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）

2025-03-01·Chinese Journal of Chromatography

Determination of cycloxaprid and paichongding residues in foods of plant origin by ultra performance liquid chromatography-tandem mass spectrometry

Article

作者: Hu, Kui ; Ma, Xin-Xin ; Li, Ju ; Wan, Jian-Chun ; Zhan, Chun-Rui ; Yu, Wan-Wan ; Han, Ying ; Wu, Hua-Wen ; Liu, Jun-Jun ; Li, Shi-Xiang

Neonicotinoid insecticides play an important role in the prevention and control of pests in crops, such as rice, wheat, corn, and vegetables, because of their broad spectrum, high efficiency, low toxicity, and low residue formation. However, their widespread use poses potential threats to the environment and human health. Cycloxaprid and paichongding are two new classes of neonicotinoid insecticides. The National Food Safety Standard Maximum Residue Limits for Pesticides in Foods (GB 2763) specifies the maximum residue limits for paichongding and cycloxaprid in rice, brown rice, wheat, and cabbage. However, the established limits are only temporary. In addition, no detection standards have yet been specified, and no relevant standards have been established in China. Therefore, establishing a method for analyzing cycloxaprid and paichongding residues in food is of particular importance. In this study, an optimized method was established to determine the residues of cycloxaprid and two noncorresponding isomers of paichongding in plant foods using ultra performance liquid chromatography-tandem mass spectrometry. The chromatographic conditions, matrix extraction methods for dried fruits and tea, and amounts of the purification materials C₁₈, PSA, GCB, and anhydrous magnesium sulfate were optimized. The pesticides were extracted using an acetonitrile solution and separated from water with the addition of sodium chloride. The samples were centrifuged at 5000 r/min for 5 min, after which 1.00 mL of the supernatant was purified using 150 mg of anhydrous magnesium sulfate, 25 mg of C₁₈, 25 mg of PSA, and 10 mg of GCB. Cycloxaprid and paichongding were separated on a ACQUITY UPLC BEH C₁₈ chromatographic column (100 mm×2.1 mm, 1.7 μm) via gradient elution with 0.1% formic acid aqueous solution containing 5 mmol/L ammonium formate-acetonitrile as the mobile phase, and detected in electrospray positive ionization mode coupled with multiple-reaction monitoring (MRM) mode. Quantitative analysis was performed using an external standard method. Cycloxaprid and paichongding demonstrated good linear relationships within their respective concentration ranges, and the corresponding correlation coefficients were all above 0.99. The limits of detection (S/N=3) and quantification were 0.05 μg/kg and 0.01 mg/kg, respectively, which meet the requirements specified in GB 2763-2021 and GB 2763.1-2022. The established method was used to validate the recoveries of cycloxaprid and paichongding spiked in foods of plant origin, such as paddy, brown rice, wheat, rice, peanut, raisin, cabbage, lettuce, green bean, tomato, potato, shiitake mushroom, apple, citrus, and tea substrates, at three levels. The spiked levels covered the limit levels specified in GB 2763-2021 and GB 2763.1-2022. The mean recoveries of the target substances added to the 15 substrates at concentrations of 1, 2, and 10 times the LOQ or the limit values specified in GB 2763-2021 and GB 2763.1-2022 were distributed between 78% and 110%, and the RSDs were within the range of 0-12.8%. One hundred batches of fruit and vegetable samples were analyzed using the developed method, and the results showed that the selected samples were not contaminated with either cycloxaprid or paichongding. The proposed method is simple, sensitive, and universal, and has wide coverage. Thus, it can be used as a confirmatory and quantitative detection method for epoxidine and piperidine in foods of plant origin.

2025-01-01·INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

Effective encapsulation of therapeutic recombinant enzyme into polymeric nanoparticles as a potential vehicle for lysosomal disease treatment

Article

作者: Lamas, Diego G ; Islan, Germán A ; Chain, Cecilia Yamil ; Crivaro, Andrea N ; Huck-Iriart, Cristián ; Boztepe, Tugce ; Cisneros, José Sebastián ; Rozenfeld, Paula ; Ceci, Romina

Gaucher Disease (GD) is a genetic disorder with defective activity of the lysosomal enzyme glucocerebrosidase. Velaglucerasa alfa is a recombinant glucocerebrosidase used for enzyme replacement therapy (ERT) of GD. Due to its limited stability and bioavailability, the use of nanosized systems carrying Velaglucerase alfa is proposed as a novel strategy to improve ERT for GD. Highly stable and low-dispersed Velaglucerase-loaded Eudragit nanoparticles (NPs) (NPs: Vela); 150-160 nm mean size, polydispersity index <0. 15, zeta potential around -32 mV, and 95 % Velaglucerase alfa encapsulation efficiency were obtained. Crystallographic structural analysis by Small Angle X-ray Scattering, confirmed that Velaglucerase alfa was incorporated into the nanoparticle matrix. In vitro studies revealed that NPs: Vela preferentially interact with immunoglobulins and fibrinogen, and a positive enzyme release from NPs:Vela was observed at acidic pH; while no release was observed in neutral conditions. A positive internalization of NPs:Vela in GD mesenchymal stem cells (MSC) was also verified, increasing enzyme cellular activity compared to non-treated cells. Confocal microscopy verified that NPs:Vela colocalized with lysosomes, but no effect of NPs: Vela in the mineralization of MSC was observed. Finally, the viability of GD cell lines is not affected by NPs: Vela, in comparison with Velaglucerase alone, that negatively affects the viability of the target cells. This nanocarrier system for Velaglucerase alfa delivery in lysosomes, initially proposed to improve ERT for GD, may also serve as a starting point to address pathophysiological mechanisms in GD and other lysosomal disorders.

2024-12-01·MethodsX

A simple analytical method for simultaneous determination of currently used pesticide (CUP) residue in river water using LC-MS/MS

Article

作者: Mustafa, M Golam ; Kabir, Md Humayun ; Kamruzzaman, Sarker ; Yasmin, Sabina ; Bhuiya, Ananya ; Jahan, Tajnin ; Moniruzzaman, Mohammad ; Rahman, Md Atikur

Pesticide residue monitoring in river water is obligatory for their negative ecotoxicological consequences. The current-use pesticides (CUPs) include a diverse range of synthetic pesticides registered by regulatory bodies worldwide as an alternative to the banned organochlorine pesticides (OCPs). Nowadays, CUPs are preferred for agricultural and non-agricultural purposes due to their low persistence and moderate toxicity. This work aimed to optimize a rapid, sensitive salting-out assisted liquid-liquid microextraction (SALLME) method for the simultaneous determination of seven multi-class CUPs. An optimized dose of C18, GCB, and PSA was employed to facilitate an effective and commercially viable clean-up for routine analysis of pesticide residues, followed by liquid chromatography-tandem mass spectrometry (LC-MS/MS). The optimized SALLME method showed good repeatability and reproducibility, along with low LOD and LOQ values, according to the SANTE/11,312/2021 guidelines. The method validation parameters confirmed the applicability of the optimized SALLME method for trace-level residue analysis in river water. The core advantages of the validated method are:•The method follows simple steps and is cost-effective for the routine analysis of multi-class pesticide residues in river water.•The analytical method is simple and highly sensitive; this could be extended to detect and quantify other pesticides depending on their physicochemical properties.

项与维拉苷酶α 相关的新闻（医药）

2025-05-17

·PipelineReview

CANbridge Announces Marketing Approval of Velaglucerase-beta for Injection (Gaurunning), a Class 1 New Drug for Treating Type I and III Gaucher Disease, in China

SUZHOU, China I May 16, 2025 I CANbridge Pharmaceuticals Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare disease, announced today that velaglucerase-beta for injection (Gaurunning), with its wholly – owned subsidiary, CANbridge (Shanghai) Life Sciences Ltd. as the holder, has obtained marketing approval from the National Medical Products Administration (NMPA). Gaurunning is the first domestically independently developed long-term enzyme replacement therapy for adolescents aged 12 and above and adults with type I and type III Gaucher disease. As a Class 1 innovative drug, it can fully replace similar imported products and will significantly enhance medication accessibility for domestic patients. In August of 2024, CANbridge announced positive top-line results from the pivotal clinical trial of Gaurunning in adult and pediatric Gaucher disease patients. In September, the Center for Drug Evaluation (CDE) of NMPA granted it priority review status. In October of the same year, CANbridge successfully passed the Manufacturing Authorization Holder (MAH) production and quality management system audit and on-site inspection and obtained the Drug Manufacturing License Type B certificate. In November, the New Drug Application (NDA) for Gaurunning was accepted by NMPA. In March 2025, it successfully passed the Pre-approval inspection and pre-marketing GMP compliance inspection for the pilot biological product of Divided manufacturing. Velaglucerase-beta for injection is the first innovative biological product in China to pass the inspection of Divided manufacturing of biological products. James Xue, Ph.D., CANbridge Founder, Chairman, and CEO, stated: “Enzyme replacement therapy (ERT) is a critical component of CANbridge’s global pipeline strategy. The approval of Gaurunning in China is a milestone. The six-month timeframe from NDA acceptance to approval not only reflects the strong safety, effectiveness and reliability of our product, as well as the professionalism in pharmaceutical regulatory affairs but also demonstrates the recognition and support of China’s drug regulatory authorities for the urgent medical needs of Gaucher disease patients. The innovative technology adopted in Gaurunning is enzyme modification and directed evolution, serving as a typical case of new quality productive forces. In the future, combined with continuous flow production process technology, it can be extended to other similar drugs beyond Gaucher disease to achieve large-scale, batch production. Going forward, the platform-based technology of Gaurunning will significantly reduce development costs, enabling patients to access safe, effective, and affordable domestic enzyme replacement therapies. The successful development and approval of Gaurunning embody the original entrepreneurial aspiration of CANbridge. It owes much to the concerted efforts of numerous partners including WuXi Biologics, benefits from the support of national and local regulatory authorities, relevant departments, and medical institutions. Above all, it carries the hope that patients with Gaucher disease across China will soon gain access to sustainable treatment. Gaurunning is the first collaborative project between CANbridge and WuXi Biologics ( 2269.HK ) in the rare disease field. We thank our partners, including WuXi Biologics, for enabling the development and production of this product. This is not only the third rare disease treatment launched by CANbridge’s Hunterase and Livmarli but also the first self-developed product to enter commercialization. We will continue to deepen our focus on localized R&D, production, launch, and accessibility of rare disease drugs to bring innovative treatment solutions to Chinese patients.” Dr. Chris CHEN, Chief Executive Officer of WuXi Biologics, said: “We are extremely honored to have empowered CANbridge to achieve this key milestone of Gaurunning, which also represents a significant breakthrough in China’s rare disease drug innovation. The journey of rare disease drug development is long and arduous, requiring the overcoming of countless technical hurdles and challenges from early laboratory discovery to ultimately benefiting patients. The successful realization of this product from concept to commercial production not only testifies to CANbridge’s decade-long commitment to deepening its presence in the rare disease field but also serves as a successful application of WuXi Biologics’ integrated technology platform and CRDMO business model. The shared vision of both parties in addressing the unmet clinical needs of rare disease patients has led to the achievement of China’s first domestically developed enzyme replacement therapy for Gaucher disease. We look forward to empowering more global partners to accelerate the development of high-quality rare disease treatments, driving rare disease drug innovation, and benefiting more rare disease patients.” About Velaglucerase-beta for Injection (Gaurunning) Velaglucerase-beta for injection is the first domestically developed recombinant human glucocerebrosidase ERT in China, now approved for clinical use in treatment of Type I and Type III Gaucher disease patients. Most Gaucher disease patients are Type I and Type III, which are chronic non-neuronopathic and chronic neuronopathic forms, respectively. velaglucerase-beta is administered as an intravenous infusion and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients. The pivotal clinical trial of velaglucerase-beta achieved positive top-line data in August 2024. The velaglucerase-beta pivotal trial is a randomized, double-blind, dose-comparison study designed to evaluate the efficacy, safety, and pharmacokinetics of intravenous velaglucerase-beta administered every other week in newly treated Gaucher Disease (GD) patients, with an extension period. The results demonstrate that the study successfully met its primary efficacy endpoint, showing a statistically significant mean percentage reduction from baseline in spleen volume at nine months for both the 60 U/kg dose (P<0.0001) and the lower 30 U/kg dose (P<0.001). The primary endpoint of this trial protocol has been agreed upon by the CDE. About Gaucher disease (GD) Gaucher disease, one of the most common lysosomal storage disorders, is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1q22 and affects both males and females equally. Gaucher disease is a clinical spectrum that comprises Type 0 (perinatal-lethal), Type I (chronic non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid b-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside (glucosylceramide) inside lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms. For 30 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trials and real-world data demonstrating significant improvement in the major non-neurological signs and symptoms of disease and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan. About CANbridge Pharmaceuticals Inc. CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 8 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global. For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com . SOURCE: CANbridge Pharmaceuticals

上市批准临床结果优先审批申请上市

2025-05-17

·PRNewswire

WuXi Biologics Congratulates Partner CANbridge Pharmaceuticals on the Approval of Innovative Velaglucerase-beta for Injection (Gaurunning) for Gaucher Disease by China NMPA

Enabled by WuXi Biologics' industry-leading integrated technology platform, velaglucerase-beta for injection has successfully advanced from concept to commercialization. Velaglucerase-beta for injection, as the first and the only locally developed enzyme replacement therapy (ERT) for Gaucher disease in China, marks a significant breakthrough for CANbridge Pharmaceuticals and WuXi Biologics in advancing R&D of innovative rare disease therapeutics in China. Velaglucerase-beta for injection is the first innovative biological product in China to pass segmented manufacturing inspection. WUXI, China and SHANGHAI, May 16, 2025 /PRNewswire/ -- WuXi Biologics (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its partner CANbridge Pharmaceuticals on the approval of its innovative velaglucerase-beta for injection (Gaurunning) by the China National Medical Products Administration (NMPA) for the treatment of adolescents aged 12 and above and adults with type I and type III Gaucher disease. This marks China's first and only locally developed enzyme replacement therapy (ERT) for Gaucher disease. Gaucher disease is a rare inherited genetic lysosomal storage disorder characterized by clinical manifestations such as hepatosplenomegaly, anemia, bone pain, and neurological symptoms, which can be life-threatening in severe cases. Recombinant human glucocerebrosidase ERT has been the standard treatment for Gaucher disease for 30 years, significantly improving major non-neurological signs and symptoms, as well as improving the quality of life for patients. It is estimated that the global prevalence of Gaucher disease is 0.7 to 1.75 per 100,000 people. Gaucher disease has been included in China's "First List of Rare Diseases". Velaglucerase-beta for injection is the first collaboration project on rare diseases between CANbridge and WuXi Biologics, advancing from concept to commercialization by leveraging WuXi Biologics' leading integrated technology platform. The project utilized innovative technology to construct high-expression cell line and achieved significant improvements in both yield and quality through WuXiUPTM, an ultra-high productivity continuous bioprocessing platform of WuXi Biologics. The overall yield increased by more than 110 times, and the specific enzyme activity increased by more than 50%, ensuring the affordability for Gaucher disease patients. Additionally, WuXi Biologics developed cell-based bioassay methods for commercial product release and stability testing, addressing the technical challenges of enzyme testing and effectively ensuring product quality control. Additionally, velaglucerase-beta for injection is the first innovative biological product in China to pass segmented manufacturing inspection. Dr. Chris Chen, CEO of WuXi Biologics, stated, "We are honored to enable CANbridge to achieve the significant milestone of approval for velaglucerase-beta for injection, which represents a critical advancement in rare disease drug innovation in China. Developing therapies for rare diseases is a long and challenging journey, requiring relentless joint efforts to bridge the gap between early-stage discovery and patients' access to medication. The successful commercialization of velaglucerase-beta for injection not only reflects CANbridge's decade-long dedication to rare diseases but also exemplifies the value of WuXi Biologics' integrated platform and CRDMO business model. Furthermore, our shared commitment to addressing the unmet clinical needs of rare disease patients has resulted in China's first locally developed ERT for Gaucher disease. We look forward to enabling more global partners to accelerate the development of high-quality rare disease therapies, and making them accessible and affordable for more patients suffering from rare diseases." Dr. James Xue, Founder, Chairman, and CEO of CANbridge Pharmaceuticals, stated, "Enzyme replacement therapy is a crucial part of CANbridge' global pipeline. The approval of velaglucerase-beta for injection in China is a milestone. By leveraging the integrated technology platform, velaglucerase-beta for injection will significantly reduce development costs, making safe and effective domestically produced ERT accessible and affordable for patients. This product is also the first collaboration project between CANbridge and WuXi Biologics in rare diseases. We extend our gratitude to our partners, including WuXi Biologics, who have empowered the development and manufacturing of velaglucerase-beta for injection. This milestone is not only CANbridge' third rare disease drug to be launched following Hunterase® and Livmarli®, but also the first in-house developed commercialized product. In the future, we will continue to focus on the localized research, manufacturing, commercialization, and accessibility of rare disease drugs, bringing innovative treatments to patients in China." Since its establishment, WuXi Biologics has been committed to enabling global innovative biotech companies to accelerate the rare disease drugs from concept to commercialization, benefiting rare disease patients worldwide. As ofthe end of 2024, there are 21 rare disease projects on the company's platform. Additionally, the company actively promotes public awareness and supports the rare disease patient community through various philanthropic initiatives. Its global "Run for Health" campaign has engaged over 2,500 participants to raise awareness for rare diseases. In China, the company's "Rare Disease Student Education Support Program" has provided scholarships to over 100 students affected by rare diseases over the past four years, fostering their academic growth. About Velaglucerase-beta for Injection (Gaurunning) Velaglucerase-beta for injection is the first domestically developed recombinant human glucocerebrosidase ERT in China, now approved for clinical use in treatment of Type I and Type III Gaucher disease patients. Most Gaucher disease patients are Type I and Type III, which are chronic non-neuronopathic and chronic neuronopathic forms, respectively. velaglucerase-beta is administered as an intravenous infusion and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients. The pivotal clinical trial of velaglucerase-beta achieved positive top-line data in August 2024. The velaglucerase-beta pivotal trial is a randomized, double-blind, dose-comparison study designed to evaluate the efficacy, safety, and pharmacokinetics of intravenous velaglucerase-beta administered every other week in newly treated Gaucher Disease (GD) patients, with an extension period. The results demonstrate that the study successfully met its primary efficacy endpoint, showing a statistically significant mean percentage reduction from baseline in spleen volume at nine months for both the 60 U/kg dose (P<0.0001) and the lower 30 U/kg dose (P<0.001). The primary endpoint of this trial protocol has been agreed upon by the CDE. About Gaucher Disease (GD) Gaucher disease, one of the most common lysosomal storage disorders, is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1q22 and affects both males and females equally. Gaucher disease is a clinical spectrum that comprises Type 0 (perinatal-lethal), Type I (chronic non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid b-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside (glucosylceramide) inside lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms. For 30 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trials and real-world data demonstrating significant improvement in the major non-neurological signs and symptoms of disease and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan. Gaucher disease has been included in China's "First List of Rare Diseases." About WuXi Biologics WuXi Biologics (stock code: 2269.HK) is a leading global Contract Research, Development and Manufacturing Organization (CRDMO) offering end-to-end solutions that enable partners to discover, develop and manufacture biologics – from concept to commercialization – for the benefit of patients worldwide. With over 12,000 skilled employees in China, the United States, Ireland, Germany and Singapore, WuXi Biologics leverages its technologies and expertise to provide customers with efficient and cost-effective biologics discovery, development and manufacturing solutions. As of December 31, 2024, WuXi Biologics is supporting 817 integrated client projects, including 21 in commercial manufacturing (excluding COVID CMO projects). WuXi Biologics regards sustainability as the cornerstone of long-term business growth. The company continuously drives green technology innovations to offer advanced end-to-end Green CRDMO solutions for its global partners while consistently achieving excellence in Environment, Social and Governance (ESG). Committed to creating shared value, it collaborates with all stakeholders to foster positive social and environmental impacts and promote responsible practices that empower the entire value chain. For more information about WuXi Biologics, please visit: . About Canbridge CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 8 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global. For more on CANbridge Pharmaceuticals Inc., please go to: . Contacts Business [email protected] Media [email protected] SOURCE WuXi Biologics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准临床结果临床研究

2023-12-14

·赛柏蓝

23个国产新药进医保！涉齐鲁、恒瑞、贝达等（附新进品种名单）

12月13日，国家医保局公布了2023版国家医保目录调整结果，最终有126个药品新增准入，1个药品因即将撤市被调出；143个目录外药品参加谈判或竞价，其中121个谈判竞价成功，成功率为84.6%，平均降价61.7%。医保局表示，2023版医保目录将于2024年1月1日起正式实施，叠加谈判降价和医保报销因素，本次调整预计未来两年将为患者减负超400亿元。01平均降价61.7%新增126个药品，1个药品被剔除12月13日，国家医保局正式公布了2023年版国家医保药品目录调整名单。据统计，2023年国家医保药品目录新增126种药品（含5个直接调入药品），包括抗肿瘤药21个，新冠治疗药和抗感染药共17个，糖尿病、精神病、风湿免疫等慢病药15个，罕见病药15个（其中阿伐替尼片同为抗肿瘤药），以及其他领域用药59个。同时，调出了1个即将撤市的药品（艾尔巴韦格拉瑞韦片）。143个目录外药品参加谈判竞价，其中121个谈判竞价成功，成功率为84.6%，平均降价61.7%，成功率和价格降幅均与2022年基本相当。叠加谈判降价和医保报销因素，预计未来两年将为患者减负超400亿元。通过本轮调整，国家医保药品目录内药品总数达3088种，其中西药1698种，中成药1390种；中药饮片未作调整，仍为892种。2016-2023年国家医保药品谈判情况来源：国家医保局、国家人社局等，米内网整理2016年至今，国家医保谈判已经进行了八轮，不断优化目录内药品结构和疗效水平，将肿瘤药、慢病药、罕见病药和儿童用药等保障短板逐步补齐。与往年相比，2023国谈新增品种数量、谈判成功率等均有质的飞越。近年来，国家医保目录实现“一年一调”，超过80%的新药能在上市两年内纳入医保，涵盖了新冠感染、肿瘤、慢病、罕见病、儿科等临床治疗领域，大量新机制、新靶点药物被纳入了目录范围，目录内医保药品结构持续优化，群众用药保障水平将进一步提升。02国产新药、罕见病药再掀热潮齐鲁、贝达、AZ成功突围在刚公布的2023国谈目录中，新增进入目录的药品有126款，其中，科伦药业、齐鲁制药、正大制药、扬子江药业等国内药企表现抢眼，均有超5款产品成功准入；进口企业方面，阿斯利康、罗氏、辉瑞等也交出满意答卷，准入产品数均超3款。与去年情况相同，国家继续加大对创新药的倾斜和支持，更好弥补罕见病等重点领域保障短板，实现更高水平、更可持续的“保基本”。在此基础上，国产1类新药、罕见病药等显然本轮国谈的焦点。新增进入2023医保目录的国产1类新药28款国产1类新药通过2023国谈形式审查，有23款药品通过谈判/竞价准入，准入成功率超82%，其中包括16个化学药、4个中成药以及3个生物制品。贝达药业有2款1类新药首次纳入，分别为第三代EGFR-TKI抑制剂甲磺酸贝福替尼胶囊、多靶点受体酪氨酸激酶抑制剂伏罗尼布片。两者均属蛋白激酶抑制剂，2022年中国城市公立医院、县级公立医院、城市社区中心以及乡镇卫生院（简称中国公立医疗机构）终端该品类销售规模突破290亿元。此外，齐鲁制药的伊鲁阿克片是首个国产ALK/ROS1抑制剂；柯菲平医药的盐酸凯普拉生片是国内首个自主研发的钾离子竞争性酸阻滞剂（P-CAB）；众生药业的来瑞特韦片是全球首款无需联用利托那韦的拟肽类3CL新冠治疗药；人福医药的广金钱草总黄酮胶囊是全球首创防治尿结石症中药新药；一力集团的虎贞清风胶囊是国内首个治疗痛风的中药1类新药；浙江博锐生物的泽贝妥单抗注射液是首款国产CD20抗体1类新药；亿帆医药的艾贝格司亭α注射液是全球首个第三代双分子升白针；豪森药业的培莫沙肽注射液是全球唯一EPO受体高特异性小多肽月激动剂。新增进入2023医保目录的罕见病药罕见病药方面，有25款药品通过此前的国谈形式审查，有15款药品新增纳入医保，覆盖16个罕见病病种，填补了10个病种的用药保障空白。其中包括用于戈谢病治疗的酒石酸艾格司他胶囊，原研产品尚未获批进口，凯莱天成医药于2022年10月首仿获批上市、首家视同过评。据新版医保目录显示，该产品医保支付标准为381.00元（84mg/粒），与武田的同类产品注射用维拉苷酶α（第三次与医保目录“失之交臂”）的高昂中标价（11380元/4.4ml/支）相比，其顺利准入目录，将进一步提高该类罕见病患者的用药可及性。此外，新增进入医保目录的罕见病药还有：阿斯利康用于治疗阵发性睡眠性血红蛋白尿症、非典型溶血性尿毒症综合征、抗乙酰胆碱受体抗体阳性的难治性全身型重症肌无力的依库珠单抗注射液，以及用于Ⅰ型神经纤维瘤病的硫酸氢司美替尼胶囊；勃林格殷格翰用于成人泛发性脓疱型银屑病治疗的佩索利单抗注射液；BMS用于治疗成人复发型多发性硬化症的盐酸奥扎莫德胶囊；Argenx用于成人全身型重症肌无力治疗的艾加莫德α注射液；罗氏用于视神经脊髓炎谱系疾病（NMOSD）治疗的药物萨特利珠单抗等。初步估算，上述15种罕见病用药纳入目录后，有望惠及近万名患者。03再有5个国采产品直接调入16个非独家产品竞价“入保”自2022版国家医保目录调整开始，“非独家药品竞价”成为了新亮点。回顾2022版目录新增情况，3个非独家产品盐酸美金刚缓释胶囊、依达拉奉注射液以及注射用替莫唑胺直接调入常规目录，上述3个产品已纳入第七批国采，另外有17个非独家产品通过竞价进入目录。2023版目录最新情况，4个非独家产品艾司奥美拉唑镁肠溶干混悬剂、拉考沙胺口服溶液、磷酸奥司他韦干混悬剂、注射用磷酸特地唑胺以及1个独家产品注射用头孢西丁钠/葡萄糖注射液直接调入常规目录，其中注射用头孢西丁钠/葡萄糖注射液、磷酸奥司他韦干混悬剂已纳入第八批国采，其余3个产品已纳入第九批国采。数据显示，截至目前，拉考沙胺口服溶液、磷酸奥司他韦干混悬剂、注射用磷酸特地唑胺已获批的企业数量超过10家，随着2023版医保目录落地实施，上述产品的竞争将会更加激烈。16个通过竞价新增进入医保目录的非独家产品目前涉及的企业数量均在10家以下，其中注射用头孢他啶阿维巴坦钠2022年在中国公立医疗机构终端的销售额超过5亿元，辉瑞一直占领市场高位，2021年起陆续有国产仿制药获批，齐鲁制药、科伦药业、南京优科生物医药将借助医保之力抢食原研药的市场。吸入用盐酸氨溴索溶液2022年在中国公立医疗机构终端的销售额超过2亿元，这个产品的“入保”之路可谓曲折，2020年、2021年、2022年、2023年通过形式审查名单均有上榜，截至目前该产品已获批的企业数量升至4家，“入保”或将成为该产品放量的新契机。值得注意的是，丁苯那嗪片、尼替西农胶囊目前仅有进口产品获批，国内药企的抢仿机会来了。042023年医保大丰收齐鲁制药、正大制药、科伦药业、扬子江药业2023年医保目录新增产品数量≥5个的企业集团情况统计显示，126个新增产品涉及企业集团超过140个（产品获批日期统计截至12月13日）。齐鲁制药（含子公司）共有2个产品调入常规目录、6个产品通过谈判/竞价顺利入保，数量领先，其中伊鲁阿克片为1类新药，阿立哌唑口溶膜和孟鲁司特钠口溶膜为改良新药。正大制药旗下南京正大天晴制药有1个产品调入常规目录、3个产品通过谈判/竞价顺利入保，独家产品芦比前列酮软胶囊在列；正大天晴药业集团有2个产品通过谈判/竞价顺利入保，正大制药(青岛)有1个产品通过谈判/竞价顺利入保。科伦药业（含子公司）共有1个产品调入常规目录、5个产品通过谈判/竞价顺利入保，公司的粉液双室袋新品及多腔袋新品将迎新的爆发期。扬子江药业（含子公司）共有2个产品调入常规目录、3个产品通过谈判/竞价顺利入保，其中独家中成药连榆烧伤膏在列。附表：2023年国家医保药品目录新增产品情况资料来源：米内网数据库、国家医保局等END作者 | 未晞苍穹来源 | 米内网赛柏蓝药械交流群扫描下方二维码加入医药代理商转型交流群扫描下方二维码加入

一致性评价申请上市

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,

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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

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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)	繭遞齋蓋鑰簾鑰憲淵構 = 醖積觸構鬱顧繭獵遞窪鑰觸選艱築鬱鬱顧鬱鏇 (淵廠憲願艱鹽網壓糧窪, 鹹繭構淵鑰選膚願鹹衊 ~ 齋膚衊淵獵構觸顧糧繭) 更多	-	2025-02-26
NCT05529992 (CTgov)	临床3期	I型戈谢病	20	Velaglucerase Alfa (Velaglucerase Alfa)	醖簾蓋齋觸窪築顧襯齋(衊壓遞壓築繭繭襯繭鏇) = 選壓簾製艱衊顧醖醖願窪顧壓鏇襯齋簾獵壓鬱 (鬱膚選築鬱繭獵醖鑰窪, 鏇餘齋醖選壓艱憲製醖 ~ 鹹憲淵鏇範繭積觸壓簾) 更多	-	2025-02-26
NCT02574286 (CTgov)	临床4期	I型戈谢病	21	Vitamin D+Velaglucerase alfa	範遞網鏇願鹽膚蓋構夢(窪蓋淵壓積觸糧鹹糧繭) = 鬱繭蓋選鹹鏇選膚壓廠網蓋憲網繭鏇獵壓糧廠 (艱繭獵餘艱夢齋願淵遞, 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	築網襯選鏇遞範蓋窪積(衊膚衊製網窪壓積襯鏇) = Six serious but non-drug-related adverse events were reported. 糧網範淵鑰襯繭獵艱夢 (夢餘夢觸鬱齋廠製遞願 ) 更多	积极	2016-07-01
NCT01842841 (CTgov)	临床3期	戈谢病	5	VPRIV	積憲獵鏇觸醖餘鬱獵窪 = 獵齋簾構網壓齋遞鑰願鹹憲鏇鏇顧蓋鹹齋積鬱 (衊鹹壓築壓廠鹹餘鹹遞, 衊網製範壓衊膚遞艱淵 ~ 憲襯觸製範遞鹹廠淵鹽) 更多	-	2015-12-14
NCT01685216 (CTgov)	临床1/2期	Ⅲ型戈谢病	7	velaglucerase alfa	網衊願簾製築鑰製鹹獵(製觸膚淵觸廠糧鹹鬱艱) = 積艱繭鹽淵顧鏇網願夢網獵膚壓衊夢窪壓簾襯 (鏇觸製網鹽淵膚鹹鏇繭, 1.213) 更多	-	2015-10-30
NCT01614574 (CTgov)	临床3期	戈谢病	6	VPRIV	繭衊淵顧艱壓鏇壓淵選 = 鬱蓋鏇願構膚艱襯膚廠淵鏇糧壓壓積構糧糧壓 (願簾網鏇鬱築窪鹹蓋製, 壓鏇餘顧簾範觸遞齋築 ~ 糧積鬱齋憲醖簾廠遞糧) 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))	夢獵鑰顧壓餘顧願淵網 = 獵鑰構鹽膚淵艱積淵蓋膚壓願鬱簾顧鏇憲構窪 (蓋遞鏇窪夢衊膚網鏇製, 壓範艱艱鹽廠鏇憲鹽鑰 ~ 製顧構餘鹹觸艱餘齋鹽) 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))	艱醖製顧鬱窪獵廠鏇鬱(膚繭襯餘廠襯範選製簾) = 淵製繭蓋膚構製觸鹽獵醖窪蓋製壓選餘膚壓齋 (醖廠鏇選膚獵選積淵選, 鏇獵構獵蓋鬱艱壓鹽淵 ~ 齋憲鹽蓋製積襯範齋獵) 更多	-	2014-01-28
NCT00478647 (Pubmed \| Am J Hematol)	临床2/3期	I型戈谢病	40	Velaglucerase alfa	積淵醖壓顧壓鏇壓壓蓋(餘繭衊鏇夢積鏇獵齋選) = 9 of 40 patients 襯衊築艱襯積獵繭鬱觸 (範簾憲遞選窪鬱蓋願餘 ) 更多	-	2013-03-01
NCT00553631 (CTgov)	临床3期	I型戈谢病	34	imiglucerase	顧憲淵築醖餘鬱餘積蓋(衊積艱夢網淵繭遞鏇選) = 築觸鏇蓋鑰選觸窪築窪簾艱夢顧衊餘鬱鹽鏇顧 (窪構範鬱糧觸艱廠齋蓋, 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))	鹽願範壓網壓夢淵遞遞(構鹽窪衊積淵鏇襯鬱淵) = 顧襯遞簾鹹鬱積憲鬱簾窪範鹹構簾憲範鏇憲鹽 (選糧廠憲夢選艱餘鑰蓋, 鑰衊憲憲蓋襯獵夢獵醖 ~ 觸構鬱壓願鬱遞膚壓齋) 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))	願鬱壓餘願簾憲窪構鏇(憲鏇壓齋窪膚餘壓壓衊) = 窪夢簾艱衊遞艱艱憲鏇窪獵壓窪艱膚鬱範夢膚 (廠築鏇醖窪糧獵膚築鏇, 壓遞醖襯淵膚顧獵範蓋 ~ 廠窪獵艱遞簾廠顧艱範) 更多	-	2010-09-10