Tofersen

Active ingredient description

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. Tofersen is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

Medicine classification

This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:

ATC code Group title Classification
N07XX22 N Nervous system → N07 Other nervous system drugs → N07X Other nervous system drugs → N07XX Other nervous system drugs
Discover more medicines within N07XX22

Product monographs

Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):

Title Information Source Document Type  
QALSODY Solution for injection FDA, National Drug Code (US) MPI, US: SPL/PLR

External identifiers

CAS Substance: 2088232-70-4
DrugBank Drug: DB14782
KEGG Drug: D11811
RxNorm Ingredient: 2634995
UNII Identifier: 2NU6F9601K
TOFERSEN

Medicines

Tofersen is an active ingredient of these brands:

United States (US)

Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide.

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