up resources for innovation, said Manoj Mishra, Partner and Tax Controversy Management Leader, Grant Thornton Bharat LLP. Notably, on the issue of inverted duty involving APIs and key starting materials—which are currently taxed at 18 per cent against 12 or 5 per cent for finished formulations—further clarity is awaited from the industry. ETPharma earlier reported that, in a letter to the Finance Minister, the national chemist body, the All India Organisation of Chemists and Druggists (AIOCD) had urged, “to bring all medicines including vitamins, probiotics, nutritional, food supplements under the 5 per cent GST slab. The rate rationalization comes days after Prime Minister Narendra Modi had pitched for introducing next-gen reforms across sectors to “increase ease of living for the common man and strengthen the economy.” However, this rationalization brings a revenue shortfall challenge for governments with states likely to bear a greater share of the burden, as they receive over 40 percent of the inter-state inflows in addition to their own SGST collections. Responding to a media query at the media briefing Arvind Shrivastava, Revenue Secretary said that, based on the consumption of FY23-24 the ministry has estimated that the net revenue implication of this proposal will be around Rs 48,000 crore. “However the exercise will trigger buoyancy effects and will have a positive impact on consumer and compliance. And therefore we believe this proposal will be fiscally sustainable both for centers and the states,” Shrivastava noted. ' S.no. Drug Indication Brand name and (Company) 1 Onasemnogene abeparvovec SMA (One-time gene therapy) Zolgensma (Novartis) 2 Asciminib Blood Cancer Scemblix (Novartis) 3 Mepolizumab Asthma Nucala (GSK) 4 Pegylated Liposomal Irinotecan Pancreatic Cancer Onivyde (Ipsen) 5 Daratumumab Cancer Darzalex (J&J subsidiary) 6 Daratumumab subcutaneous Cancer Darzalex Faspro (J&J subsidiary) 7 Teclistamab Blood Cancer Tecvayli (J&J subsidiary) 8 Amivantamab Lung Cancer Rybrevant (J&J subsidiary) 9 Alectinib Lung Cancer Alecensa (Roche) 10 Risdiplam SMA Evrysdi (Roche) 11 Obinutuzumab Blood Cancer Gazyva (Roche) 12 Polatuzumab vedotin Blood Cancer Polivy (Roche) 13 Entrectinib Cancer Rozlytrek (Roche) 14 Atezolizumab Cancer (PD-L1 immunotherapy) Tecentriq (Roche) 15 Spesolimab GPP flares Spevigo (Boehringer Ingelheim) 16 Velaglucerase Alpha Gaucher Vpriv (Takeda) 17 Agalsidase Alfa Fabry disease Replagal (Takeda) 18 Rurioctocog Alpha Pegol Hemophilia Adynovate (Takeda) 19 Idursulphatase Type II Mucopolysaccharidosis Elaprase (Takeda) 20 Alglucosidase Alfa Pompe Myozyme (Sanofi) 21 Laronidase Type I Mucopolysaccharidosis Aldurazyme (Sanofi) 22 Olipudase Alfa ASMD Xenpozyme (Sanofi) 23 Tepotinib Lung Cancer Tepmetko (Maerck KGaA) 24 Avelumab Cancer (PDL-1 immunotherapy) Bavencio (Pfizer) 25 Emicizumab Hemophilia Hemlibra (Roche subsidiary) 26 Belumosudil cGVHD - 27 Miglustat Gaucher Zavesca (J&J subsidiary) 28 Velmanase Alfa Alpha-mannosidosis - 29 Alirocumab Cholesterol lowering drug (LDL-C) Praluent (Sanofi) 30 Evolocumab Cholesterol lowering drug (LDL-C) Repatha (Amgen) 31 Cystamine Bitartrate Cystagon - 32 CI-Inhibitor injection Hereditary Angioedema - 33 Inclisiran Cholesterol lowering drug (LDL-C) Leqvio (Novartis) 34 Agalsidase Beta Fabry disease Fabrazyme (Sanofi) 35 Imiglucerase Gaucher Cerezyme (Sanofi) 36 Eptacog alfa activated recombinant coagulation factor VIIa Hemophilia NovoSeven Note: Above is the list of medicines exempted from GST tax recommended by the 56th GST Council Meet/ Source: PIB By Abhijeet Singh ,

基因疗法

2025-08-20

·BiG生物创新社

依靠分段生产续命，能否换来商业成功？

关于生物制品的分段生产，此前已经陆续讨论了五次。今年上半年，终于迎来了一个重大里程碑。【案例讨论】首家ADC分段生产获批试点【案例讨论】生物制品分段生产试点，首个产品通过检查亦喜亦忧！再议分段生产上海试点生物制品分段生产！花落谁家？进口药境外生产转境内，利好CDMO几何？ 5月15日国家药监局公告：通过优先审评审批程序批准北海康成（上海）生物科技有限公司申报的注射用维拉苷酶β（商品名：戈芮宁）上市，适用于12岁及以上青少年和成人I型和Ⅲ型戈谢病患者的长期酶替代治疗。戈谢病是一种因溶酶体中葡萄糖脑苷脂酶功能缺陷导致的常染色体隐性遗传代谢病，被纳入《第一批罕见病目录》。注射用维拉苷酶β能减少葡萄糖脑苷脂在体内的贮积，从而发挥治疗作用。同日，上海市药监局就此也有公告：该药获益于上海率先推进的生物制品分段生产试点改革，北海康成（上海）生物科技有限公司作为上市许可持有人，原液委托上海药明生物技术有限公司生产，制剂委托无锡药明生物技术股份有限公司生产。不是因为美国法案，国内biotech脱离药明生物上海药品审评核查中心、江苏省药监局审核查验中心在国家药监局核查中心的统筹协调下，通过串联检查等方式使得各阶段的生产和检验数据能够无缝追溯，实现全链条生产质量管理中信息的共享和互通，加快了罕见病创新生物药上市进程。由此可见，5月15日国家药监局批准注射用维拉苷酶β上市，实现多方共赢。无论是企业还是药监部门，不仅仅是多了一个药物获批上市，更是为生物制品分段生产试点提供了实际案例。对于医生和患者来说，当然是有了新的治疗选择。作为首个通过生物制品分段生产检查的创新生物药，注射用维拉苷酶β（研发代号CAN103）的获批无疑在国内药品监管进程中写下浓墨重彩的一笔。国家药监局和上海市药监局两级主管机构都专门发文介绍，足以见得其重大意义。虽然首个通过生物制品分段生产检查获批的药品是落在珠海联邦的利拉鲁肽，不过，维拉苷酶β的获批在药品监管进程上显然更具有突破性。首先，在珠海联邦的案例中，持证人和生产商是属于同一个集团，长期以来都在同一质量体系之中，管理难度相对较低，风险自然也就更低；而此次北海康成的案例，持证人和生产商是相对独立的，尽管仔细深究的话，其背后也存在资本上的关联性（2024年底药明康德持有北海康成9.50%股份），但从内部管理上来说都是独立个体。其次，在珠海联邦的案例中，持证人和生产商都在广东药监部门的管辖范围内，且分段生产的两个场地相聚只有百米（安基路2380号和安基路2428号），如果珠海联邦向药监部门去争取视作同一场地，或许也都是可以商议的；而北海康成的案例中，分段生产的两个场地则是相隔180公里，分属于上海和江苏两地省局管辖，在监管层面的多方协调也是一次重大实践；据上海药监局公告：国家药监局药品审评核查长三角分中心与上海市药监局组建专门服务团队，在检验、审评、核查、生产许可等注册上市的全环节持续跟踪辅导，并联合市相关部门多措并举、精准发力，共同支持企业创新研发，让企业在申报注册上市过程中少走“弯路”。最后，利拉鲁肽是一个生物类似药，此前的原研产品以及后来的多家生物类似药都已经让监管部门对其基本性质是十分熟悉的；而北海康成的注射用维拉苷酶β则是一个新分子，虽然相较于武田的注射用维拉苷酶α可能就只是糖基化存在差异，但毕竟其人体用药实践以及生产工艺和质量控制经验都是十分有限的，也就增加了监管难度。不过，对于北海康成来说，虽然在药政上取得突破性进展并实现产品获批，自然是一片喜悦。然而在短暂喜悦过后，北海康成面对羸弱的财务状况却不敢有所懈怠，前路依然面临重重挑战。首要面对的可能就是，产品获批必然触发一笔里程碑付款，但钱从哪里来？根据北海康成2024年报，综合现金流量表所示现金及现金等价物在2024年底还有1003万元人民币。这点钱大概率是不足以向药明生物支付这笔里程碑款项的，可能也就是够给高管发工资，2024年董事及最高行政人员薪酬为840万元，而雇员福利开支则是8905万元。截至2024年12月31日，北海康成已经将员工人数精简至67名全职员工，并且截至2025年3月中旬，进一步精简人员架构至50名全职雇员，以减少运营成本。改善财务状况的两条路径，除了节流之外，最重要的当然是开源。 2024年收入8510万元，相比2023年达到最高峰的1.03亿元是减少的。抛开奈拉替尼在香港的分销于2023年底结束的因素之外，仍然是较2023年减少6.4%。或许，仅凭艾度硫酸酯酶β和氯馬昔巴特这两款产品销售，难以在短期内大幅度提升收入。更何况这两款产品仅有大中华区权益。或许也可以继续向银行申请授信贷款。不够截止2024年底，公司的银行贷款和其他借款为3000万元人民币。还能从银行拿到多少钱帮助度过难关？或许还真的只能指望刚刚获批的注射用维拉苷酶β了。有新产品可以销售，也就有了新的收入来源；参考武田收购夏尔（Shire）而获得类似品种维拉苷酶α，在过去几年的收入长期稳定在4亿美元；此外，北海康成拥有这款产品的全球权益，如果将海外权益找了合适的买家，或许能极大缓解资金压力。作为罕见病药物第一股，北海康成所作出的各种努力对于全社会的意义，显然不言而喻。面对罕见病药物高昂的价格，支付体系的建立和完善仍然在艰难前行。无论是商业保险还是各地惠民保，都在通过全社会努力极力减轻患者家庭的负担，同时平衡药企持续发展的基本需求。作为一家商业公司，赚钱是其基本使命，但北海康成所面临的最大尴尬是，可能无法坚持到赚钱的那一天。好在依靠分段生产试点工作，已经续命到今天。而药政突破所取得的产品成功注册，能否换来商业上的成功，是不是到头来又是做了一件公益呢？近期活动 9月26-29日，BiG将召开以「创新出海云起潮升」为主题的第十一届IMPACT年会，届时上海杭州双城联动，向全球化，再出发！ ▼ 时间及地点： 9月26-27日上海-张江希尔顿；规模：600-800人 9月29日杭州-西湖大学；规模：100人（邀请制，不对外开放）报名入口会议门票：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、投资机构付费门票：详见上二维码，注册内信息 1v1资本对接会路演项目征集可扫码填写项目信息现场开放1v1咨询台，与各大头部VC面对面 ▼ 电脑端链接：https://event.mymova.com/spa4/#/?eventname=11bigvc 共建Biomedical创新生态圈！如何加入BiG会员？

优先审批上市批准抗体药物偶联物申请上市

100 项与维拉苷酶α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09029	维拉苷酶α	A16AB10	DB06720

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
戈谢病	日本	Takeda Pharmaceutical Co., Ltd.	2014-07-04
I型戈谢病	美国	Takeda Pharmaceuticals U.S.A., Inc.	2010-02-26

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
Ⅲ型戈谢病	临床2期	埃及	Shire Plc	2012-09-14
Ⅲ型戈谢病	临床2期	印度	Shire Plc	2012-09-14
Ⅲ型戈谢病	临床2期	突尼斯	Shire Plc	2012-09-14

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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


NCT05529992 (CTgov)	临床3期	I型戈谢病	20	VPRIV (VPRIV)	壓簾構顧夢鹽廠繭簾獵 = 願遞膚夢夢築築獵衊築範願艱糧遞蓋顧蓋廠顧 (鏇夢蓋蓋淵鏇襯淵蓋範, 蓋壓築築廠願艱鑰構艱 ~ 鏇獵蓋製繭築鑰窪齋鬱) 更多	-	2025-02-26
NCT05529992 (CTgov)	临床3期	I型戈谢病	20	Velaglucerase Alfa (Velaglucerase Alfa)	遞繭築窪顧艱製簾鹽餘(遞糧醖艱鹹構膚範齋衊) = 鏇鹽築構鑰鹹襯選選製鹽選觸簾選襯憲膚膚醖 (觸鑰觸築製鏇築選廠遞, 鹹選遞築積淵艱窪願築 ~ 鏇鑰願製積鑰淵淵鑰網) 更多	-	2025-02-26
NCT02574286 (CTgov)	临床4期	I型戈谢病	21	Vitamin D+Velaglucerase alfa	選簾糧構繭獵鑰製觸鏇(餘選衊繭糧構糧構糧鏇) = 網夢簾繭蓋簾顧網構鑰鑰選遞壓艱繭醖鑰築艱 (窪製築繭夢築鬱選糧膚, 0.394) 更多	-	2022-02-01
NCT01842841 (Pubmed \| Blood Cells Mol Dis) 人工标引	临床3期	戈谢病	5	velaglucerase alfa	鏇糧願簾範糧鏇鑰獵艱(鑰蓋選繭鹹膚遞鏇夢蓋) = Six serious but non-drug-related adverse events were reported. 選壓網獵齋淵築廠齋膚 (製淵蓋觸蓋簾鏇構積構 ) 更多	积极	2016-07-01
NCT01842841 (CTgov)	临床3期	戈谢病	5	VPRIV	襯憲糧觸膚範製窪鬱鹽 = 築觸構構艱鏇網鹽餘淵範遞網範選齋蓋觸範製 (夢鏇蓋觸鹹願製餘簾鬱, 積製鏇鬱鹹製築鹽鏇餘 ~ 鬱淵積繭鹽蓋鏇範製範) 更多	-	2015-12-14
NCT01685216 (CTgov)	临床1/2期	Ⅲ型戈谢病	7	velaglucerase alfa	築鹽衊鏇鏇艱觸鑰醖鏇(獵願顧簾醖膚築壓夢遞) = 積憲遞鏇顧衊積獵醖顧蓋製壓憲蓋餘鹹襯夢齋 (鹽壓鹹製鏇鬱鏇艱顧鬱, 1.213) 更多	-	2015-10-30
NCT01614574 (CTgov)	临床3期	戈谢病	6	VPRIV	壓築餘憲繭顧衊衊壓願 = 鑰襯網鹹選簾鬱鹹網衊網窪齋簾簾憲網醖醖膚 (鏇憲膚廠餘遞築糧顧觸, 衊窪獵鏇襯憲蓋餘衊糧 ~ 鹽鬱蓋願襯糧鑰築糧夢) 更多	-	2014-08-04
NCT00635427 (CTgov)	临床3期	I型戈谢病	95	VPRIV® (VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034))	製壓觸獵簾膚壓襯簾餘 = 憲艱積壓網遞糧窪積壓鬱鑰衊膚夢壓繭選壓築 (網憲製壓襯範觸範鹽憲, 壓糧廠廠範顧窪觸膚觸 ~ 糧齋繭構觸鏇廠簾淵製) 更多	-	2014-01-28
NCT00635427 (CTgov)	临床3期	I型戈谢病	95	VPRIV® (VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039))	網齋餘鹹膚鹽醖淵蓋鹹(鬱選構遞壓製願廠網遞) = 衊築鹽鬱壓餘顧蓋鬱獵鹹憲願繭齋築選積衊醖 (夢糧願齋製夢選齋簾淵, 製鏇餘觸鬱鏇窪選獵糧 ~ 顧鹹襯廠鏇鑰憲繭憲鬱) 更多	-	2014-01-28
NCT00478647 (Pubmed \| Am J Hematol)	临床2/3期	I型戈谢病	40	Velaglucerase alfa	網顧醖觸製製廠顧憲鬱(鏇廠醖窪淵築積糧繭網) = 9 of 40 patients 鑰鹹夢選願糧鏇構網簾 (膚蓋遞壓膚襯選鑰艱蓋 ) 更多	-	2013-03-01
NCT00553631 (CTgov)	临床3期	I型戈谢病	34	imiglucerase	窪窪繭淵鬱鬱簾蓋鬱觸(餘夢願顧夢鏇網鹹廠製) = 選遞構醖襯鬱壓醖鏇選網艱顧鑰願醖夢積製獵 (鏇廠鏇鏇醖鹹顧簾構簾, 0.281) 更多	-	2011-01-04
NCT00430625 (CTgov)	临床3期	I型戈谢病	25	VPRIV (VPRIV® (60 U/kg, IV, Every Other Week))	壓繭壓鹽憲衊觸獵願夢(壓膚顧艱襯餘簾襯製遞) = 醖淵廠醖鑰齋鬱製獵製齋鏇艱顧壓遞觸遞觸鹹 (獵廠選顧鏇構願廠淵夢, 鹽餘觸艱製鹹選觸簾淵 ~ 構觸齋襯艱選鬱壓網積) 更多	-	2010-09-10
NCT00430625 (CTgov)	临床3期	I型戈谢病	25	VPRIV (VPRIV® (45 U/kg, IV, Every Other Week))	鏇構獵廠獵廠鹹鏇網夢(廠鬱艱遞廠構餘蓋艱鏇) = 鬱鏇憲糧夢築齋願廠願鹽顧餘憲積襯顧淵憲積 (鏇淵艱糧鹽壓醖網夢膚, 窪顧壓蓋選壓壓觸蓋選 ~ 餘夢鹽範製顧繭願醖鑰) 更多	-	2010-09-10