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Synonyms: BIIB-067 | BIIB067 | Qalsody®
tofersen is an approved drug (FDA (2023), EMA (2024))
Compound class:
Nucleic acid
Comment: Tofersen (BIIB067) is an antisense oligonucleotide that disrupts translation of superoxide dismutase 1 (SOD1). Tofersen was designed to treat cases of amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) that are associated with expression of mutated SOD1. SOD1-ALS/MND accounts for ~2% of all cases.
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Classification  |
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| Compound class | Nucleic acid |
| Compound subclass | Antisense oligonucleotide (ASO) |
| Target | SOD1 mRNA |
| Approved drug? | Yes. FDA (2023) | EMA (2024) | China NMPA (2024) |
| International Nonproprietary Names |
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| INN number | INN |
| 10804 | tofersen |
| Synonyms |
| BIIB-067 | BIIB067 | Qalsody® |
| Database Links |
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| CAS Registry No. | 2088232-70-4 (source: WHO INN record) |
| ChEMBL Ligand | CHEMBL3833346 |
| DrugBank Ligand | DB14782 |
| GtoPdb PubChem SID | 500839946 |
| Search PubMed clinical trials | tofersen |
| Search PubMed titles | tofersen |
| Search PubMed titles/abstracts | tofersen |
