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tofersen   Click here for help

GtoPdb Ligand ID: 13536

Synonyms: BIIB-067 | BIIB067 | Qalsody®
Approved drug
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.
Classification Click here for help
Compound class Nucleic acid
Compound subclass Antisense oligonucleotide (ASO)
Target SOD1 mRNA
Approved drug? Yes. China NMPA (2024)  |  EU EMA (2024)  |  US FDA (2023)
International Nonproprietary Names Click here for help
INN number INN
10804 tofersen
Synonyms Click here for help
BIIB-067 | BIIB067 | Qalsody®
Database Links Click here for help
CAS Registry No. 2088232-70-4 (source: WHO INN record)
ChEMBL Ligand CHEMBL3833346
DrugBank Ligand DB14782
GtoPdb PubChem SID 500839946
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