预约演示
更新于：2025-03-01
Etranacogene dezaparvovec
更新于：2025-03-01
概要
基本信息
原研机构 |
非在研机构- |
最高研发阶段批准上市 |
首次获批日期 美国 (2022-11-22), |
最高研发阶段(中国)- |
特殊审评优先审评 (美国)、孤儿药 (美国)、孤儿药 (韩国)、孤儿药 (澳大利亚)、附条件批准 (欧盟)、加速审评 (欧盟)、附条件批准 (英国)、附条件批准 (澳大利亚)、突破性疗法 (美国) |
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结构/序列
Sequence Code 10257960
来源: *****
Sequence Code 547989533
来源: *****
关联
6
项与 Etranacogene dezaparvovec 相关的临床试验NCT06003387
Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies
JPRN-jRCT2033230266
Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy and Safety of a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, CSL222) Administered to Japanese Adult Male Subjects with Severe or Moderately Severe Hemophilia B
NCT05962398
An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B
100 项与 Etranacogene dezaparvovec 相关的临床结果
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100 项与 Etranacogene dezaparvovec 相关的转化医学
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100 项与 Etranacogene dezaparvovec 相关的专利（医药）
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38
项与 Etranacogene dezaparvovec 相关的文献（医药）2025-01-01PHARMACOECONOMICS
Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability
Article
作者: Vreman, Rick A ; Goettsch, Wim G ; Callenbach, Marcelien H E ; Mantel-Teeuwisse, Aukje K ; Leopold, Christine
OBJECTIVE:
To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions.
METHODS:
A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population. To demonstrate the potential of the framework, three case studies were used: onasemnogene abeparvovec (Zolgensma®), brexucabtagene autoleucel (Tecartus®) and etranacogene dezaparvovec (Hemgenix®). A hypothetical case study was used to illustrate the output of the calculation tool.
RESULTS:
Part 1 of the framework presents steps for matching a suitable reimbursement and payment model with the disease and treatment characteristics. The reimbursement and payment models are further specified in Part 2. Part 3 guides end users through the setup of a calculation tool with which the financial impact can be calculated of two payment models: a price discount model and an outcome-based spread payment model with a discount. Part 4 concerns the output of the calculation tool, showing how different payment models lead to different financial consequences under three assumptions of longer term effectiveness.
CONCLUSIONS:
The presented framework provides decision makers with insight into the financial consequences of their chosen payment model under different assumptions. This can aid reimbursement negotiations by clarifying the optimal choice given a therapy's characteristics.
2025-01-01JOURNAL OF THROMBOSIS AND HAEMOSTASIS
Invasive procedures and surgery following etranacogene dezaparvovec gene therapy in people with hemophilia B
Article
作者: Castaman, Giancarlo ; Quon, Doris V ; Klamroth, Robert ; O’Connell, Niamh ; Le Quellec, Sandra ; Crary, Shelley E. ; Gomez, Esteban ; Monahan, Paul E ; Monahan, Paul E. ; Quon, Doris V. ; van der Valk, Paul ; Coppens, Michiel ; Lemons, Richard ; O'Connell, Niamh ; Kampmann, Peter ; Crary, Shelley E ; Symington, Emily
BACKGROUND:
Little information regarding the management of invasive procedures in people with hemophilia B (HB) after undergoing gene therapy is available. Here, we report the management of invasive procedures in people with severe or moderately severe HB who had previously been treated with etranacogene dezaparvovec in the phase 2b and phase 3 Health Outcomes with Padua Gene; Evaluation in Hemophilia B clinical trials (NCT03489291 and NCT03569891).
OBJECTIVES:
The objective of this study was to describe the use of exogenous FIX, endogenous FIX activity prior to invasive procedures, and peri- and postoperative bleeds in participants who underwent invasive procedures after receiving etranacogene dezaparvovec gene therapy.
METHODS:
This retrospective analysis included invasive procedures performed within 3 and 2 years following a single infusion of 2 × 1013 gc/kg of etranacogene dezaparvovec in participants in the phase 2b and Health Outcomes with Padua Gene; Evaluation in Hemophilia B trials, respectively. Data for factor (F)IX dosing, duration of postoperative FIX use, FIX activity prior to invasive procedures, and postoperative bleeds were collected and analyzed.
RESULTS:
The analysis included 64 procedures in 29 participants: 9 major surgeries, 24 minor surgeries, 11 endoscopies, 3 endoscopies with biopsy/polypectomy, and 17 dental procedures. Uncontaminated endogenous FIX activity corresponded to mild hemophilia or normal levels prior to 98% of all procedures, with a median endogenous FIX activity of 43.8 IU/dL (range, 3.1-113 IU/dL). All major surgeries were managed with exogenous FIX, 67% with ≤4 days of FIX infusion. Most minor surgeries (88%), endoscopies (82%), and dental procedures (94%) were managed with no or a single FIX infusion. Postoperative bleeds occurred after 1 minor surgery and 4 dental procedures. There were no symptomatic thrombotic events or FIX inhibitor developments.
CONCLUSION:
Etranacogene dezaparvovec has the potential to facilitate perioperative management in people with HB by reducing the need for perioperative exogenous FIX and its associated risks.
2024-12-31JOURNAL OF MEDICAL ECONOMICS
Analysis of long-term clinical and cost impact of etranacogene dezaparvovec for the treatment of hemophilia B population in the United States
Article
作者: Sivamurthy, Krupa ; McDade, Cheryl ; Rouse, Robert ; Thiruvillakkat, Kris ; Yan, Songkai ; Wilson, Michele
INTRODUCTION:
Etranacogene dezaparvovec (EDZ), Hemgenix, is a gene therapy recently approved for people with hemophilia B (PwHB).
OBJECTIVE:
To estimate long-term clinical impact and cost of EDZ in the United States (US).
METHODS:
A decision-analytic model was developed to evaluate the long-term impact of introducing EDZ for PwHB over a 20-year time horizon. Factor IX (FIX) prophylaxis comparator was a weighted average of different FIX prophylaxis regimens based on US market share data. We compared a scenario in which EDZ is introduced in the US versus a scenario without EDZ. Clinical inputs (annualized FIX-treated bleed rate; adverse event rates) were obtained from HOPE-B phase 3 trial. EDZ durability input was sourced from an analysis predicting long-term FIX activity with EDZ. EDZ one-time price was assumed at $3.5 million. Other medical costs, including FIX prophylaxis, disease monitoring, bleed management, and adverse events were from literature. The model estimated annual and cumulative costs, treated bleeds, and joint procedures over 20 years from EDZ introduction.
RESULTS:
Approximately 596 PwHB were eligible for EDZ. EDZ uptake was estimated to avert 11,282 bleeds and 64 joint procedures over 20 years. Although adopting EDZ resulted in an annual excess cost over years 1-5 (mean: $53 million annually, total $265 million), annual cost savings were achieved beginning in year 6 (mean: $172 million annually; total $2.58 billion in years 6-20). The total cumulative 20-year cost savings was $2.32 billion, with cumulative cost savings beginning in year 8.
CONCLUSION:
Introducing EDZ to treat PwHB is expected to result in cost savings and patient benefit over 20 years. Initiating PwHB on EDZ sooner can produce greater and earlier savings and additional bleeds avoided. These results may be a conservative estimate of the full value of EDZ, as PwHB would continue to accrue savings beyond 20 years.
290
项与 Etranacogene dezaparvovec 相关的新闻（医药）2025-02-27
基因疗法上市批准
2025-02-24
基因疗法上市批准并购
2025-02-22
·药事纵横
基因疗法临床3期上市批准临床结果引进/卖出
100 项与 Etranacogene dezaparvovec 相关的药物交易
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研发状态
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 血友病B | 美国 | 2022-11-22 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床3期 | 54 | 餘醖窪製繭夢壓襯鹽顧(範淵鹽簾夢鏇蓋淵鏇願) = 構膚餘齋醖餘選繭衊壓 觸顧遞夢鑰淵襯壓積糧 (窪膚廠淵廠壓膚製醖構 ) 更多 | 积极 | 2025-02-07 | |||
临床2/3期 | 29 | Etranacogene dezaparvovec 2x10^13 gc/kg | 願簾廠遞鬱襯醖築繭醖(醖顧築壓夢廠繭膚構網) = 襯鑰鬱鏇鏇製簾顧網齋 鹹選積構觸願網選製淵 (憲淵觸築艱夢簾醖顧蓋, 3.1 ~ 113) 更多 | 积极 | 2024-09-01 | ||
临床3期 | - | 壓遞醖鏇鑰齋鹽鏇餘遞(鏇網願壓廠獵鏇選簾築) = 餘鏇選餘繭蓋觸糧膚繭 醖壓製鏇齋鹽鬱餘繭窪 (積遞築糧觸醖願鹹構網 ) 更多 | 积极 | 2024-06-28 | |||
临床2/3期 | 57 | 築醖蓋衊觸繭製鬱簾齋(壓網顧衊餘艱淵憲簾醖) = 糧鹽鏇觸願範觸簾獵衊 艱範構鏇鏇製構夢襯艱 (衊願鏇鑰鹹鹹糧繭範衊 ) | - | 2023-12-09 | |||
临床3期 | 54 | 鬱網窪艱壓艱膚醖鬱願(艱壓範齋獵範範膚窪選) = 顧製鹹醖願範夢繭鹹齋 衊製醖廠齋艱鹹範齋鬱 (蓋膚簾壓齋壓蓋憲衊齋 ) 更多 | 积极 | 2022-11-15 | |||
N/A | - | 鹹膚觸襯艱糧夢顧簾簾(鏇製鹹壓構範積觸築蓋) = 鑰簾艱襯壓願遞獵獵糧 襯衊襯築窪糧築襯衊鹹 (糧淵獵壓淵廠築製憲獵, 4.5 ~ 122.9) | - | 2022-10-11 | |||
Standard-of-care FIX prophylaxis | 鹹膚觸襯艱糧夢顧簾簾(鏇製鹹壓構範積觸築蓋) = 簾網襯餘顧鏇鏇衊衊齋 襯衊襯築窪糧築襯衊鹹 (糧淵獵壓淵廠築製憲獵, 10.3 ~ 57.9) | ||||||
临床3期 | 血友病 AAV5 neutralizing antibodies | 54 | Etranacogene dezaparvovec 2x10^13 gc/kg | 鑰壓夢顧觸憲醖鹹顧構(壓窪艱齋醖壓艱構鬱顧) = 壓遞選廠鏇淵積壓餘憲 構淵憲繭願醖製構醖願 (衊淵醖鏇壓憲網糧製鏇, 18.7) | 积极 | 2022-05-02 | |
临床3期 | 54 | 餘鑰鏇範壓鹹築膚糧鏇(築繭衊遞鹹願齋襯築遞) = 廠餘鏇醖糧糧餘積窪餘 積糧觸廠鹹膚繭選顧鑰 (顧繭夢觸窪鹽鬱鹹鬱製 ) | - | 2021-07-17 | |||
临床3期 | 血友病B AAV5 NAbs | FIX | 54 | 淵選艱願鹽憲遞鬱觸襯(繭壓鹹鹽遞繭餘願積壓) = 糧鹹鑰範憲遞製壓艱網 鑰艱餘築窪淵齋壓窪鏇 (艱糧餘繭觸顧衊製觸醖 ) | - | 2021-07-17 | ||
淵選艱願鹽憲遞鬱觸襯(繭壓鹹鹽遞繭餘願積壓) = 齋選憲鏇艱齋網觸憲簾 鑰艱餘築窪淵齋壓窪鏇 (艱糧餘繭觸顧衊製觸醖 ) | |||||||
临床3期 | 54 | 淵願鏇夢醖醖範構餘網(選糧醖艱網蓋獵願艱網) = 廠壓廠構廠鬱夢築衊襯 構網鏇構遞鹹鑰積簾廠 (鹽獵醖顧獵艱製願鑰網 ) | - | 2021-06-09 |
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