A Single-arm, Open-label, Multiple-dose, Phase 3 Study to Evaluate Usability of Subcutaneous Auto-injector of CT-P47 in Patients with Moderate to Severe Active Rheumatoid Arthritis

开始日期2023-01-26

申办/合作机构

Celltrion, Inc.

NCT05617183

/ Recruiting临床1期

A Phase 1, Randomized, Open-label, Two-arm, Parallel Group, Single-dose Study to Compare the Pharmacokinetics and Safety of the Auto-injector and Pre-filled Syringe of CT-P47 in Healthy Subjects

This is a phase 1 study to compare the Pharmacokinetics and Safety of the Auto-injector and Pre-filled syringe of CT-P47 in Healthy Subjects.

开始日期2022-11-15

申办/合作机构

Celltrion, Inc.

NCT05489224

/ Completed临床3期

A Randomized, Active-Controlled, Double-Blind, Phase 3 Study to Compare Efficacy and Safety of Two Intravenous Infusion Formulations of Tocilizumab (CT-P47 and RoActemra) When Co-administered With Methotrexate in Patients With Rheumatoid Arthritis

This is a phase 3 study to compare efficacy and safety of CT-P47 and RoActemra in patients with moderate to severe active rheumatoid arthritis.

开始日期2022-09-14

申办/合作机构

Celltrion, Inc.

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项与托珠单抗生物类似药 (Celltrion) 相关的文献（医药）

2024-10-01·RMD Open

Efficacy and safety of CT-P47 versus reference tocilizumab: 32-week results of a randomised, active-controlled, double-blind, phase III study in patients with rheumatoid arthritis, including 8 weeks of switching data from reference tocilizumab to CT-P47

Article

作者: Zielińska, Agnieszka ; Jeka, Sławomir ; Dudek, Anna ; Krogulec, Marek ; Jaworski, Janusz ; Trefler, Jakub ; Krajewska-Włodarczyk, Magdalena ; Bae, YunJu ; Kim, SungHyun ; Jung, YooBin ; Smolen, Josef S ; Wojciechowski, Rafał ; Burmester, Gerd R ; Klimiuk, Piotr Adrian ; Yang, GoEun ; Racewicz, Artur ; Kolossa, Katarzyna ; Hong, JiWoo ; Keystone, Edward ; Hrycaj, Paweł

Objectives:

To demonstrate efficacy equivalence of CT-P47 and EU-approved reference tocilizumab (r-TCZ) in patients with rheumatoid arthritis (RA).

Methods:

This double-blind, phase III study randomised (1:1) patients to receive CT-P47 or r-TCZ (8 mg/kg) every 4 weeks until week 20 during treatment period (TP) 1. Prior to week 24 dosing, patients receiving r-TCZ were randomised (1:1) to continue r-TCZ or switch to CT-P47; patients receiving CT-P47 continued CT-P47 (TP2, 8 mg/kg every 4 weeks until week 48). The dual primary endpoints (for different regulatory requirements) were mean changes from baseline in Disease Activity Score in 28 joints (DAS28; erythrocyte sedimentation rate (ESR)) at week 12 and week 24. Efficacy equivalence was determined if CIs for the treatment difference were within predefined equivalence margins: (95% CI −0.6, 0.6 (analysis of covariance (ANCOVA)) at week 12 or 90% CI −0.6, 0.5 (ANCOVA with multiple imputation) at week 24). Additional efficacy, pharmacokinetic (PK) and safety endpoints, including immunogenicity, were investigated. Findings up to week 32 are presented.

Results:

In TP1, 471 patients were randomised (234 CT-P47; 237 r-TCZ). The 95% and 90% CIs for the estimated treatment differences were contained within the predefined equivalence margins; the estimated difference in DAS28-ESR at week 12 was –0.01 (95% CI −0.26, 0.24) and at week 24 was −0.10 (90% CI −0.30, 0.10). Secondary efficacy endpoints, PKs and overall safety were comparable between groups up to week 32.

Conclusions:

Efficacy equivalence, alongside comparable PK, safety and immunogenicity profiles, was determined between CT-P47 and r-TCZ in adults with RA, including after switching from r-TCZ to CT-P47.

2024-07-02·EXPERT OPINION ON BIOLOGICAL THERAPY

Pharmacokinetics and safety of candidate tocilizumab biosimilar CT-P47 administered by auto-injector or pre-filled syringe: a randomized, open‑label, single-dose phase I study

Article

作者: Jang, JiYoung ; Keystone, Edward ; Yu, Kyung-Sang ; Burmester, Gerd R ; Shin, Dongseong ; Jeon, DaBee ; Moon, Seol Ju ; Ryu, Hyunwook ; Kim, Min-Gul ; Bae, JiHun ; Park, MinKyu ; Kim, SungHyun ; Lee, JaeYong ; Yang, GoEun ; Bae, YunJu ; Smolen, Josef S ; Hwang, JunGi

BACKGROUND:

This study compared the pharmacokinetics (PK), immunogenicity, and safety of candidate tocilizumab biosimilar, CT-P47, administered via auto-injector (CT-P47 AI) or pre-filled syringe (CT-P47 PFS), in healthy Asian adults.

RESEARCH DESIGN AND METHODS:

In this phase I, multicenter, open-label study, participants were randomized 1:1 to receive a single 162 mg/0.9 mL dose of CT-P47 via AI or PFS. Primary endpoints were area under the concentration - time curve from time zero to infinity (AUC_0-inf) and maximum serum concentration (C_max). PK equivalence was determined if 90% confidence intervals (CIs) for the ratios of geometric least-squares means (gLSMs) were within the predefined 80-125% equivalence margin. Secondary PK parameters, immunogenicity, and safety outcomes were also assessed.

RESULTS:

Of 314 participants randomized (155 CT-P47 AI; 159 CT-P47 PFS), 310 received the study drug (153 CT-P47 AI; 157 CT-P47 PFS). Primary and secondary PK results, immunogenicity and safety were similar between groups. Ninety percent CIs for the ratio of gLSMs were within the predefined equivalence margin for AUC_0-inf (85.87-102.94) and C_max (82.98-98.16).

CONCLUSIONS:

PK equivalence between CT-P47 AI and CT-P47 PFS was demonstrated in healthy Asian adults, with comparable immunogenicity and safety between the two devices.

TRIAL REGISTRATION:

ClinicalTrials.gov: NCT05617183.

2023-05-01·Expert opinion on investigational drugs

Pharmacokinetics and safety of candidate tocilizumab biosimilar CT-P47 versus reference tocilizumab: a randomized, double-blind, single-dose phase I study.

Article

作者: Park, Min Kyu ; Kim, Byungwook ; Yu, Kyung-Sang ; Ghim, JongLyul ; Chung, Jae-Yong ; Chung, Hyewon ; Shin, Dongseong ; Jeon, DaBee ; Smolen, Josef S ; Bae, JiHun ; Kim, Min-Gul ; Keystone, Edward ; Burmester, Gerd R ; Hwang, Jun Gi ; Yoo, JaeKyoung ; Kim, SungHyun ; Yang, GoEun ; Bae, YunJu

BACKGROUND:

CT-P47 is a candidate tocilizumab biosimilar. This study assessed the pharmacokinetic (PK) equivalence of CT-P47 and European Union-approved reference tocilizumab (EU-tocilizumab) in healthy Asian adults.

RESEARCH DESIGN AND METHODS:

This double-blind, multicenter, parallel-group trial randomized healthy adults (1:1) to receive a single (162 mg/0.9 mL) subcutaneous dose of CT-P47 or EU-tocilizumab. The primary endpoint (Part 2) was PK equivalence by area under the concentration - time curve (AUC) from time zero to last quantifiable concentration (AUC_0-last), AUC from time zero to infinity (AUC_0-inf), and maximum serum concentration (C_max). PK equivalence was concluded if 90% confidence intervals (CIs) for the ratios of geometric least-squares means (gLSMs) were within the 80-125% equivalence margin. Additional PK endpoints, immunogenicity, and safety were evaluated.

RESULTS:

In Part 2, 289 participants were randomized (146 CT-P47; 143 EU-tocilizumab); 284 received study drug. AUC_0-last, AUC_0-inf, and C_max were equivalent between CT-P47 and EU-tocilizumab: 90% CIs for the ratios of gLSMs were within the 80-125% equivalence margin. Secondary PK endpoints, immunogenicity, and safety were comparable between groups.

CONCLUSIONS:

CT-P47 demonstrated PK equivalence with EU-tocilizumab and was well tolerated, following a single dose in healthy adults.

CLINICAL TRIAL REGISTRATION:

www.clinicaltrials.gov identifier is NCT05188378.

项与托珠单抗生物类似药 (Celltrion) 相关的新闻（医药）

2025-02-03

·Pharmaceutical Technology

Celltrion’s Avtozma receives FDA approval for multiple conditions

The approval represents the seventh marketing authorisation of Celltrion in the country. Credit: © CELLTRION. The US Food and Drug Administration (FDA) has granted approval for Celltrion ‘s Avtozma (tocilizumab-anoh) in both intravenous and subcutaneous formulations as an Actemra biosimilar for treating several conditions. These include giant cell arteritis, Covid-19, polyarticular juvenile idiopathic arthritis, systemic juvenile idiopathic arthritis and rheumatoid arthritis. The FDA’s approval is grounded in an evidence totality and comprehensive data package, which includes outcomes from a Phase III global study showcasing Avtozma’s biosimilarity to reference tocilizumab in subjects with moderate to severe active rheumatoid arthritis. The study met its primary endpoint, demonstrating a comparable change from baseline in disease activity score utilising 28 joints (DAS28)-ESR at week 24. Final one-year outcomes further backed the biosimilar’s comparability in secondary efficacy, safety, immunogenicity and pharmacokinetics. Clinical evidence has confirmed that the biosimilar and its reference to tocilizumab are said to be ‘highly similar’, with no clinically meaningful differences in safety, immunogenicity or efficacy. This approval represents the company’s seventh marketing authorisation in the country and adds to its portfolio as the company’s fifth immunology biologic. Celltrion USA chief commercial officer Thomas Nusbickel said: “This approval represents a strategic addition to our immunology portfolio, further strengthening our commitment to delivering accessible and high-quality treatment options for patients and healthcare providers. “Our goal is to provide safe and effective alternatives and ensure appropriate access so plan sponsors can address unique population needs.” Avtozma comprises a recombinant humanised monoclonal antibody, tocilizumab, which blocks the interleukin 6 (IL-6) receptor. Celltrion filed for regulatory approval with the FDA and the European Medicines Agency in January and February last year, respectively, based on the data from the Phase III global trial. In August last year, the European Commission approved the company’s Stelara biosimilar, SteQeyma, for treating chronic inflammatory conditions.

上市批准临床3期临床结果免疫疗法生物类似药

2025-01-31

·PRNewswire

U.S. FDA approves Celltrion's AVTOZMA® (tocilizumab-anoh), a biosimilar to ACTEMRA®

AVTOZMA® will be available as both intravenous (IV) and subcutaneous (SC) formulations with the same formulations and dosages as the reference product Approval was received for multiple indications, including rheumatoid arthritis (RA), giant cell arteritis (GCA), polyarticular juvenile idiopathic arthritis (pJIA), systemic juvenile idiopathic arthritis (sJIA) and COVID-19[1] AVTOZMA® becomes Celltrion's fifth immunology biologic and seventh biosimilar approved by the FDA JERSEY CITY, N.J., Jan. 30, 2025 /PRNewswire/ -- Celltrion today announced that the U.S. Food and Drug Administration (FDA) has approved AVTOZMA® (CT-P47, tocilizumab-anoh) in both an intravenous (IV) and subcutaneous (SC) formulation as a biosimilar to ACTEMRA®. AVTOZMA is indicated for the treatment of multiple diseases including rheumatoid arthritis (RA), giant cell arteritis (GCA), polyarticular juvenile idiopathic arthritis (pJIA), systemic juvenile idiopathic arthritis (sJIA) and coronavirus disease (COVID-19).[1] "Introducing both IV and SC formulations of AVTOZMA provides flexibility and a wider range of treatment options," said Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. "This approval represents a strategic addition to our immunology portfolio, further strengthening our commitment to delivering accessible and high-quality treatment options for patients and healthcare providers. Our goal is to provide safe and effective alternatives and ensure appropriate access so plan sponsors can address unique population needs." The FDA's decision is based on a comprehensive data package and the totality of evidence, including the results from a phase III study demonstrating biosimilarity between AVTOZMA and reference tocilizumab in patients with moderate to severe active RA. The primary endpoint was met in terms of change from baseline in disease activity score using 28 joints (DAS28)-ESR at Week 24, and the final 1-year results supported comparability in secondary efficacy, pharmacokinetic (PK), safety and immunogenicity results between AVTOZMA and reference tocilizumab. The clinical results demonstrated that AVTOZMA and its reference tocilizumab are highly similar and have no clinically meaningful differences in terms of efficacy, safety, pharmacokinetics (PK) and immunogenicity.[2] AVTOZMA will be available in both IV and SC formulations, with the same formulation and dosages as the reference product. The IV infusion is provided as 80 mg/4 mL (20 mg/mL), 200 mg/10 mL (20 mg/mL), 400 mg/20 mL (20 mg/mL) and the SC injection comes in 162 mg/0.9 mL in a single-dose prefilled syringe or single-dose autoinjector. AVTOZMA is Celltrion's seventh biosimilar granted marketing authorization in the U.S. About AVTOZMA ® (tocilizumab-anoh)[1] AVTOZMA ® (tocilizumab-anoh), containing the active ingredient tocilizumab, is a recombinant humanized monoclonal antibody that acts as an interleukin 6 (IL-6) receptor antagonist. Based on data from the global Phase III clinical trial designed to evaluate the efficacy, pharmacokinetics (PK), safety, and immunogenicity of CT-P47 compared to reference tocilizumab, AVTOZMA was filed for regulatory approval with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) in January and February 2024, respectively. INDICATION AVTOZMA® (tocilizumab-anoh) is an interleukin-6 (IL-6) receptor antagonist indicated for treatment of: Rheumatoid Arthritis (RA): Adult patients with moderately to severely active RA who have had an inadequate response to one or more Disease-Modifying Anti-Rheumatic Drugs (DMARDs). Giant Cell Arteritis (GCA): Adult patients with GCA. Polyarticular Juvenile Idiopathic Arthritis (pJIA): Patients 2+ years-old with active pJIA. Systemic Juvenile Idiopathic Arthritis (sJIA): Patients 2+ years-old with active sJIA. COVID-19: Hospitalized adult patients with COVID-19 who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). IMPORTANT SAFETY INFORMATION WARNING: RISK OF SERIOUS INFECTIONS AVTOZMA® and other tocilizumab products may increase the risk of serious infections, potentially leading to hospitalization or death, especially in patients using concurrent immunosuppressants. If a serious infection develops, interrupt AVTOZMA until the infection is controlled. Reported infections include: Active tuberculosis (TB) which may present with pulmonary or extrapulmonary disease. Test for latent TB before and during treatment (except in COVID-19 patients) and treat latent infections before starting AVTOZMA. Invasive fungal infections: Such as candidiasis, aspergillosis, and pneumocystis, may present as disseminated rather than localized disease. Opportunistic infections, including bacterial, viral and other opportunistic pathogens. Monitor patients for signs of infection, including TB, during and after AVTOZMA treatment. Contraindications: Known hypersensitivity to tocilizumab products. Serious Infections. Serious and sometimes fatal infections have been reported with AVTOZMA. Do not use during active infections, including localized infections. Discontinue AVTOZMA if a serious infection occurs and resume only once controlled. Gastrointestinal (GI) Perforation. Gastrointestinal perforations, often linked to diverticulitis, have been reported with tocilizumab. Use AVTOZMA cautiously in high-risk patients and promptly evaluate new abdominal symptoms for early detection and management. Hepatoxicity. Monitor for hepatic injury signs. Avoid AVTOZMA if ALT/ AST >1.5x ULN (RA/GCA) or >10x ULN (COVID-19); discontinue if ALT/AST >5x ULN or symptoms of liver disease develop. Changes in Laboratory Parameters. Monitor neutrophils, platelets, liver enzymes, and lipids due to potential treatment-related changes; avoid initiating AVTOZMA in patients with critically low ANC or platelet counts. Immunosuppression. The impact of AVTOZMA on malignancy development is unknown, but it may increase risk as an immunosuppressant. Hypersensitivity Reactions, including anaphylaxis, and death, have occurred; administer IV infusions with anaphylaxis management support, discontinue permanently if reactions occur, and avoid use in patients with known hypersensitivity. Demyelinating Disorders. The impact of tocilizumab on demyelinating disorders is unknown, but rare cases were reported; monitor symptoms and use caution with preexisting or recent disorders. Active Hepatic Disease and Hepatic Impairment. Treatment with AVTOZMA is not recommended. Live Vaccines. Avoid concurrent use with AVTOZMA. Adverse Reactions (≥5%) include upper respiratory tract infections, nasopharyngitis, headache, hypertension, elevated ALT, and injection site reactions. For more information, see Full Prescribing Information. About Celltrion Celltrion is a leading biopharmaceutical company based in Incheon, South Korea that specializes in researching, developing, manufacturing, marketing and sales of innovative therapeutics that improve people's lives worldwide. Celltrion endeavors to offer high-quality, cost-effective solutions through an extensive global network that spans more than 110 countries. Celltrion has seven biosimilars approved by the U.S. FDA: INFLECTRA® (infliximab-dyyb), TRUXIMA® (rituximab-abbs), HERZUMA® (trastuzumab-pkrb), VEGZELMA® (bevacizumab-adcd), YUFLYMA®(adalimumab-aaty), STEQEYMA® (ustekinumab-stba), and AVTOZMA® (tocilizumab-anoh), as well as novel biologic ZYMFENTRA® (infliximab-dyyb). For more information, please visit . FORWARD-LOOKING STATEMENT Certain information set forth in this press release contains statements related to our future business and financial performance and future events or developments involving Celltrion Inc. and its subsidiaries that may constitute forward-looking statements, under pertinent securities laws. This press release contains forward looking statements. These statements may be also identified by words such as "prepares," "hopes to," "upcoming," "plans to," "aims to," "to be launched," "is preparing," "once gained," "could," "with the aim of," "may," "once identified," "will," "working towards," "is due," "become available," "has potential to," the negative of these words or such other variations thereon or comparable terminology. In addition, our representatives may make oral forward-looking statements. Such statements are based on the current expectations and certain assumptions of Celltrion Inc. and its subsidiaries' management, of which many are beyond its control. Forward-looking statements are provided to allow potential investors the opportunity to understand management's beliefs and opinions in respect of the future so that they may use such beliefs and opinions as one factor in evaluating an investment. These statements are not guarantees of future performance and undue reliance should not be placed on them. Such forward-looking statements necessarily involve known and unknown risks and uncertainties associated with the company's business, including the risk factors disclosed in its Annual Report and/or Quarterly Reports, which may cause actual performance and financial results in future periods to differ materially from any projections of future performance or results expressed or implied by such statements. Celltrion Inc. and its subsidiaries undertake no obligation to update forward-looking statements if circumstances or management's estimates or opinions should change except as required by applicable securities laws. Trademarks AVTOZMA® is a registered trademark of Celltrion Inc. ACTEMRA® is a registered trademark of Chugai Pharmaceutical Co., Ltd. References [1] AVTOZMA U.S. prescribing information (2024) [2] Gerd Burmester et al., Similar Efficacy, PK, Safety, and Immunogenicity of Tocilizumab Biosimilar (CT-P47) and Reference Tocilizumab in Patients with Moderate-to-Severe Active Rheumatoid Arthritis: Week 52 Results from the Phase III Single Transition Study. Poster Presentation (abstract no. 0502). Presented at ACR 2024. Available at: [Last accessed December 2024] For further information please contact:  Andria Arena [email protected] +1 516-578-0057 SOURCE Celltrion WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期上市批准临床结果免疫疗法

2025-01-06

·CPHI制药在线

FDA 2025年第一季度审批全览

关注并星标CPHI制药在线 01 Ozempic (semaglutide) ● 开发商：诺和诺德 ● 模态：多肽 ● PDUFA日期：2025年1月 ● 适应症类型/说明：代谢、肾脏/每周一次皮下注射semaglutide的新适应症，用于治疗患有 2 型糖尿病和慢性肾脏病 (CKD) 的成年人，包括延缓 CKD 进展并降低肾脏和心血管死亡风险。 ● 审评类型：标准审评 ● 申请递交时间：2024年3月 ● 审评情况：FDA于2024年6月24日接受申请 02 Subvenite (lamotrigine oral liquid) ● 开发商：OWP Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年1月3日 ● 适应症类型/说明：中枢神经系统疾病/用于治疗癫痫和躁郁症的抗癫痫药物的新型口服液制剂 ● 审评类型：505(b)(2) NDA标准审评 ● 申请递交时间：2024年3月 ● 审评情况：FDA于2024年5月20日接受申请 03 TX05 (trastuzumab 生物类似药) ● 开发商：Tanvex ● 模态：单抗 ● PDUFA日期：不晚于2025年1月6日 ● 适应症类型/说明：癌症/Genetech的 HER2 靶向抗体Herceptin的生物类似药 ● 审评类型：351(k) BLA标准审评 ● 申请递交时间：2024年7月6日（重新递交） ● 审评情况：FDA之前于2022年8月签发了完整回复函 04 Ryoncil (remestemcel-L-rknd，新生物制品) ● 开发商：Mesoblast ● 模态：细胞疗法 ● PDUFA日期：2025年1月7日 ● 适应症类型/说明：免疫学/同种异体扩增间充质基质细胞 (MSC) 干细胞疗法，用于治疗类固醇难治性急性移植物抗宿主病 (SR-aGVHD) 的儿科患者 ● 审评类型：优先审评 ● 申请递交时间：2024年7月8日（重新递交） ● 审评情况：FDA之前于2022年8月3日签发了第二封完整回复函（第一封签发于2020年10月1日） 05 Tabelecleucel （新生物制品） ● 开发商：Atara Biotherapeutics/Pierre Fabre ● 模态：细胞疗法 ● PDUFA日期：2025年1月15日 ● 适应症类型/说明：免疫学、支持性治疗/同种异体、Epstein-Barr 病毒 (EBV) 特异性 T 细胞免疫疗法，用于单药治疗 2 岁及以上患有 EBV 阳性移植后淋巴增生性疾病 (EBV+ PTLD) 的成人和儿童患者，这些患者至少接受过一次先前治疗 ● 审评类型：优先审评 ● 申请递交时间：2024年5月20日 ● 审评情况：突破性疗法认定 06 Lumakras (sotorasib) ● 开发商：安进 ● 模态：小分子 ● PDUFA日期：2025年1月17日 ● 适应症类型/说明：癌症 /新适应症，与panitumumab (Vectibix) 组成联合疗法，用于治疗 KRAS G12C 突变转移性结直肠癌 (CRC) 。 ● 审评类型：优先审评 ● 申请递交时间：2024年4月 ● 审评情况：延期，原PDUFA日期为2024年10月17日 07 Leqembi (lecanemab-irmb) ● 开发商：卫材/渤健 ● 模态：抗体 ● PDUFA日期：2025年1月25日 ● 适应症类型/说明：中枢神经系统疾病/新的维持给药方案，为已完成每两周一次的阿尔茨海默病治疗初始阶段的患者提供每月一次的Leqembi静脉注射 ● 审评类型：标准审评 ● 申请递交时间：2024年4月1日 ● 审评情况：FDA于2024年6月9日接受申请 08 CT-P47 (tocilizumab生物类似药) ● 开发商：Celltrion ● 模态：单抗 ● PDUFA日期：不晚于2025年1月28日 ● 适应症类型/说明：免疫学/抗白细胞介素 6 (IL-6) 抗体Actemra生物类似药，针对类风湿关节炎和幼年特发性关节炎等适应症 ● 审评类型：351(k) BLA标准审评 ● 申请递交时间：2024年1月28日 09 Elamipretide （新分子实体） ● 开发商：Stealth BioTherapeutics ● 模态：多肽 ● PDUFA日期：2025年1月29日 ● 适应症类型/说明：心血管先天性疾病/与磷脂心磷脂结合的线粒体保护肽，用于治疗巴特综合征 ● 审评类型：优先审评（最初为标准审评，但在Stealth Bio的要求下更改为优先审评） ● 申请递交时间：2024年1月 ● 审评情况：最初申请递交时间为2021年8月，FDA 2021年10月签署拒绝备案(RTF)信函；重新递交后，FDA于2024年4月8日接受申请；罕见儿科疾病认定。 10 Suzetrigine (VX-548，新分子实体) ● 开发商：Vertex ● 模态：小分子 ● PDUFA日期：2025年1月30日 ● 适应症类型/说明：中枢神经系统疾病/选择性 NaV1.8 疼痛信号抑制剂，口服、非阿片类药物，治疗中度至重度急性疼痛 ● 审评类型：优先审评 ● 申请递交时间：2024年5月30日 ● 审评情况：FDA 2024年7月30日接受申请；突破性疗法认定 11 Treosulfan (新药) ● 开发商：medac GmbH/Medexus ● 模态：小分子 ● PDUFA日期：2025年1月30日 ● 适应症类型/说明：癌症、血液疾病/双功能烷化剂，与氟达拉滨联合使用，作为接受异基因造血干细胞移植 (allo-HSCT) 的成人和儿科患者的减毒预处理方案 ● 审评类型：标准审评 ● 申请递交时间：2024年4月 ● 审评情况：FDA 2021年8月3日签署完整回复函；重新提交申请后FDA于2024年6月6日接受申请；PDUFA日期由最初的2024年10月30日推迟。 12 AXS-07 (meloxicam/rizatriptan) ● 开发商：Axsome Therapeutics ● 模态：小分子 ● PDUFA日期：2025年1月31日 ● 适应症类型/说明：中枢神经系统疾病/偏头痛的急性治疗 ● 审评类型：505(b)(2) NDA标准审评 ● 申请递交时间：2024年7月 ● 审评情况：2021 年 7 月首次提交申请，FDA 于 2022 年 5 月 2 日签署完整回复函；2024 年 9 月 4 日FDA接受重新提交审查。 13 Translarna (ataluren，新分子实体) ● 开发商：PTC Therapeutics ● 模态：小分子 ● PDUFA日期：2025年2月或3月 ● 适应症类型/说明：中枢神经系统疾病/治疗无义突变杜氏肌营养不良症 (nmDMD) ● 审评类型：优先审评 ● 申请递交时间：2024年8至9月 14 Omvoh (mirikizumab-mrkz) ● 开发商：礼来 ● 模态：单抗 ● PDUFA日期：不迟于2025年2月 ● 适应症类型/说明：胃肠道疾病/ Omvoh的新适应症，治疗患有中度至重度活动性克罗恩病的成年人 ● 审评类型：标准审评 ● 申请递交时间：2024年3至4月 15 Enhertu (fam-trastuzumab deruxtecan-nxki) ● 开发商：第一三共/阿斯利康 ● 模态：ADC ● PDUFA日期：2025年2月1日 ● 适应症类型/说明：癌症/Enhertu新适应症，治疗不可切除或转移性 HER2 低或 HER2 超低乳腺癌的成年人 ● 审评类型：优先审评 ● 申请递交时间：2024年8月1日 ● 审评情况： FDA 于 2024年10月1日接受申请；突破性疗法认定 16 SPN-830 (apomorphine infusion pump) ● 开发商：Supernus/US WorldMeds/Brittania ● 模态：小分子 ● PDUFA日期：2025年2月1日 ● 适应症类型/说明：中枢神经系统疾病/多巴胺激动剂apomorphine的持续皮下输注制剂，用于治疗帕金森病成人的运动波动（OFF 发作） ● 审评类型：标准审评 ● 申请递交时间：2024年8月1日 ● 审评情况：经历一次RTF（Refuse to File）和两次完整回复函。 17 Bentracimab （新生物制品） ● 开发商：SFJ Pharmaceuticals/SERB Pharmaceuticals ● 模态：抗体 ● PDUFA日期：不晚于2025年2月2日 ● 适应症类型/说明：心血管疾病/针对因使用替格瑞洛而引起的紧急出血事件，或在紧急手术情况下需要逆转替格瑞洛的抗血小板效果 ● 审评类型：优先审评 ● 申请递交时间：2024年5至6月 ● 审评情况：FDA 2024年8月2日接受BLA申请；突破性疗法认定 18 Datopotamab deruxtecan (Dato-DXd，新生物制品) ● 开发商：第一三共/阿斯利康 ● 模态：ADC ● PDUFA日期：不晚于2025年2月2日 ● 适应症类型/说明：癌症/转移性激素受体 (HR) 阳性、HER2 阴性（IHC 0、IHC 1+ 或 IHC 2+/ISH-）乳腺癌成人患者 ● 审评类型：标准审评 ● 申请递交时间：2024年1至2月 ● 审评情况：FDA 2024年4月2日接受BLA申请 19 Sublocade (buprenorphine depot injection) ● 开发商：Indivior ● 模态：小分子 ● PDUFA日期：2025年2月7日 ● 适应症类型/说明：中枢神经系统疾病/皮下注射阿片类激动剂，用于治疗中度至重度阿片类药物使用障碍 (OUD) ● 审评类型：优先审评 ● 申请递交时间：2024年8月 ● 审评情况：FDA 2024年10月7日接受申请 20 Rexulti (brexpiprazole) ● 开发商：Otsuka/Lundbeck ● 模态：小分子 ● PDUFA日期：2025年2月8日 ● 适应症类型/说明：中枢神经系统疾病/血清素-多巴胺活性调节剂 (SDAM) 非典型抗精神病药，新适应症，与舍曲林联合使用，用于治疗成人创伤后应激障碍 (PTSD) ● 审评类型：标准审评 ● 申请递交时间：2024年4月10日 ● 审评情况：FDA 2024年6月25日接受申请 21 CHIKV VLP (chikungunya vaccine, 此前称作PXVX0317，新生物制品) ● 开发商：Bavarian Nordic ● 模态：疫苗 ● PDUFA日期：2025年2月14日 ● 适应症类型/说明：传染病/用于成人和 12 岁及以上的儿童，预防基孔肯雅病毒感染 ● 审评类型：优先审评 ● 申请递交时间：2024年6月17日 ● 审评情况：突破性疗法认定 22 MenABCWY (meningococcal group A,B,C,Y/W-135 vaccine，新生物制品) ● 开发商：GSK ● 模态：疫苗 ● PDUFA日期：2025年2月14日 ● 适应症类型/说明：传染病/预防侵袭性脑膜炎球菌病 ● 审评类型：标准审评 ● 申请递交时间：2024年2月 ● 审评情况：FDA 2024年4月16日接受申请 23 Vimseltinib （新分子实体） ● 开发商：Deciphera (Ono Pharmaceutical) ● 模态：小分子 ● PDUFA日期：2025年2月17日 ● 适应症类型/说明：癌症/治疗腱鞘巨细胞瘤 (TGCT) 患者 ● 审评类型：优先审评 ● 申请递交时间：2024年6月 ● 审评情况：FDA 2024年8月15日接受申请 24 Mirdametinib （新分子实体） ● 开发商：SpringWorks Therapeutics ● 模态：小分子 ● PDUFA日期：2025年2月18日 ● 适应症类型/说明：癌症/治疗患有 1 型神经纤维瘤病相关丛状神经纤维瘤 (NF1-PN) 的儿童和成人患者 ● 审评类型：优先审评 ● 申请递交时间：2024年6月 ● 审评情况：FDA 2024年8月15日接受申请 25 ET-400 (hydrocortisone oral solution) ● 开发商：Eton Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年2月28日 ● 适应症类型/说明：内分泌、免疫学/氢化皮质酮(又称氢化可的松)，新型糖皮质激素口服溶液剂型，在室温下稳定，适用于婴儿和儿科患者 ● 审评类型：505(b)(2) NDA标准审评 ● 申请递交时间：2024年4月30日 ● 审评情况：FDA 2024年7月15日接受申请 26 Adcetris (brentuximab vedotin) ● 开发商：辉瑞 ● 模态：ADC ● PDUFA日期：2025年3月 ● 适应症类型/说明：癌症/CD30 靶向 ADC brentuximab vedotin 的新适应症，用于与来那度胺和利妥昔单抗联合治疗复发或难治性大 B 细胞淋巴瘤 (LBCL) ● 审评类型：标准审评 ● 申请递交时间：2024年5月 ● 审评情况：FDA 2024年7月30日接受申请 27 Susvimo (眼部植入物玻璃体注射 ranibizumab) ● 开发商：罗氏 ● 模态：单抗 ● PDUFA日期：不晚于2025年3月 ● 适应症类型/说明：眼科/ranibizumab的新适应症，该植入物可提供定制配方的 VEGF 抑制剂（不同于 Lucentis 玻璃体内注射），最长可使用六个月，用于治疗糖尿病性黄斑水肿和糖尿病性视网膜病变 ● 审评类型：标准审评 ● 申请递交时间：2024年5月 ● 审评情况：FDA 2024年7月18日接受申请 28 neffy (肾上腺素 1mg 鼻喷雾剂) ● 开发商：ARS Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年3月6日 ● 适应症类型/说明：免疫学/1mg 新浓度和扩大的儿科适应症，适用于体重 15-30 公斤儿童的 I 型过敏反应（包括过敏性休克）的鼻内紧急治疗 ● 审评类型：优先审评；505(b)(2) NDA ● 申请递交时间：2024年9月9日 29 Furoscix (furosemide, 皮下制剂) ● 开发商：scPharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年3月6日 ● 适应症类型/说明：心血管疾病/新适应症，皮下注射，治疗慢性肾病 (CKD) 患者因液体超负荷引起的水肿 ● 审评类型：标准审评 ● 申请递交时间：2024年5月 ● 审评情况：FDA 2024年7月25日接受申请 30 CT-P39 (omalizumab生物类似药) ● 开发商：Celltrion ● 模态：单抗 ● PDUFA日期：不晚于2025年3月11日 ● 适应症类型/说明：免疫学/Genetech的Xolair 的可互换生物类似药，用于治疗严重持续性过敏性哮喘、鼻息肉、荨麻疹和免疫球蛋白 E 介导的食物过敏等。 ● 审评类型：标准审评；351(k) BLA ● 申请递交时间：2024年3月11日 31 Condoliase (SI-6603，新生物制品) ● 开发商：Seikagaku/Ferring Pharmaceutical ● 模态：蛋白质 ● PDUFA日期：不晚于2025年3月14日 ● 适应症类型/说明：中枢神经系统疾病/成人腰椎间盘突出症 (LDH) 相关的根性腿痛 ● 审评类型：标准审评 ● 申请递交时间：2024年3月 ● 审评情况：FDA 2024年5月14日接受申请，2025年1月1日FDA顾问委员会会议 32 Revakinagene taroretcel (NT-501，新生物制品) ● 开发商：Neurotech Pharmaceuticals ● 模态：基因疗法 ● PDUFA日期：2025年3月18日 ● 适应症类型/说明：眼科/治疗 2 型黄斑毛细血管扩张症（MacTel，即特发性近焦点黄斑毛细血管扩张症） ● 审评类型：优先审评 ● 申请递交时间：2024年4月 ● 审评情况：FDA 2024年6月20日接受申请，PDUFA日期从最初的2024年12月17日延期 33 Camrelizumab (SHR-1210，新生物制品) ● 开发商：江苏恒瑞 (2023年10月授权Elevar) ● 模态：单抗 ● PDUFA日期：2025年3月20日 ● 适应症类型/说明：癌症/PD-1 抑制剂，与 Elevar 的 VEGFR-2 激酶抑制剂rivoceranib联合用于不可切除或转移性肝细胞癌 (uHCC) 的一线全身治疗 ● 审评类型：标准审评 ● 申请递交时间：2024年9月23日 ● 审评情况：首次BLA申请2023年5月17日，FDA 2024年5月27日签发完全回复函；再次申请获得接受。 34 Amvuttra (vutrisiran) ● 开发商：Alnylam ● 模态：寡核苷酸 ● PDUFA日期：2025年3月23日 ● 适应症类型/说明：心血管疾病、中枢神经系统疾病/ vutrisiran 新适应症，治疗伴有心肌病的转甲状腺素蛋白淀粉样变性 (ATTR-CM) ● 审评类型：使用一张优先审评券PRV ● 申请递交时间：2024年10月9日 ● 审评情况：FDA 2024年11月25日接受申请。 35 Rivoceranib （新分子实体） ● 开发商：Elevar Therapeutics (HLB LifeScience) ● 模态：小分子 ● PDUFA日期：不晚于2025年3月23日 ● 适应症类型/说明：癌症/与恒瑞医药的 PD-1 抑制剂Camrelizumab联合用于不可切除的肝细胞癌 (uHCC) 的一线治疗 ● 审评类型：标准审评 ● 申请递交时间：2024年9月23日 ● 审评情况：2023年5月17日递交的NDA申请被拒，再申请获得接受。 36 TLX007-CDx ● 开发商：Telix ● 模态：放射性示踪剂 ● PDUFA日期：2025年3月24日 ● 适应症类型/说明：癌症成像剂/前列腺癌诊断 ● 审评类型：标准审评 ● 申请递交时间：2024年5月27日 ● 审评情况：FDA 2024年7月24日接受申请。 37 Gepotidacin （新分子实体） ● 开发商：GSK ● 模态：小分子 ● PDUFA日期：2025年3月26日 ● 适应症类型/说明：传染病/口服治疗患有无并发症尿路感染 (uUTI) 的成年女性（≥40 公斤）和青少年（≥12 岁，≥40 公斤） ● 审评类型：优先审评 ● 申请递交时间：2024年7月 ● 审评情况：FDA 2024年10月16日接受申请。 38 Cardamyst (etripamil，新分子实体) ● 开发商：Milestone Pharmaceuticals ● 模态：小分子 ● PDUFA日期：2025年3月26日 ● 适应症类型/说明：心血管疾病/鼻腔喷雾剂，治疗阵发性室上性心动过速 (PSVT) ● 审评类型：标准审评 ● 申请递交时间：2024年3月28日 ● 审评情况：FDA 2023年12月签发RTF信函，之后重新递交申请，FDA 2024年5月26日接受申请。 39 Egrifta MDV (tesamorelin F8 formulation) ● 开发商：Theratechnologies ● 模态：多肽 ● PDUFA日期：不晚于2025年3月26日 ● 适应症类型/说明：代谢疾病/用于治疗 HIV 相关脂肪营养障碍 ● 审评类型：标准审评 ● 申请递交时间：2024年11月26日 ● 审评情况：FDA接受之前遭受一份完整回复函。 40 DCCR (diazoxide choline) ● 开发商：Soleno Therapeutics ● 模态：小分子 ● PDUFA日期：2025年3月27日 ● 适应症类型/说明：内分泌疾病/治疗 4 岁及以上患有贪食症的普拉德-威利综合征 (PWS) 患者 ● 审评类型：优先审评 ● 申请递交时间：2024年6月27日 ● 审评情况：最初PDUFA日期为2024年12月27日，延期。 41 Fitusiran （新分子实体） ● 开发商：赛诺菲 ● 模态：寡核苷酸 ● PDUFA日期：2025年3月28日 ● 适应症类型/说明：血液疾病/成人和青少年A型或B型血友病 A 或 B 患者 ● 审评类型：标准审评 ● 申请递交时间：2024年3月 ● 审评情况：B型血友病疗法获得突破性疗法认定。 42 Darzalex Faspro (daratumumab/ hyaluronidase-fihj) ● 开发商：强生/Genmab ● 模态：抗体/酶 ● PDUFA日期：不晚于2025年3月30日 ● 适应症类型/说明：癌症/使用 Halozyme 的 Enhanze 技术皮下注射daratumumab，用于治疗新诊断的多发性骨髓瘤 (NDMM) 成人患者。 ● 审评类型：优先审评 ● 申请递交时间：2024年9月30日 43 Sepiapterin (PTC923, 最初称为CNSA-001，新分子实体) ● 开发商：PTC Therapeutics ● 模态：小分子 ● PDUFA日期：不晚于2025年3月30日 ● 适应症类型/说明：先天性缺陷/治疗儿童和成人苯丙酮尿症 (PKU) 患者，包括所有年龄段和疾病亚型 ● 审评类型：标准审评 ● 申请递交时间：2024年7月30日 ● 审评情况：FDA 2024年10月1日接受申请。 44 Jynneos (smallpox/monkeypox 疫苗) ● 开发商：Bavarian Nordic ● 模态：疫苗 ● PDUFA日期：不晚于2025年3月31日 ● 适应症类型/说明：传染病/基于减毒活痘苗病毒的新型冻干非复制型疫苗配方，适合长期储存，用于预防 18 岁及以上成年人的天花和猴痘 ● 审评类型：标准审评 ● 申请递交时间：2024年5月31日 Ref. Pink Sheet User Fee Goals Date. Retrieved on 02. 01. 2025. END 【智药研习社近期直播】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

优先审批免疫疗法生物类似药细胞疗法突破性疗法

100 项与托珠单抗生物类似药 (Celltrion) 相关的药物交易

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研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
新型冠状病毒感染	美国	Celltrion, Inc.	2025-01-24
巨细胞动脉炎	美国	Celltrion, Inc.	2025-01-24
多关节型幼年特发性关节炎	韩国	Celltrion, Inc.	2024-12-20
类风湿关节炎	韩国	Celltrion, Inc.	2024-12-20
全身型幼年特发性关节炎	韩国	Celltrion, Inc.	2024-12-20

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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


NCT05489224 (CTgov)	临床3期	类风湿关节炎	471	CT-P47	構鏇膚鹹顧獵鏇繭衊繭(窪觸廠鬱構壓憲蓋夢糧) = 範窪憲廠鏇積鏇壓選簾襯顧壓淵鏇選淵廠網餘 (齋鬱鏇糧糧壓夢衊壓鏇, 夢窪觸鹽鏇網製鑰鏇範 ~ 糧憲選鹽鹹壓積願憲鬱) 更多	-	2024-10-08
NCT05617183 (EULAR 12024 \| EULAR 22024) 人工标引	临床1期	类风湿关节炎	314	CT-P47 auto-injector	糧繭鏇簾願觸顧遞觸選(夢淵製艱遞網範醖觸蓋) = 鹽觸鏇夢選糧積範築願艱簾醖衊鹽範鹽鏇糧獵 (蓋製艱鑰餘鏇觸膚鏇餘 ) 更多	积极	2024-06-14
				CT-P47 pre-filled syringe	糧繭鏇簾願觸顧遞觸選(夢淵製艱遞網範醖觸蓋) = 願顧鬱遞膚糧壓獵選網艱簾醖衊鹽範鹽鏇糧獵 (蓋製艱鑰餘鏇觸膚鏇餘 ) 更多
EULAR2024 人工标引	临床1期	-	132	CT-P47	繭廠積憲齋廠鏇構醖構(襯蓋衊鏇遞淵餘襯壓築) = 淵窪遞餘膚餘憲鏇壓鬱鑰繭選廠積選觸觸窪餘 (製艱網淵簾鑰積積鹽淵 ) 更多	积极	2024-06-05
				EU-tocilizumab	繭廠積憲齋廠鏇構醖構(襯蓋衊鏇遞淵餘襯壓築) = 餘遞獵願繭範鹹簾鹹鏇鑰繭選廠積選觸觸窪餘 (製艱網淵簾鑰積積鹽淵 ) 更多