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更新于：2026-07-07
Donidalorsen
更新于：2026-07-07
概要
基本信息
非在研机构- |
最高研发阶段批准上市 |
首次获批日期 美国 (2025-08-21), |
最高研发阶段(中国)- |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (澳大利亚) |
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结构/序列
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Sequence Code 538916969
来源: *****
关联
10
项与 Donidalorsen 相关的临床试验NCT07298447
An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
JPRN-jRCT2051240291
A single-center, randomized, double-blind, placebo-controlled, parallel-group phase 1 trial to assess the pharmacokinetics, pharmacodynamics, safety, and tolerability of donidalorsen by multiple-dose subcutaneous administration in healthy adults
NCT05392114
An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
100 项与 Donidalorsen 相关的临床结果
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100 项与 Donidalorsen 相关的转化医学
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100 项与 Donidalorsen 相关的专利（医药）
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13
项与 Donidalorsen 相关的文献（医药）2026-06-01ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY
Donidalorsen for hereditary angioedema
Article
作者: de Lange, Mats ; Tachdjian, Raffi ; Newman, Kenneth B ; Bernstein, Jonathan A ; Manning, Michael E ; Raasch, Jason ; Deng, Yiwen ; Craig, Timothy ; Cohn, Danny M ; Bordone, Laura ; Lumry, William R
BACKGROUND:
Hereditary angioedema (HAE) is a rare disorder caused by kallikrein-kinin system dysregulation. Donidalorsen is a prekallikrein-directed antisense oligonucleotide that can rebalance this system by reducing prekallikrein production and is indicated for prophylaxis to prevent HAE attacks in patients 12 years of age and older.
OBJECTIVE:
To report results from a 4-year, phase 2, open-label extension study of donidalorsen (NCT04307381).
METHODS:
The study on-treatment period included fixed (weeks 0-12) and flexible (weeks 16-208) treatment periods. Patients received donidalorsen 80 mg subcutaneously every 4 weeks (Q4W) in the fixed treatment period and could receive 80 mg every 8 weeks (Q8W) in the flexible treatment period if attack free for greater than or equal to 12 weeks or 100 mg Q4W if disease was not adequately controlled. End points are summarized through the end of the study (week 208).
RESULTS:
Of 17 patients with HAE-C1INH-type 1 or HAE-C1INH-type 2, 13 (76%) completed 4 years of treatment (median exposure: 3.99 years). Eight patients changed to donidalorsen Q8W; 5 stayed on Q8W to study completion. One patient received 100 mg Q4W. Mean monthly HAE attack rates decreased by 97% overall from baseline to the on-treatment period, with 97% (Q4W) and 83% (Q8W) decreases to the flexible treatment period. The median longest attack-free interval was 990 days. No serious treatment-emergent adverse events (TEAEs) occurred. One patient (6%) (100 mg Q4W) discontinued due to a TEAE (injection-site discoloration). Seven patients (41%) reported treatment-related TEAEs; the only treatment-related TEAEs reported in more than 1 patient were injection-site reactions.
CONCLUSION:
In this 4-year study, donidalorsen led to sustained reductions in monthly HAE attack rate with an acceptable safety profile.
TRIAL REGISTRATION:
ClinicalTrials.gov Identifier: NCT04307381 (https://www.
CLINICALTRIALS:
gov/study/NCT04307381).
2026-03-01World Allergy Organization Journal
Association of muscle instability and long-term prophylaxis in hereditary angioedema
Article
作者: Hitomi, Hirofumi ; Hollers, Eleanor ; Grim, Kara ; Yu, Yunting ; Luong, Long ; Richwine, Kristina ; Al-Shaikhly, Taha ; Germak-Sovereign, Rita ; Craig, Timothy ; Sheetz, James
Introduction:
Hereditary angioedema (HAE) types 1 and 2 are caused by C1 inhibitor deficiency or dysfunction, leading to increased prekallikrein activity and bradykinin production. HAE causes vasodilation and edema resulting in obstruction of the upper airway, gastrointestinal symptoms, and skin swelling. Evidence of involvement of other organ systems has been sparse. Herein, we demonstrate evidence of creatinine kinase (CK) elevation in HAE patients suggesting an effect of bradykinin on skeletal muscle with subsequent improvement with long term prophylaxis (LTP).
Methods:
CK levels from participants with type 1 or 2 HAE enrolled in the Phase 2 and 3 clinical trials evaluating the safety and efficacy of donidalorsen for LTP in patients with HAE, was measured at baseline (before treatment initiation) and Week 17 (for participants enrolled in Phase 2 Study) and Week 25 (for participants enrolled in Phase 3 study). Mixed effect model with repeated measures was used to assess the influence of time and treatment (donidalorsen vs. placebo) on serum CK levels.
Results:
CK levels were available from 20 patients enrolled in the Phase 2 study and the mean CK level was numerically lower by Week 17; however, these results were not statistically significant. Among the 90 participants enrolled in the Phase 3 study who had CK levels checked at baseline and Week 25, a significantly lower CK level at Week 25 was observed among those receiving Q4W donidalorsen, but not among those receiving donidalorsen Q8W or placebo.
Conclusion:
Bradykinin appears to cause instability of skeletal muscle, causing CK release with even minor exercise. The effect of increases in bradykinin in HAE on muscle needs further research but may account for some of the atypical HAE symptoms patients often describe and which are noted in quality-of-life assessments. LTP, therefore, may confer additional benefits beyond reduction of HAE symptoms, potentially contributing to stabilization of skeletal muscle and improvement of fatigue and weakness.
2026-02-01CPT-Pharmacometrics & Systems Pharmacology
Population Pharmacokinetic/Pharmacodynamic Modeling of Donidalorsen, an Antisense Oligonucleotide in Development for Prophylaxis of Hereditary Angioedema
Article
作者: Gao, Xiang ; Diep, John K. ; Cohn, Danny M. ; Singh, Pratap ; Dorow, Steve ; Bordone, Laura ; Liu, Miao ; Newman, Kenneth B.
ABSTRACT:
Hereditary angioedema (HAE) is a rare disorder linked to kallikrein‐kinin system dysregulation, which leads to uncontrolled activation of plasma prekallikrein. Donidalorsen is an antisense oligonucleotide designed to selectively degrade prekallikrein messenger RNA and thereby reduce prekallikrein production. We aimed to develop population pharmacokinetic and pharmacokinetic/pharmacodynamic models of donidalorsen and evaluate the impact of potential intrinsic/extrinsic covariates on exposure and prekallikrein response. Plasma donidalorsen and prekallikrein data were obtained from phase 1 to 3 studies in healthy volunteers (NCT03263507, 721744‐CS9) and adult and adolescent patients with HAE (NCT04030598, NCT05139810). The evaluated doses were 20, 40, 60, and 80 mg every 4 weeks (Q4W) and 80 mg every 8 weeks (Q8W), administered subcutaneously over 13–21 weeks. Donidalorsen pharmacokinetics were well described by a linear 2‐compartment model with first‐order absorption. The population terminal elimination half‐life was 31.4 days. Prekallikrein was well described by an indirect response model with inhibition of prekallikrein production by donidalorsen. Covariate analysis identified body weight as the main factor affecting pharmacokinetic exposure; however, this effect was not considered clinically significant. The developed population pharmacokinetic/pharmacodynamic model well characterized the donidalorsen exposure–prekallikrein response relationship. Modeling analyses support that no dose adjustment is needed with respect to intrinsic/extrinsic factors in adults and adolescents with HAE. The nearly identical simulated pharmacokinetic or prekallikrein time courses for Q4W versus monthly dosing and for Q8W versus every‐2‐month dosing regimens support switching to more convenient regimens for patients.
100 项与 Donidalorsen 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 |
|---|---|---|---|
| 遗传性血管性水肿 | 美国 | 2025-08-21 |
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临床结果
临床结果
适应症
分期
评价
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临床2期 | 20 | (HAE-1/HAE-2) | 齋膚範築積鏇壓製夢繭 = 鬱鏇選艱壓顧蓋襯廠繭 壓鬱範觸願廠餘艱選觸 (鏇餘鏇鑰餘鹹壓鏇蓋廠, 鏇餘襯願構顧築鹹築淵 ~ 簾遞積醖膚淵積遞膚鹹) | - | 2026-05-08 | ||
(HAE-nC1-INH) | 齋膚範築積鏇壓製夢繭 = 構鏇廠鬱築製鹹壓築壓 壓鬱範觸願廠餘艱選觸 (鏇餘鏇鑰餘鹹壓鏇蓋廠, 鏇簾憲鹹衊鑰壓襯醖廠 ~ 簾壓壓遞製襯膚築襯糧) | ||||||
临床3期 | 4 | (Switched from prior HAE LTP) | 鏇鹽淵糧艱範範鹽構艱(餘膚襯網膚遞齋襯獵糧) = 繭壓餘鹹網遞選獵範鏇 鏇築蓋觸餘窪壓獵選鏇 (艱鏇廠築鹹齋範構襯淵 ) 更多 | 积极 | 2026-02-27 | ||
临床3期 | 65 | (32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat.) | 鹽鹹鏇夢鹽獵鑰蓋選積(壓遞顧壓網齋網選餘鹹) = 選壓襯醖齋襯範網膚壓 餘膚廠繭選積醖醖窪選 (願選壓憲網窪構襯遞繭 ) 更多 | 积极 | 2025-07-17 | ||
临床3期 | 91 | (Pooled Placebo) | 獵構憲蓋築顧鹹鏇鏇餘(鏇獵艱鹽網餘鏇製鹹積) = 簾獵顧選壓築襯齋膚蓋 窪鬱簾鏇餘選積鑰艱觸 (憲簾顧醖觸獵醖鏇餘顧, 鬱窪簾餘壓壓製壓選觸 ~ 鑰獵憲窪獵醖構窪餘廠) 更多 | - | 2025-03-06 | ||
(Cohort A: Donidalorsen 80 mg) | 獵構憲蓋築顧鹹鏇鏇餘(鏇獵艱鹽網餘鏇製鹹積) = 構憲遞顧膚艱糧艱積鹽 窪鬱簾鏇餘選積鑰艱觸 (憲簾顧醖觸獵醖鏇餘顧, 糧積鬱鹽繭鬱衊夢獵繭 ~ 廠糧鬱憲艱簾糧遞廠鬱) 更多 | ||||||
临床3期 | 7 | Donidalorsen Q4W | 醖鹽鏇積壓顧鬱壓簾鬱(鹽製艱鬱廠窪繭艱糧顧) = 構鬱築願醖範鹽築蓋醖 窪廠範憲夢鹹構膚窪艱 (製選觸餘繭壓範衊鬱艱 ) 更多 | 积极 | 2025-02-28 | ||
Donidalorsen Q8W | 醖鹽鏇積壓顧鬱壓簾鬱(鹽製艱鬱廠窪繭艱糧顧) = 觸獵構願繭鹹憲窪艱齋 窪廠範憲夢鹹構膚窪艱 (製選觸餘繭壓範衊鬱艱 ) 更多 | ||||||
临床2期 | - | 鏇憲夢壓齋獵簾鏇鬱獵(遞夢願膚窪鑰鏇製衊簾) = 憲選衊窪範網顧選遞糧 鑰艱壓醖積選鏇壓簾築 (構糧構壓醖選築餘製夢 ) | 不佳 | 2024-12-01 | |||
Placebo | 鏇憲夢壓齋獵簾鏇鬱獵(遞夢願膚窪鑰鏇製衊簾) = 鏇壓鑰淵餘憲餘鑰壓鬱 鑰艱壓醖積選鏇壓簾築 (構糧構壓醖選築餘製夢 ) | ||||||
临床2期 | 17 | 願糧顧衊網衊衊鑰廠鏇(繭簾淵襯艱憲壓艱製積) = 積鏇餘壓醖鬱餘遞獵艱 積鹹齋鹽夢製鏇觸壓簾 (憲廠製觸顧廠獵鬱醖衊, 7.81) | 积极 | 2024-10-30 | |||
膚製鏇繭衊窪壓衊製鑰(窪窪鏇鹽簾構願衊網鹽) = 壓廠鏇膚網簾糧壓糧製 鬱範鏇遞艱繭餘鹹醖憲 (艱獵顧顧鹽鬱選製艱觸 ) 更多 | |||||||
临床3期 | 64 | 顧鹹窪構廠鹽襯鑰構顧(構衊淵願襯構鹽鑰壓淵) = 觸廠鬱襯鏇憲網艱鬱鹽 範獵選齋願顧蓋淵選願 (獵獵築繭廠憲顧襯觸憲 ) 更多 | 积极 | 2024-10-24 | |||
临床3期 | 遗传性血管性水肿 kallikrein-kinin system dysregulation | 83 | 艱顧衊鏇選廠窪壓壓壓(築範憲鹽網壓鑰醖願積) = Patients previously on placebo also reported improvements in AE-QoL total scores (24 points from OASIS-HAE baseline) 簾窪鹽鹽衊製醖觸遞選 (獵繭觸選淵鹹繭積醖積 ) 更多 | 积极 | 2024-10-24 | ||
临床3期 | 90 | Donidalorsen 80 mg every 4 weeks | 構餘餘艱夢構鏇構夢淵(齋餘製齋觸鬱遞壓遞襯) = 憲範製繭構齋窪範築簾 願夢簾齋構衊願顧網艱 (積願衊簾觸襯廠夢糧網, 0.27 ~ 0.73) 更多 | 积极 | 2024-07-04 | ||
Donidalorsen 80 mg every 8 weeks | 構餘餘艱夢構鏇構夢淵(齋餘製齋觸鬱遞壓遞襯) = 蓋蓋觸淵餘齋積鏇築壓 願夢簾齋構衊願顧網艱 (積願衊簾觸襯廠夢糧網, 0.65 ~ 1.59) 更多 |
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