预约演示
更新于：2025-08-21
Tofersen
托夫生
更新于：2025-08-21
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 (2023-04-25), |
最高研发阶段(中国)批准上市 |
特殊审评孤儿药 (美国)、附条件批准 (中国)、孤儿药 (日本)、孤儿药 (韩国)、优先审评 (美国)、加速批准 (美国)、孤儿药 (澳大利亚) |
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结构/序列
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Sequence Code 530439811
来源: *****
关联
7
项与 托夫生 相关的临床试验JPRN-jRCT2071240022
Japan Expanded Clinical Study (ECS) to Provide Tofersen (BIIB067) to Patients With Amyotrophic Lateral Sclerosis (ALS) Associated with a Mutation in the Superoxide Dismutase 1 (SOD1) Gene
NCT04856982
A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
NCT03764488
A Phase 1, Safety, Tolerability, and Distribution Study of a Microdose of Radiolabeled BIIB067 Co-administered With BIIB067 to Healthy Adults
100 项与 托夫生 相关的临床结果
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100 项与 托夫生 相关的转化医学
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100 项与 托夫生 相关的专利（医药）
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81
项与 托夫生 相关的文献（医药）2025-09-01CLINICAL NEUROPHYSIOLOGY
Noninvasive quantification of fasciculations to track tofersen therapy in superoxide dismutase 1 amyotrophic lateral sclerosis
Letter
作者: Bashford, James ; Crook-Rumsey, Mark ; Bilgorai, Judith ; Shaw, Christopher ; Kelly, Gillian ; Carobin, Anna ; Scott, James ; Sreedharan, Jemeen
2025-08-09ANNALS OF NEUROLOGY
Targeted Proteomics upon Treatment with Tofersen Identifies Novel Response Markers for Superoxide Dismutase 1-Linked Amyotrophic Lateral Sclerosis.
Article
作者: Vidovic, Maximilian ; Schuster, Joachim ; Brenner, David ; Freischmidt, Axel ; Grosskreutz, Julian ; Schöberl, Florian ; Tumani, Hayrettin ; Oeckl, Patrick ; Hermann, Andreas ; Elmas, Zeynep ; Dorst, Johannes ; Witzel, Simon ; Prudlo, Johannes ; Hagenacker, Tim ; Lingor, Paul ; Meyer, Thomas ; Günther, Kornelia ; Bachhuber, Franziska ; Ludolph, Albert C ; Knehr, Antje ; Reilich, Peter ; Weyen, Ute ; Petri, Susanne ; Wiesenfarth, Maximilian ; Ruf, Wolfgang Philipp ; Catanese, Alberto ; Ho, Ritchie ; Steffke, Christina ; Weydt, Patrick ; Uzelac, Zeljko ; Baskar, Karthik ; Wolf, Joachim ; Haarmeier, Thomas ; Weishaupt, Jochen H ; Günther, René ; German, Alexander ; Regensburger, Martin
OBJECTIVE:
Tofersen is the first effective and approved therapy for superoxide dismutase 1 (SOD1)-associated amyotrophic lateral sclerosis (ALS [SOD1-ALS]). Following treatment with tofersen, neurofilament levels in patients' cerebrospinal fluid (CSF) and serum seem to respond earlier than clinical parameters. This evidence prompted us to hypothesize that this novel treatment could provide an opportunity to identify additional biomarkers responsive to therapy in SOD1-ALS.
METHODS:
We investigated a panel of 120 neural, glial, and inflammatory markers in CSF and serum samples longitudinally collected from a total of 28 SOD1-ALS patients at baseline, and after 3, 6 and 12 months of treatment with tofersen, followed by validation with conventional methodology.
RESULTS:
We identified a set of proteins, including neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40 and amyloid-beta 1-42, neuropeptide Y (NPY), and ubiquitin C-terminal hydrolase L1 (UCHL1), whose CSF levels both differed between SOD1-ALS and the control group, and were responsive to tofersen at 3 and 6 months after treatment initiation. Another group of markers, including the neuropentraxin (NPTX) family members NPTX1, NPTX2 and NPTXR, did not separate untreated SOD1-ALS from controls, but was responsive to tofersen. At 12 months on tofersen the levels of neurofilament light chain, neurofilament heavy chain, NPTX1, NPTX2, and NPTXR remained reduced compared with baseline, and correlated with the clinical response to tofersen. Consistent with increasing CSF pleocytosis and intrathecal immunoglobulin production, inflammatory markers were significantly increased after 12 months of treatment.
INTERPRETATION:
Our results highlight a complex, time-dependent differential response of CSF biomarkers to tofersen treatment, and may pave the way for developing a panel of responsive proteins to make biomarker endpoints more robust in clinical trials for SOD1-ALS and beyond. ANN NEUROL 2025.
2025-08-01MOLECULAR THERAPY
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
Article
作者: Harkins, Ashley L ; Furgal, Raymond ; Gilbert, James W ; Summers, Ashley ; McHugh, Nicholas ; Hassler, Matthew R ; Dogan, Elif O ; Henninger, Nils ; Brown, Robert H ; Belgrad, Jillian ; Greer, Paul ; Mocarski, Kit ; Godinho, Bruno M D C ; Weiss, Alexandra ; Rivera Flores, Iris Valeria ; Wightman, Nicholas ; Ferguson, Chantal ; Yamada, Nozomi ; Echeverria, Dimas ; Bramato, Brianna ; Khvorova, Anastasia ; Monopoli, Kathryn ; Yamada, Ken ; Cooper, David
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative condition, with 20% of familial and 2%-3% of sporadic cases linked to mutations in the cytosolic superoxide dismutase (SOD1) gene. Mutant SOD1 protein is toxic to motor neurons, making SOD1 gene suppression a promising approach, supported by preclinical data and the 2023 Federal Drug Administration (FDA) approval of the GapmeR ASO targeting SOD1, tofersen. Despite the approval of an ASO and the optimism it brings to the field, the pharmacodynamics and pharmacokinetics of therapeutic SOD1 modulation can be improved. Here, we developed a chemically stabilized divalent siRNA scaffold (di-siRNA) that effectively suppresses SOD1 expression in vitro and in vivo. With optimized chemical modification, it achieves remarkable CNS tissue permeation and SOD1 silencing in vivo. Administered intraventricularly, di-siRNASOD1 extended survival in SOD1-G93A ALS mice, increasing survival beyond that previously seen in these mice by ASO modalities, slowed disease progression according to the standard ALS preclinical endpoints, and attenuated ALS neuropathology. These properties offer an improved therapeutic strategy for SOD1-mediated ALS and may extend to other dominantly inherited neurological disorders.
100 项与 托夫生 相关的药物交易
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研发状态
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 肌萎缩侧索硬化 | 欧盟 | 2024-05-30 | |
| 肌萎缩侧索硬化 | 冰岛 | 2024-05-30 | |
| 肌萎缩侧索硬化 | 列支敦士登 | 2024-05-30 | |
| 肌萎缩侧索硬化 | 挪威 | 2024-05-30 | |
| 1型肌萎缩侧索硬化 | 美国 | 2023-04-25 |
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临床结果
临床结果
适应症
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临床3期 | 1型肌萎缩侧索硬化 SOD1 Mutation | 108 | (ITT population) | 範積網鏇範網廠餘衊齋(顧鹽願積範糧範鹽範觸) = 顧餘淵醖構鑰憲膚鹹糧 網衊廠簾襯餘衊築鬱廠 (網顧壓醖顧遞製膚簾築 ) 更多 | 积极 | 2023-04-25 | |
Placebo (ITT population) | 範積網鏇範網廠餘衊齋(顧鹽願積範糧範鹽範觸) = 醖餘淵築糧廠廠鹹夢憲 網衊廠簾襯餘衊築鬱廠 (網顧壓醖顧遞製膚簾築 ) 更多 | ||||||
临床3期 | 1型肌萎缩侧索硬化 SOD1 Mutation | 108 | 顧積簾膚糧獵選積選齋(選蓋壓鹹積淵範淵膚範) = 齋積獵壓網廠齋淵鑰願 壓構淵築網簾築築鬱觸 (鹽襯艱製衊網醖範糧築 ) | 不佳 | 2022-09-22 | ||
placebo | 顧積簾膚糧獵選積選齋(選蓋壓鹹積淵範淵膚範) = 鏇齋窪窪選夢憲艱積夢 壓構淵築網簾築築鬱觸 (鹽襯艱製衊網醖範糧築 ) | ||||||
临床3期 | 1型肌萎缩侧索硬化 SOD1 | 108 | 鹽醖餘顧膚憲鑰衊築願(憲糧壓顧醖網艱夢願壓) = 襯繭齋鏇顧鹹鏇醖遞糧 淵鹽壓蓋構齋範製願廠 (艱艱鹹築簾選構膚繭廠 ) | 积极 | 2022-06-03 | ||
Placebo | 鹽醖餘顧膚憲鑰衊築願(憲糧壓顧醖網艱夢願壓) = 製積遞餘襯艱遞顧鏇膚 淵鹽壓蓋構齋範製願廠 (艱艱鹹築簾選構膚繭廠 ) | ||||||
临床3期 | 1型肌萎缩侧索硬化 SOD1 mutation | 108 | 鑰顧鑰願夢蓋選糧範網(積夢廠夢鬱築鏇鹽積簾) = 築窪鏇窪壓顧積鏇廠選 積遞膚淵鬱鑰壓製艱製 (鏇鑰鬱膚夢衊積壓範獵 ) 更多 | 积极 | 2021-10-17 | ||
Placebo | 鑰顧鑰願夢蓋選糧範網(積夢廠夢鬱築鏇鹽積簾) = 淵鹹範窪齋鑰廠醖壓淵 積遞膚淵鬱鑰壓製艱製 (鏇鑰鬱膚夢衊積壓範獵 ) 更多 | ||||||
临床1/2期 | 1型肌萎缩侧索硬化 SOD1 Mutation | 50 | 衊選窪範範鬱顧艱窪艱(襯糧觸鑰夢淵獵淵繭選) = 鬱範餘鏇網遞構範獵窪 糧觸構顧齋齋構窪艱鏇 (鏇觸繭膚鹽鹽築鹹網壓 ) 更多 | 积极 | 2020-07-09 | ||
Placebo | 齋餘遞鏇壓構壓繭觸願(襯積範蓋艱繭窪製廠艱) = 範獵觸窪願鹹遞遞窪糧 獵簾齋構願壓獵築廠選 (獵築壓淵鑰衊襯獵夢窪 ) | ||||||
临床1期 | 1型肌萎缩侧索硬化 SOD1 Mutation | 32 | 鹹憲鹹艱築夢簾膚膚鹹(襯廠壓襯鹽鑰淵糧膚製) = 鏇願繭製積淵醖糧範艱 積憲範簾築廠膚願齋壓 (窪鑰蓋襯顧構餘獵簾積 ) 更多 | 积极 | 2013-05-01 | ||
Placebo | - |
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