Tofersen is an antisense oligonucleotide indicated for the treatment of adult amyotrophic lateral sclerosis caused by SOD1 mutation. Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented.
- Mechanism
- Curator reviewed · 1 reference
Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.
- Primary indication
- Tofersen is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.Curator reviewed · 1 structured indication
- First approval
- Canada, 2025 · United States, 2023 · European Union, 2024
- Also known as
- Antisense Oligonucleotide Inhibitor Of The Expression Of Superoxide Dismutase 1 Gene · DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))R · IONIS SOD1Rx
- Code names
- BIIB 067
- Brand names
Resolves to
Compound
Clinical / RWD
Targets
What you can answer from here — as of April 11, 2025
2Protein targetsEach mapped to UniProt, with action and pharmacological actionListed above
Which drugs share a target with Tofersen, and which of those have an active Phase 3 trial?
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