Last update 08 May 2025

Tofersen

Overview

Basic Info

Drug Type
ASO
Synonyms
ISIS-SOD1RX, Tofersen (JAN/USAN/INN), Tofersen Sodium
+ [10]
Target
Action
inhibitors
Mechanism
SOD1 gene inhibitors
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
United States (25 Apr 2023),
RegulationConditional marketing approval (China), Orphan Drug (United States), Priority Review (United States), Accelerated Approval (United States)
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Structure/Sequence

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External Link

KEGGWikiATCDrug Bank
D11811--

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Amyotrophic Lateral Sclerosis
Iceland
30 May 2024
Amyotrophic Lateral Sclerosis
European Union
30 May 2024
Amyotrophic Lateral Sclerosis
Liechtenstein
30 May 2024
Amyotrophic Lateral Sclerosis
Norway
30 May 2024
Amyotrophic Lateral Sclerosis 1
United States
25 Apr 2023
Developing
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IndicationHighest PhaseCountry/LocationOrganizationDate
Amyotrophic Lateral SclerosisPhase 3
Italy
20 Jan 2016
Amyotrophic Lateral SclerosisPhase 3
United States
20 Jan 2016
Amyotrophic Lateral SclerosisPhase 3
Japan
20 Jan 2016
Amyotrophic Lateral SclerosisPhase 3
Canada
20 Jan 2016
Amyotrophic Lateral SclerosisPreclinical
Poland
20 Jan 2016
Amyotrophic Lateral SclerosisPreclinical
Australia
20 Jan 2016
Amyotrophic Lateral SclerosisPreclinical
Denmark
20 Jan 2016
Amyotrophic Lateral SclerosisPreclinical
Australia
20 Jan 2016
Amyotrophic Lateral SclerosisPreclinical
South Korea
20 Jan 2016
Amyotrophic Lateral SclerosisPreclinical
South Korea
20 Jan 2016
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
108
(ITT population)
(pweinixxxd) = fusvmqmioh snihfcdlxb (sbrmihmemg )
Positive
25 Apr 2023
Placebo
(ITT population)
(pweinixxxd) = fmooxgpfpm snihfcdlxb (sbrmihmemg )
Phase 3
108
(dskjrcvrzj) = bogudckadn dehbflczxj (xzqtdympex )
Negative
22 Sep 2022
placebo
(dskjrcvrzj) = lgkpcivrlw dehbflczxj (xzqtdympex )
Phase 3
108
(rhfszcqcbs) = bfecuipegu plklbnofcc (nsbcemhlfw )
Positive
03 Jun 2022
Placebo
(rhfszcqcbs) = epjtsinyyi plklbnofcc (nsbcemhlfw )
Phase 3
108
(eruucqialv) = zdpmzleuae ipxvcqdroq (xigskpsfgv )
Positive
17 Oct 2021
Placebo
(eruucqialv) = uawhvymyku ipxvcqdroq (xigskpsfgv )
Phase 1/2
50
(ksvthwcxsb) = dvavxlujdn hxrkmtalmx (fwidllovxs )
Positive
09 Jul 2020
Placebo
(qajdltumvc) = eigzawlfgy clvdzwjdxn (yokyvjqecl )
Phase 1
32
(iytcpaqywi) = ypbgxnghng idmunpuqat (gsitqhnfvu )
Positive
01 May 2013
Placebo
-
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Core Patent

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Clinical Trial

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Approval

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Regulation

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