Compound Detail
CHEMBL3833346
TOFERSEN 💊 Approved Drug
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💊 Approved Drug
No structure available
Basic Information
| ChEMBL ID | CHEMBL3833346 |
| Name | TOFERSEN |
| Phase | Approved |
| Structure Type | SEQ |
| Therapeutic | ✓ Yes |
0
Targets
0
Activities
0
Assay Types
0
PK Parameters
0
Publications
5
Known Names
1
Indications
1
Mechanisms
Drug Information
| Molecule Type | Oligonucleotide |
| First Approval | 2023 |
| Availability | Prescription |
| Chirality | Single Enantiomer |
Mechanism of Action
| Mechanism | Action Type | Target | Direct | Efficacy |
|---|---|---|---|---|
| SOD1 mRNA antisense inhibitor | ANTISENSE INHIBITOR | SOD1 mRNA | ✓ | ✓ |
Indications
Amyotrophic Lateral Sclerosis
ATC Classification
N07XX22
tofersen
Level 1: NERVOUS SYSTEM
Level 2: OTHER NERVOUS SYSTEM DRUGS
Data from ChEMBL 36 via Core Engine