SOD1-ALS is a primarily autosomal-dominant disorder affecting approximately 2% of the ALS population. Mutations in the SOD1 gene lead to accumulation of a toxic form of SOD1 protein. Tofersen is an antisense oligonucleotide (ASO) that is complementary to a portion of the 3′ untranslated region (3′UTR) of the mRNA for human SOD1 and binds to the mRNA by Watson-Crick base pairing (hybridisation). This hybridisation of tofersen to the cognate mRNA results in RNase-Hmediated degradation of the mRNA for SOD1, which reduces the amount of SOD1 protein synthesis.
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 | |
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 | |
| QALSODY Solution for injection | European Medicines Agency (EU) | MPI, EU: SmPC |
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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